About five years ago, UGBC President Jack Bracher joined the registry to become a potential stem cell donor. A few years later, Bracher found out that he had a match.

I got a call from the nonprofit that Id done it through saying that I was a match for a patient with leukemia, so I had started going through the process of being able to donate to him, Bracher, MCAS 22, said. Fortunately he went into remission, so my donation wasnt needed, but I wanted to find a way to bring back the stem cell drive to Boston Colleges campus and register more students to donate.

The Undergraduate Government of Boston College (UGBC), Student Athlete Advisory Committee (SAAC), and Project Life Movement teamed up on Thursday and Friday to encourage students to join the global registry of potential bone marrow and stem cell donors.

Project Life Movement ambassador Luke Kuechly, former linebacker for the Carolina Panthers and BC 11, returned to campus to encourage students to get swabbed.

The University last hosted a Project Life Movement bone marrow registration event when BC retired Kuechlys jersey in 2016. Over 800 people joined the registry. This year, 777 people registered, according to Bracher, with 480 students getting swabbed on the first day. Bracher said this was the most swabs Project Life Movement got on a single day at a college campus.

Steve Luquire, a co-chair of Project Life Movement, said the number of students swabbed at BC exceeds what it defines as a good number for most colleges.

On most college campuses where we go to do these drives, 200 is a good drive, Luquire said. Were gonna be here today because of SAAC and the student government, and weve already done I think close to 250 in less than two hours.

College campuses are the best place to find a healthy, diverse group willing to join the registry, according to Luquire.

My wife of 41 years died of myelodysplasia syndrome, Luquire said. Her only match was her brother, who was 60 years old, and frankly, it works so much better if you have a person who is 18 to 35. And theres no place better than a college campus to find people who are willing to look at the vision and mission and join us.

Kuechly, who met Luquire in 2013, said they have been hosting drives together since then to raise awareness and improve the chances of finding donor matches.

We just know this little bit of time that we spend today and tomorrow and having you guys come by, we can raise awareness to potentially have a match for somebody that needs it, Kuechly said.

Finding even a few donor matches, Kuechly said, is a huge deal.

You might have five to 500 to 1000 people here, but if you can get a couple donors that match, thats whats powerful, Kuechly said.

Students joining the registry, Kuechly said, is a perfect example of BC students being men and women for others.

The big pillar in the Jesuit community is how can you help other people by being selfless with your time, and this is a perfect example of it, he said.

The impact of becoming a donor goes beyond just saving a persons life, according to Ann Henegar, executive director of Project Life Movement.

When you donate your stem cells or your bone marrow to a patient, youre not only saving that persons life, youre affecting a community, youre affecting a family, you know, a workplace, a campus, Henegar said.

Henegar said she encourages people to think about the impact they can make by registering to be a donor.

This is what I tell everybody If it were your sister, your boyfriend, your girlfriend, your aunt, your child, wouldnt you want someone to say yes? she said.

For Lubens Benjamin, CSOM 23, joining the registry is a great way of fulfilling BCs mission.

I think part of being someone who goes to BC is being a person for others and, like, this is right along with that mission, Benjamin said. If I could be a match for someone, if I can help someone extend their life, thats just something great to be a part of and I dont see why Id say no to that.

Jostine Rozenich, MCAS 25, spoke to the importance of taking time out of the day to join the registry.

Its such a crucial and important thing, even if it only takes a few minutes and it can save lives, Rozenich said. I think thats all about finding ways to put service into your daily life.

Rozenich said she has family members who have needed various transplants that rely on others to donate, which has shaped her perspective on joining the registry.

Why not go ahead and do that and save the life of somebody? she said. That is such a scary feeling to not know whether or not youre going to get a match.

Ultimately, it is a privilege to be part of a drive like this, Bracher said, and hosting the event just before the Red Bandanna Gamean annual football game that commemorates Welles Crowther, the BC alumnus credited with saving the lives of about a dozen people during the Sept. 11 terrorist attacksis a great way of uniting the mission of Project Life Movement and Crowthers story.

I think its just a great privilege to be able to work with Project Life and Luke, as well as the Student Athlete Advisory Committee, who hosted the drive that I was a match for, and for all that to come full circle, Bracher said. And for us to of course be doing it on Red Bandanna weekend of all weekends means a lot.

Featured Image by Ikram Ali / Heights Editor

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Kuechly Returns to Campus for Stem Cell and Bone Marrow Registry Drive The Heights - The Heights

Bone Marrow Stem Cells Comments Off on Kuechly Returns to Campus for Stem Cell and Bone Marrow Registry Drive The Heights – The Heights | November 8th, 2021

TORONTO -- Owen Snider was given as little as three months to live. His blood cancer had returned and the prognosis was not good. The news, delivered over the phone during the height of the early pandemic lockdown in spring 2020, was devastating.

The Ottawa-area retiree began putting his affairs in order, preparing for what appeared to be inevitable.

It was terrible, his wife Judith Snider told CTV News. But we finally decided that what we had to do was to live each day not to look forward to the end, but to look forward to tomorrow.

And yet, a year later, Snider is alive -- transformed, even -- and his non-Hodgkins lymphoma is in remission. His second chance is all thanks to a promising, Canadian-run program for cancer treatment called CAR T-cell therapy.

Snider became one of the first patients to participate in a national research program that is assessing whether this experimental treatment can be done safely in Canada and cheaper than in the U.S., where costs can run upwards of half a million dollars per patient.

I think I am a pretty successful lab rat, Snider, who previously endured chemotherapy treatments and a stem cell transplant, said in an interview.

Thirty days after treatment, the lymphoma was gone. So how can you not be happy about that?

CAR T-cell therapy is a type of gene therapy that trains or engineers a patients own immune system to recognize cancerous cells. A type of white blood cell, called a T-cell, is a key component of a bodys immune system. They are developed from stem cells in the bone marrow and help fight infection and disease by searching and targeting specific foreign substances, known as antigens, in the body.

The protein receptors on T cells bind to the protein antigens on the surfaces of foreign particles that fit those receptors, like a lock and key. The foreign substance is eradicated once their antigens are bound to a T-cell. But blood cancer cells are normal cells that undergo mutations, so they are not recognized as a foreign threat to the body. In other words, T-cells generally do not have the right receptor key to fit with the antigens of a cancer cell.

CAR T-cell therapy modifies the cells so they are able to identify the cancer cells and destroy them. Its a labour-intensive process that involves taking blood from a patient and separating the T-cells. Then scientists add a gene to the cells that gives them instructions to develop an artificial receptor called a chimeric antigen receptor, or CAR.

We actually take the T-cells out and we modify them in the lab and put them back into the patient. So now they're able to recognize the cancer and kill it off, explained Dr. Kevin Hay, Medical Director for Clinical Cell Therapy with BC Cancer.

I think we're just at the cusp of really understanding what this is going to do for patients in the future.

The therapy is a labour-intensive process -- Snider's cells were shipped to Victoria, B.C to be processed in a special lab facility, then shipped back to Ottawa about a week later, where they were infused back into his body.

The treatment is still being studied, but is already available for some cancers in the U.S. and Canada at a steep price.

Researchers began trials in Canada in 2019 to see if it could be done domestically at a lower cost, highlighting the importance of having key medical production and therapies available in Canada.

We knew we had to do domestic manufacturing and if we've learned anything from COVID-19, it's that domestic capability is really important when it comes to science and medicine, and this is a perfect example of that, said Dr. Natasha Kekre, a hematologist and lead researcher on the trail based at the Ottawa Hospital.

Progress was impacted slightly by the pandemic, but Snider was fortunate enough to participate and is the first patient to come forward to discuss their experience and why he hopes the program will expand across Canada to help others dealing with otherwise untreatable forms of cancer.

Scientists are hoping to release more data in the coming months -- more than 20 patients have been treated so far, according to Dr. Kekre.

This is hopefully just the beginning for us. So this first trial was a foundation to prove that we could actually manufacture T cells, that we could do this in a clinical trial. And so this trial will remain open for patients who are in need, she said.

So absolutely we feel like were opening a door.

Snider's first experience with cancer treatment was more than a decade ago, in 2010, when he underwent a powerful and aggressive chemotherapy regimen that helped him stay cancer-free for six years.

But the treatment was so harsh that when his cancer came back in 2016, doctors told him he could not go through that kind of chemotherapy again. Instead, Snider underwent a stem cell transplant, which gave him another four years without cancer, until April 2020.

This time the outlook was grim, so doctors decided to try and get him into the CAR T-cell trials that started just before the pandemic hit. The study was specifically for patients with acute lymphoblastic leukemia and non-Hodgkins lymphoma who were not responding to other treatments.

Snider said the entire process was a walk in the park compared to what he had gone through before. He was given a mild chemotherapy treatment for three days while his T-cells were being modified in a lab on the other side of the country.

[The T-cells] went to work right away. There's a period of time where there's a lot going on inside fighting each other and that sort of thing. You don't feel great or you don't really know how you feel, Snider described. The treatment was met with outstanding success.

And in 30 days, there was no lymphoma. I couldn't believe it.

For Dr. Kekre, the results bring hope. Snider has done quite well and does not have any evidence of lymphoma at the moment, she said.

I'm unfortunately in a business where I often have to give bad news, and it is really motivating and exciting to be able to offer therapies to patients who didn't have options and to make them better, she said.

The trial is currently at the stage where scientists are making sure the product remains safe. Side-effects can include neurotoxicity, which harms the nervous system, and cytokine release syndrome, which triggers an acute system-wide inflammatory response that can result in organs not functioning properly. But so far researchers have, for the most part, been able to manage and reverse any side effects.

With such promising outcomes for patients who otherwise had no options left, researchers are talking about expanding these studies across Canada and to other forms of cancer. For now, the lab in Victoria is the only facility equipped to make these cell modifications.

I think its really going to be revolutionary with how we treat cancer in the future, not just blood cancers, but all cancers, said Dr. Hay.

Today, Snider is healthy and strong, even able to chop wood at his home near Ottawa. He and his wife Judith, a retired federal judge, are enjoying life anew.

It certainly has given us a future that we didnt know we had, she said.

The treatment not only bought Snider extra time, but also significantly improved his quality of life.

What was given to me is practically a normal life, he added.

It's really just transformed, not just extended, but transformed my life.

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CAR T-cell therapy: Hear from a Canadian patient - CTV News

Bone Marrow Stem Cells Comments Off on CAR T-cell therapy: Hear from a Canadian patient – CTV News | November 8th, 2021

PlateletBio, a company developing a new class of cell therapies based on the biology of platelets, has raised $75.5 million to advance its drug pipeline, including a lead candidate for a rare bleeding disorder on track to reach the clinic next year.

Platelets are components of blood best known for their role forming clots that stop bleeding. But Watertown, Massachusetts-based PlateletBio notes that platelets have other properties, including a role delivering growth factors and proteins throughout the body. PlateletBio is developing therapies that take advantage of these properties, but rather than using platelets from a patient or healthy donors, the startup makes them.

In the body, platelets are formed in bone marrow. PlateletBio produces its platelet-like cells, or PLCs, inside a bioreactor that mimics bone marrow conditions. The source material for its PLCs are stem cells, which have the ability to become almost any cell or tissue in the body.

Platelets are technically not cells. They dont have a nucleus, but thats an advantage for therapeutic applications. Since a PlateletBio therapy wont introduce DNA into a patients body, the potential risks that come from introducing foreign genetic material are avoided. PlateletBio says it can produce PLCs with new features and therapeutic payloads that include antibodies, signaling proteins, therapeutic proteins, and nucleic acids.

PlateletBios lead cell therapy candidate is being developed to treat immune thrombocytopenia, a blood disorder in which the immune system mistakenly sees a patients platelets as foreign and destroys them. Immune thrombocytopenia patients have dangerously low platelet counts that make them susceptible to bleeding.

There is no FDA-approved treatment for the underlying cause of immune thrombocytopenia, but corticosteroids are used to try to dampen the immune systems attack on platelets. Platelet transfusions are another option, but the National Organization for Rare Disorders notes that these treatments are usually reserved for emergencies because the platelets are likely to be destroyed by antibodies produced by the patient.

Patients who have not responded to earlier treatments have two FDA-approved small molecule options: Tavalisse, from Rigel Pharmaceuticals, and the Swedish Orphan Biovitrum drug Doptelet. Sanofi aims to treat the disease with a small molecule called rilzabrutinib. That drug is designed to block Brutons tyrosine kinase, a protein that plays a role in the development of a B cells, a type of immune cell. Sanofi acquired the molecule last year via its $3.7 billion acquisition of Principia Biopharma.

The lead disease target for the Principia drug was multiple sclerosis. In September, Sanofi reported that rilzabrutinib failed that Phase 3 study. A separate Phase 3 test in immune thrombocytopenia is ongoing, as is a mid-stage clinical trial in another autoimmune condition called IgG4-related disease.

PlateletBio isnt the only company trying to turn a component of the blood into a new type of cell therapy. Cambridge, Massachusetts-based Rubius Therapeutics is developing cell therapies based on red blood cells. After disappointing early clinical trial results in the rare disease phenylketonuria last year, Rubius shifted its focus to cancer and immune system disorders. PlateletBios PLCs would represent an entirely new approach to treating immune thrombocytopenia. According to PlateletBios website, the company plans to file an investigational new drug application for its therapeutic candidate in the first half of next year.

PlateletBio is based on the research of Harvard University scientist Joseph Italiano, who co-founded the company under the name Platelet BioGenesis. When the startup emerged in 2017, it was developing platelets that could address the platelet shortage problem facing blood donation centers. Two years ago, the startup expanded its Series A round with $26 million in additional financing and plans to make its platelets into cell therapies. Besides immune thrombocytopenia, other diseases the biotech aims to treat include osteoarthritis and liver fibrosis.

PlateletBios latest financing, a Series B round, adds new investors SymBiosis, K2 HealthVentures, and Oxford Finance. Earlier investors Ziff Capital Partners and Qiming Venture Partners also participated in the new round.

This is a major milestone for PlateletBio, adding capital and resources needed to advance our innovative platelet-like cell therapy science and manufacturing platform and support key corporate initiatives over the next 18 to 24 months, Sam Rasty, the startups president and CEO, said in a prepared statement.

Photo by Flickr user Marco Verch via a Creative Commons license

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Cell therapy biotech PlateletBio reels in $75M as it looks ahead to first clinical test - MedCity News

Bone Marrow Stem Cells Comments Off on Cell therapy biotech PlateletBio reels in $75M as it looks ahead to first clinical test – MedCity News | November 8th, 2021

TEL AVIV, Israel, Nov. 4, 2021 /PRNewswire/ --BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a late clinical-stage biopharmaceutical Company focused on oncology, today announced an oral presentation and three poster presentations at the 63rd American Society of Hematology (ASH) Annual Meeting & Exposition, which is being held December 11-14, 2021 in Atlanta, GA, and virtually.

The oral presentation will elaborate on the successful results of the Company's GENESIS Phase 3 pivotal trial. The study showed highly significant and clinically meaningful results supporting the use of Motixafortide on top of G-CSF for mobilization of stem cells for subsequent collection and transplantation in patients with multiple myeloma. In addition, the poster presentations will show that extended inhibition of the CXCR4 receptor by Motixafortide results in the mobilization of high numbers of stem cells, including specific sub-populations, which were correlated with reduced time to engraftment when infused in high numbers.

The Company is also presenting findings from in-vivo and in-vitro pre-clinical studies demonstrating that Motixafortide acts as an immunomodulator by affecting the biology of regulatory T cells (Tregs), supporting biomarker findings from the Company's COMBAT Phase 2 study in pancreatic cancer patients.

"We are very pleased with the breadth of our oral and poster presentations at this year's ASH meeting, which reflect the versatility of Motixafortide as the potential backbone of promising new treatments for both hematological and solid tumor cancers," stated Philip Serlin, Chief Executive Officer of BioLineRx. "Of particular note is the oral presentation on the outstanding results from our GENESIS Phase 3 pivotal study in stem cell mobilization demonstrating that Motixafortide effectively mobilizes a high number of cells enabling ~90% of patients to undergo transplantation following a single administration of Motixafortide and a single apheresis session. In addition, the high number of cells mobilized by Motixafortide enables infusion of an optimal number of cells, which could result in faster time to engraftment, and also allows for cryopreservation for future transplantation(s). These results, together with our recently completed successful pharmacoeconomic study, strongly support our view that Motixafortide on top of G-CSF can become the new standard of care in SCM, if approved, to the benefit of patients and payers alike. We look forward to submitting an NDA in the first half of next year, as previously communicated."

Further details of the presentations are provided below.

Oral Presentation

Title: Motixafortide (BL-8040) and G-CSF Versus Placebo and G-CSF to Mobilize Hematopoietic Stem Cells for Autologous Stem Cell Transplantation in Patients with Multiple Myeloma: The GENESIS Trial

Date: Sunday, December 12, 2021

Time: 12:00 PM

Location: Georgia World Congress Center, Hall A1

This oral presentation describes the GENESIS Phase 3 pivotal trial design, endpoints and results. The GENESIS study was a double blind, placebo controlled, multicenter trial, in which 122 patients were randomized (2:1) to receive either Motixafortide + G-CSF or placebo + G-CSF for stem cell mobilization prior to stem cell transplant in multiple myeloma patients. Total CD34+ cells/kg were analyzed on site to determine if patients mobilized to the goal and all samples were subsequently sent for assessment by a central laboratory. The number of CD34+ cells infused was determined independently by each investigator according to local practice.

The study concluded that a single administration of Motixafortide on top of G-CSF significantly increased the proportion of patients mobilizing 6x106 CD34+ cells/kg for stem cell transplantation (92.5%) vs G-CSF alone (26.2%) in up to two apheresis days (p<0.0001), while enabling 88.8% to collect 6x106 CD34+ cells/kg in just one apheresis day (vs 9.5% with G-CSF alone; p<0.0001). In addition, the median number of hematopoietic stem cells mobilized in one apheresis day with Motixafortide + G-CSF was 10.8x106 CD34+cells/kg vs 2.1x106 CD34+ cells/kg with G-CSF alone.

Poster Presentations

Title:Autologous Hematopoietic Cell Transplantation with Higher Doses of CD34+ Cells and Specific CD34+ Subsets Mobilized with Motixafortide and/or G-CSF is Associated with Rapid Engraftment A Post-hoc Analysis of the GENESIS Trial

Date: Sunday, December 12, 2021

Time: 6:00 PM - 8:00 PM

The CD34+ hematopoietic stem and progenitor cell (HSPC) dose infused during stem cell transplantation remains one of the most reliable clinical parameters to predict quality of engraftment. A minimum stem cell dose of 2-2.5x106 CD34+ cells/kg is considered necessary for reliable engraftment, while optimal doses of 5-6x106 CD34+ cells/kg are associated with faster engraftment, as well as fewer transfusions, infections, and antibiotic days.

An analysis was performed using pooled data from all patients in the GENESIS trial to evaluate time to engraftment based on the total number of CD34+ cells/kg infused, as well as specific numbers of CD34+ cell sub-populations infused.

The addition of Motixafortide to G-CSF enabled significantly more CD34+ cells to be collected in one apheresis (median 10.8x106 CD34+ cells/kg) compared to G-CSF alone (2.1x106 CD34+ cells/kg), as well as 3.5-5.6 fold higher numbers of hematopoietic stem cells (HSCs), multipotent progenitors (MPPs), common myeloid progenitors (CMPs) and granulocyte and macrophage progenitors (GMPs) (all p-values <0.0004). A dose response was observed with a significant correlation between faster time to engraftment and infusion of higher number of total CD34+ HSPC doses (6x106 CD34+ cells/kg) and combined HSC, MPP, CMP and GMP subsets. The high number of CD34+ cells/kg mobilized with Motixafortide on top of G-CSF enables the potential infusion of 6x106 CD34+ cells/kg, as well as cryopreservation of cells for later use.

Title: Immunophenotypic and Single-Cell Transcriptional Profiling of CD34+ Hematopoietic Stem and Progenitor Cells Mobilized with Motixafortide (BL-8040) and G-CSF Versus Plerixafor and GCSF Versus Placebo and G-CSF: A Correlative Study of the GENESIS Trial

Date: Monday, December 13, 2021

Time: 6:00 PM - 8:00 PM

CD34 expression remains the most common immunophenotypic cell surface marker defining human hematopoietic stem and progenitor cells (HSPCs). The addition of CXCR4 inhibitors to G-CSF has increased mobilization of CD34+ HSPCs for stem cell transplantation; yet the effect of CXCR4 inhibition, with or without G-CSF, on mobilization of specific immunophenotypic and transcriptional CD34+ HSPC subsets is not well-characterized.

Motixafortide is a novel cyclic peptide CXCR4 inhibitor with a low receptor-off rate and extended in vivo action when compared to plerixafor. GENESIS Phase 3 trial patients were prospectively randomized (2:1) to receive either Motixafortide + G-CSF or placebo + G-CSF for HSPC mobilization. Demographically similar multiple myeloma patients undergoing mobilization with plerixafor + G-CSF prior to stem cell transplant were prospectively enrolled in a separate tissue banking protocol.

Extended CXCR4 inhibition with Motixafortide + G-CSF mobilized significantly higher numbers of combined CD34+ HSCs, MPPs and CMPs compared to plerixafor + G-CSF or G-CSF alone (p<0.05). Additionally, Motixafortide + G-CSF mobilized a 10.5 fold higher number of immunophenotypically primitive CD34+ HSCs capable of broad multilineage hematopoietic reconstitution compared to G-CSF alone (p<0.0001) and similar numbers compared to plerixafor + G-CSF. Furthermore, lack of CXCR4 inhibition resulted in mobilization of more-differentiated HCSs, whereas extended CXCR4 inhibition with Motixafortide + G-CSF (but not plerixafor + G-CSF) mobilized a unique MPP-III subset expressing genes specifically related to leukocyte differentiation.

Title: The High Affinity CXCR4 Inhibitor, BL-8040, Impairs the Infiltration, Migration, Viability and Differentiation of Regulatory T Cells

Date: Sunday, December 12, 2021

Time: 6:00 PM - 8:00 PM

This poster describes results of pre-clinical in-vivo and in-vitro studies demonstrating that Motixafortide potentially acts as an immunomodulator by affecting the biology of regulatory T cells. Motixafortide reduced the amount of infiltrating Tregs into the tumors, impaired the migration of Tregs toward CXCL12 and induced Tregs cell death. Furthermore, Motixafortide was found to inhibit the differentiation of nave CD4 T cells toward Tregs.

About BioLineRx

BioLineRx Ltd. (NASDAQ/TASE: BLRX) is a late clinical-stage biopharmaceutical company focused on oncology. The Company's business model is to in-license novel compounds, develop them through clinical stages, and then partner with pharmaceutical companies for further clinical development and/or commercialization.

The Company's lead program, Motixafortide (BL-8040), is a cancer therapy platform that was successfully evaluated in a Phase 3 study in stem cell mobilization for autologous bone-marrow transplantation, has reported positive results from a pre-planned pharmacoeconomic study, and is currently in preparations for an NDA submission. Motixafortide was also successfully evaluated in a Phase 2a study for the treatment of pancreatic cancer in combination with KEYTRUDA and chemotherapy under a clinical trial collaboration agreement with MSD (BioLineRx owns all rights to Motixafortide), and is currently being studied in combination with LIBTAYO and chemotherapy as a first-line PDAC therapy.

BioLineRx is also developing a second oncology program, AGI-134, an immunotherapy treatment for multiple solid tumors that is currently being investigated in a Phase 1/2a study.

For additional information on BioLineRx, please visit the Company's website at http://www.biolinerx.com, where you can review the Company's SEC filings, press releases, announcements and events.

Various statements in this release concerning BioLineRx's future expectations constitute "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. These statements include words such as "may," "expects," "anticipates," "believes," and "intends," and describe opinions about future events. These forward-looking statements involve known and unknown risks and uncertainties that may cause the actual results, performance or achievements of BioLineRx to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Factors that could cause BioLineRx's actual results to differ materially from those expressed or implied in such forward-looking statements include, but are not limited to: the initiation, timing, progress and results of BioLineRx's preclinical studies, clinical trials and other therapeutic candidate development efforts; BioLineRx's ability to advance its therapeutic candidates into clinical trials or to successfully complete its preclinical studies or clinical trials; BioLineRx's receipt of regulatory approvals for its therapeutic candidates, and the timing of other regulatory filings and approvals; the clinical development, commercialization and market acceptance of BioLineRx's therapeutic candidates; BioLineRx's ability to establish and maintain corporate collaborations; BioLineRx's ability to integrate new therapeutic candidates and new personnel; the interpretation of the properties and characteristics of BioLineRx's therapeutic candidates and of the results obtained with its therapeutic candidates in preclinical studies or clinical trials; the implementation of BioLineRx's business model and strategic plans for its business and therapeutic candidates; the scope of protection BioLineRx is able to establish and maintain for intellectual property rights covering its therapeutic candidates and its ability to operate its business without infringing the intellectual property rights of others; estimates of BioLineRx's expenses, future revenues, capital requirements and its needs for additional financing; risks related to changes in healthcare laws, rules and regulations in the United States or elsewhere; competitive companies, technologies and BioLineRx's industry; risks related to the COVID-19 pandemic; and statements as to the impact of the political and security situation in Israel on BioLineRx's business. These and other factors are more fully discussed in the "Risk Factors" section of BioLineRx's most recent annual report on Form 20-F filed with the Securities and Exchange Commission on February 23, 2021. In addition, any forward-looking statements represent BioLineRx's views only as of the date of this release and should not be relied upon as representing its views as of any subsequent date. BioLineRx does not assume any obligation to update any forward-looking statements unless required by law.

Contact:

Tim McCarthyLifeSci Advisors, LLC+1-212-915-2564[emailprotected]

or

Moran MeirLifeSci Advisors, LLC+972-54-476-4945[emailprotected]

SOURCE BioLineRx Ltd.

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BioLineRx Announces an Oral Presentation and Three Poster Presentations at the 63rd American Society of Hematology (ASH) Annual Meeting &...

Bone Marrow Stem Cells Comments Off on BioLineRx Announces an Oral Presentation and Three Poster Presentations at the 63rd American Society of Hematology (ASH) Annual Meeting &… | November 8th, 2021

Two kidney transplant patients who received a stem cell therapy developed by Talaris Therapeutics were able to come off all immunosuppressant drugs within a year, without any evidence of graft rejection.

The first findings from Talaris phase 3 trial of the cell therapy called FCR001 suggest it may be possible to eliminate the need entirely for patients to take what may be dozens of tablets daily after organ transplants, according to the US biotech.

While still preliminary, the experience with the two patients back up Talaris hope that giving a one-shot dose of FCR001 the day after an organ transplant could stimulate immune tolerance in the recipient, and avoid the side effects of current drug treatments such as infections, heart disease, and some forms of cancer.

The companys approach relies on administering haematopoietic stem cells from the individual who donated the organ, in order to generate what Talaris refers to as chimerism, with both donor and recipient cells present in the bone marrow. That allows the immune system to see the transplanted organ as self rather than foreign.

The first two recipients in Talaris FREEDOM-1 phase 3 trial had received FCR001 at least 12 months earlier, and showed stable kidney function, according to Talaris.

A larger group of five patients who were at least three months from the cell therapy maintained more than 50% chimerism in their T cells, which the biotech said was a sign of long-term, immunosuppression-free tolerance to the donated kidney in its phase 2 trials.

The FREEDOM-1 results reported at the American Society of Nephrology (ASN) meeting this week were accompanied by updated results from Talaris phase 2 study, in which all 26 patients originally weaned off immunosuppressants have continued to remain off them without rejecting their donated kidney.

Some transplant patients treated with Talaris therapy in earlier trials have now been off all immunosuppression for more than 12 years without signs of kidney rejection.

Talaris intends to enrol 120 subjects into the phase 3 trial, which is scheduled to generate results in 2023.

Earlier this year, Talaris raised $150 million via a Nasdaq listing that will be used to take FCR001 through the phase 3 programme in organ transplantation and as a treatment for rare autoimmune disease scleroderma.

It also recently started a phase 2 trial of the cell therapy to see if it is able to induce immune tolerance to a transplanted kidney in patients who received the transplant from a living donor up to a year prior to administration of FCR001.

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Talaris therapy ends need for immune drugs in transplant patients - - pharmaphorum

Bone Marrow Stem Cells Comments Off on Talaris therapy ends need for immune drugs in transplant patients – – pharmaphorum | November 8th, 2021

Rachel B. Salit, MD, discussed the case of a 48-year-old patient with graft-versus-host-disease.

Rachel B. Salit, MD, associate professor, Clinical Research Division, Fred Hutchinson Cancer Research Center at the University of Washington School of Medicine in Seattle, WA, discussed the case of a 48-year-old patient with graft-versus-host-disease.

Targeted OncologyTM: What are your thoughts on the currently accepted options for acute GVHD (aGVHD) prophylaxis?

SALIT: Between calcineurin inhibitors, if we have a choice, my preference is usually tacrolimus. Tacrolimus is better tolerated [than cyclosporin] in terms of adverse events [AEs], blood pressure, kidney function, and [even] the smell.

Methotrexate is a tried-and-true prophylaxis, especially in the myeloablative or high-intensity transplant setting. [In contrast], mycophenolate mofetil [MMF]; [CellCept] is usually used in the nonmyeloablative or reduced-intensity setting. When calcineurin inhibitors were used with MMF as prophylaxis for GVHD, the GVHD was higher. Thats why we [use] methotrexate [instead of MMF].1

Sirolimus [Rapamune] is often combined with a calcineurin inhibitor and MMF, or with a calcineurin inhibitor and methotrexate. Sirolimus is very well tolerated, except for some triglyceride AEs. Additionally, the combination of sirolimus plus MMF and a calcineurin inhibitor has been shown to significantly decrease GVHD in the reduced-intensity setting compared with [the effect observed with] MMF and a calcineurin inhibitor alone.2

CAR [chimeric antigen receptor] T-cellantibody therapy plus antithymocyte globulin [ATG] and alemtuzumab [Lemtrada] are more frequently used in Europe [than in the United States]. There have been mixed results, and there is some concern of increased relapse with [anti-thymocyte globulin (ATG) therapy]. Ex vivo T-cell depletion and CD34-positive cell selection [are] also uncommon in the United States.

Posttransplant cyclophosphamide [Cytoxan] [is becoming more common], and it was originally [used in the setting of] haploidentical transplants. Now it is increasingly used in the unrelated donor setting, and Im sure it will be translated to the sibling setting, too. [This regimen] has been shown to decrease effector T cells.3 Moreover, chronic GVHD is [reduced by this regimen], but aGVHD is not changed.

A recent study retrospectively compared many patients [with haploidentical donors] to a smaller number of patients [who had] unrelated donors and who received posttransplant cyclophosphamide. The data showed that the patients with unrelated donors and posttransplant cyclophosphamide had better overall survival [OS] and decreased relapse compared with the patients with haploidentical donors.4

For a long time, [most trials that compared GVHD and OS between patients with haploidentical] vs unrelated or sibling donors have shown that posttransplant [cyclophosphamide in the setting of haploidentical] transplants is associated with reduced chronic GVHD, but the other outcomes were the same. Is this result attributable to the fact that the transplant is haploidentical, or is it attributable to the posttransplant cyclophosphamide? I think that question will be answered within the next [few] years.

What risk factors for GVHD do you notice in the case described?

There are multiple risk factors. The fact that the donor is multiparous puts the recipient at higher risk for GVHD. The patients high intensity, myeloablative conditioning regimen increases the risk for GVHD, as do the donors CMV seropositivity and the fact that the patient and donor are not sex matched. The risk of GVHD also increases with donor age.

Risk for GVHD is also increased by major human leukocyte antigen [HLA] disparity. We look at class I [HLA-A, -B, and -E] and class II [HLA-DR and -DQ] antigens, with a 10 out of 10 score constituting a match. There are data coming out that show that the class II antigen HLA-DP also matters in certain cases5; a match that includes this antigen [a 12 out of 12 (score)] is better than a 10 out of 10 [score]. [This patients donor was HLA-matched, but] minor HLA mismatches can increase the risk of GVHD [in patients like this one whose donor is unrelated].

Stem cell source and graft composition are other considerations, but this patient received peripheral blood, which confers a higher risk of GVHD than does bone marrow. Peripheral blood has a higher CD34-positive cell count, therefore a higher T-cell dose; both factors increase GVHD risk. At our center, we dont often cap CD34-positive cell count or T-cell dose, except in the haploidentical setting.

I would not include ABO blood type as a risk factor. There are mixed data regarding whether major and minor ABO mismatches lead to increased GVHD.6

What standardized guidelines exist for organ staging and grading in the context of aGVHD?

[According to Mount Sinai Acute GVHD International Consortium], the skin, the liver, and gastrointestinal [GI] tract are the 3 organs included in aGVHD staging. The skin is [described in terms of] the percentage of body surface area [BSA] affected. Stage 0 is no rash, stage 1 is a rash covering less than 25% of the BSA, stage 2 is a rash covering 25% to 50% of the BSA, stage 3 is a rash covering greater than 50% of the BSA, and stage 4 is generalized erythroderma.7

According to the liver status [bilirubin level], staging starts at stage 0 [less than 2 mg/dL] and progresses through stage 1 [2-3 mg/dL], stage 2 [3.1-6 mg/dL], and stage 3 [6.1-15 mg/dL] to a final stage of 4 [greater than 15 mg/dL]. The lower GI staging system [counts] the number of episodes per day of liquid stool output. Stage 0 is fewer than 3 episodes [of stool output], stage 1 is 3 to 4 episodes, stage 2 is 5 to 7 episodes, and stage 3 is greater than 7 episodes. If you have an inpatient, then you can use these exact quantities. If you have an outpatient, you can use these values as rough markers. Regarding the upper GI staging system, in stage 0, nausea, vomiting, or anorexia are absent or intermittent, but in stage 1, they are persistent.

The other thing I often look at [to judge] severity [of GI involvement] is the electrolytes. For example, if the patient says they are having 5 episodes of stool a day, but their potassium and magnesium are normal and theyre not becoming acidemic, then you [might consider that] these stools are only of small volume. If the patient starts to have electrolyte abnormalities or starts to become acidemic, then you [should consider that] maybe theyre having more diarrhea than theyre [telling you].

When we grade according to most severe target organ involvement, grade I reflects the presence only of stage 1 to 2 skin involvement. Any GI or liver involvement is automatically [at least grade] II, the point at which you would consider treating symptoms topically. Grade IIA indicates upper GI involvement, and grade IIB indicates lower GI involvement.8 Once the patient gets to grade III, they almost always [require] systemic therapy.7

What stage and grade would you give this patient?

With 60% skin involvement, he would have a skin stage of 3, and with 4 episodes of diarrhea per day, he would have a lower GI stage of 1. [He would have an overall clinical grade of IIB.]

What are GVHD and aGVHD biomarkers, and how are they used?

Biomarkers of GVHD are markers of inflammation found in the blood that will tell you the patient is at a higher risk for developing GVHD. These biomarkers include elafin, IL-2 receptor-, IL-8, tumor necrosis factor receptor-1, hepatocyte growth factor, and regenerating islet-derived 3- [NCT00224874].9 The use of biomarkers [to predict patients risk of developing GVHD could guide physicians as they choose] a starting steroid dosage, eg, 2 mg/kg vs 1 mg/kg.

What data guide your decisions about steroid therapy in GVHD?

Although the concept of GVHD and aGVHD risk stratification is not generally used in practice, high-risk GVHD vs standard-risk GVHD has been shown to be associated with a lower rate of complete response to steroids [27% vs 48%, respectively; P < .001] and higher treatment-related mortality [incidence at 6 months after steroid therapy onset, 44% vs 22%, respectively; P < .001].10 If a patient has a higher grade of GVHD, they are more likely to be steroid refractory.

The steroid response of GVHD is classified as steroid refractory or resistant if GVHD progresses within the first 3 to 5 days of prednisone therapy onset [ 2 mg/kg per day], fails to improve within 5 to 7 days of treatment initiation at 1 mg/kg or shows an incomplete response after more than 28 days [of immunosuppressive treatment including steroids]. Steroid dependence [means that either] the prednisone cannot be tapered below 2 mg/kg daily or the GVHD recurs during the steroid taper.11

You cant really [know] who is going to respond to steroids without [trying]. Our initial treatment for any patient with GVHD is steroids. There are no data to suggest that we [should] add something other than steroids as the first line or that we [should] add double therapy for the first line. Its going to be different for every individual.

Also, regarding steroid therapy, the question has been raised: If patients receive higher doses sooner, will that result in a lower [total] exposure to steroids? In the study we did at our institution, we found that when patients with skin GVHD were randomly assigned [to receive] 1 mg/kg vs 0.5 mg/kg, patients [who received the lower dose] had a longer and higher overall exposure to steroids.12 [In cases of skin GVHD], we tend to undertreat patients, and it may help to give them at least 1 mg/kg, but for GI GVHD, we usually give 1 mg/kg. It may not help to give 2 mg/kg unless the GVHD is severe.

Other than steroids, what therapy options exist for aGVHD, according to the National Comprehensive Cancer Network (NCCN)?

Ruxolitinib [Jakafi] is the only approved therapy, and it is supported by category 1 evidence. Some other therapies, such as MMF and sirolimus, are [relatively] benign. Other treatments, like ATG, are more toxic, whereas extracorporeal photopheresis [ECP] doesnt have a lot of data [to support it]. However, we do use a lot of ECP, [primarily for] steroid-dependent GVHD of the skin.13

What data support the use of ruxolitinib for aGVHD?

In the REACH1 study [NCT02953678], patients with steroid refractory grade 2 to 4 aGVHD received ruxolitinib, 5 mg twice a day. Later, patients could increase to 10 mg twice a day.14,15

The overall response rate [ORR] at day 28 was about 55%. The best ORR at any time during treatment was 73%. Time to response was about 7 days [range, 6-49]. The median duration of response was almost a year. Death from causes other than malignancy relapse was found in about 50% of patients. The median OS was about 5 months, whereas median OS for steroid refractory GVHD was 1 month, [but median OS for day 28 responders was not reached].16,17 The overall response rate [ORR] at day 28 was about 55%. The best ORR at any time during treatment was 73%. Time to response was about 7 days [range, 6-49]. The median duration of response was almost a year. Death from causes other than malignancy relapse was found in about 50% of patients. The median OS was about 5 months, whereas median OS for steroid-refractory GVHD was 1 month, [but median OS for day 28 responders was not reached].15

The ORR at day 28 was 62% in the ruxolitinib group vs 39% in the control group [odds ratio (OR), 2.64; 95% CI, 1.65-4.22; P < .001]; the durable ORR at day 56 was 40% in the ruxolitinib group vs 22% in the control group [OR, 2.38; 95% CI, 1.43-3.94; P < .001].18 These results led to the FDA approval of ruxolitinib for second-line therapy for steroid-refractory aGVHD.19

[Separate analyses were conducted of] GI and skin GVHD. In the ruxolitinib group, aGVHD staging of the lower GI was stage 3 and 4 for most patients at baseline. This was reduced in most patients to stage 0, 1, and 2 by day 28. In contrast, most patients treated with BAT still presented with stage 2 to 4 GVHD by day 28. Likewise for the skin, the GVHD stage was more likely to decrease following treatment with ruxolitinib than with BAT.19

Median failure-free survival was 5 months in the ruxolitinib group vs 1 month in the BAT group [HR, 0.46; 95% CI, 0.35-0.60]; 5 months was a big achievement compared with our previous standard. After 1 year, 40% of the patients in the experimental group were still alive. Regarding AEs associated with ruxolitinib, the most difficult [AE to manage] is thrombocytopenia [in REACH2, affecting 33% of the ruxolitinib group vs 18% of the BAT group]. Infections with ruxolitinib [in the context of GVHD] probably are equivalent to [those observed with] any other immune suppression drug [for cytomegalovirus, 26% in the ruxolitinib group, 21% in the BAT group].19

REFERENCES:

1. Yoshida S, Ohno Y, Nagafuji K, et al. Comparison of calcineurin inhibitors in combination with conventional methotrexate, reduced methotrexate, or mycophenolate mofetil for prophylaxis of graft-versus-host disease after umbilical cord blood transplantation. Ann Hematol. 2019;98(11):2579-2591. doi:10.1007/s00277-019-03801-z

2. Bejanyan N, Rogosheske J, DeFor TE, et al. Sirolimus and mycophenolate mofetil as calcineurin inhibitor-free graft-versus-host disease prophylaxis for reduced-intensity conditioning umbilical cord blood transplantation. Biol Blood Marrow Transplant. 2016;22(11):2025-2030. doi:10.1016/j. bbmt.2016.08.005

3. Wodarczyk M, Ograczyk E, Kowalewicz-Kulbat M, Druszczyska M, Rudnicka W, Fol M. Effect of cyclophosphamide treatment on central and effector memory T cells in mice. Int J Toxicol. 2018;37(5):373-382.

4. Shaw BE. Related haploidentical donors are a better choice than matched unrelated donors: counterpoint. Blood Adv. 2017;1(6):401-406. doi:10.1182/bloodadvances.2016002188

5. Zachary AA, Leffell MS. HLA mismatching strategies for solid organ transplantation - a balancing act. Front Immunol. 2016;7:575. doi:10.3389/ fimmu.2016.00575

6. Brierley CK, Littlewood TJ, Peniket AJ, et al. Impact of ABO blood group mismatch in alemtuzumab-based reduced-intensity conditioned haematopoietic SCT. Bone Marrow Transplant. 2015;50(7):931-938. doi:10.1038/bmt.2015.51

7. Harris AC, Young R, Devine S, et al. International, multicenter standardization of acute graft-vs-host disease clinical data collection: a report from the Mount Sinai Acute GVHD International Consortium. Biol Blood Marrow Transplant. 2016;22(1):4-10. doi:10.1016/j.bbmt.2015.09.001

8. Lee SJ. Classification systems for chronic graft-versus-host disease. Blood. 2017;129(1):30-37. doi:10.1182/blood-2016-07-686642

9. Levine JE, Logan BR, Wu J, et al. Acute graft-vs-host disease biomarkers measured during therapy can predict treatment outcomes: a Blood and Marrow Transplant Clinical Trials Network study. Blood. 2012;119(16):3854-3860. doi:10.1182/blood-2012-01-403063

10. MacMillan ML, Robin M, Harris AC, et al. A refined risk score for acute graft-vs-host disease that predicts response to initial therapy, survival, and transplant-related mortality. Biol Blood Marrow Transplant. 2015;21(4):761-767. doi:10.1016/j.bbmt.2015.01.001

11. Schoemans HM, Lee SJ, Ferrara JL, et al; European Society for Blood and Marrow Transplantation [EBMT] Transplant Complications Working Party; EBMT-National Institutes of Health [NIH]-Center for International Blood and Marrow Transplant Research [CIBMTR] GVHD Task Force. EBMT-NIH-CIBMTR Task Force position statement on standardized terminology & guidance for graft-vs-host disease assessment. Bone Marrow Transplant. 2018;53(11):1401-1415. doi:10.1038/s41409-018-0204-7

12. Mielcarek M, Furlong T, Storer BE, et al. Effectiveness and safety of lower dose prednisone for initial treatment of acute graft-versus-host disease: a randomized controlled trial. Haematologica. 2015;100(6):842-848. doi:10.3324/haematol.2014.118471

13. NCCN. Clinical Practice Guidelines in Oncology. Hematopoietic cell transplantation, version 5.2021. Accessed October 13, 2021. https://www.nccn.org/professionals/physician_gls/pdf/hct.pdf

14. Chao N. Finally, a successful randomized trial for GVHD. N Engl J Med. 2020;382(19):1853-1854. doi:10.1056/NEJMe2003331

15. Jagasia M, Zeiser R, Arbushites M, Delaite P, Gadbaw B, von Bubnoff N. Ruxolitinib for the treatment of patients with steroid-refractory GVHD: an introduction to the REACH trials. Immunotherapy. 2018;10(5):391-402. doi:10.2217/ imt-2017-0156

16. Jagasia M, Perales MA, Schroeder MA, et al. Ruxolitinib for the treatment of steroid-refractory acute GVHD (REACH1): a multicenter, open-label phase 2 trial. Blood. 2020;135(20):1739-1749. doi:10.1182/blood.2020004823

17. Jagasia M, Ali H, Schroeder MA, et al. Ruxolitinib in combination with corticosteroids for the treatment of steroid-refractory acute graft-vs-host disease: results from the phase 2 REACH1 trial. Biol Blood Marrow Transplant. 2019;25(suppl 3):S52. doi:10.1016/j.bbmt.2018.12.130

18. Zeiser R, von Bubnoff N, Butler J, et al; REACH2 Trial Group. Ruxolitinib for glucocorticoid-refractory acute graft-vs-host disease. N Engl J Med. 2020;382(19):1800-1810. doi:10.1056/NEJMoa1917635

19. Przepiorka D, Luo L, Subramaniam S, et al. FDA approval summary: ruxolitinib for treatment of steroid-refractory acute graft-versus-host disease. Oncologist. 2020;25(2):e328-e334. doi:10.1634/theoncologist.2019-0627

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Salit Discusses the Use of Staging and Grading for Patients With GVHD to Choose Appropriate Treatment - Targeted Oncology

Bone Marrow Stem Cells Comments Off on Salit Discusses the Use of Staging and Grading for Patients With GVHD to Choose Appropriate Treatment – Targeted Oncology | November 8th, 2021

Updates from OTL-201 Clinical Proof-of-Concept Study in MPS-IIIA and OTL-204 Preclinical Study for GRN-FTD at ESGCT Showcase Potential for HSC Gene Therapy in Multiple Neurodegenerative Disorders

Launch Activities for Libmeldy Across Key European Countries, including Reimbursement Discussions, Progressing in Anticipation of Treating Commercial Patients

Frank Thomas, President and Chief Operating Officer, to Step Down Following Transition in 2022; Search for a Chief Financial Officer Initiated

Cash and Investments of Approximately $254M Provide Runway into First Half 2023

BOSTONandLONDON, Nov. 04, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today reported financial results for the quarter ended September 30, 2021, as well as recent business updates and upcoming milestones.

This quarter, we are pleased by the progress demonstrated by our investigational neurometabolic HSC gene therapy programs with promising preclinical and clinical updates at ESGCT, said Bobby Gaspar, M.D., Ph.D., chief executive officer of Orchard. With follow-up in OTL-201 for MPS-IIIA patients now ranging between 6 and 12 months, biomarker data remain highly encouraging, showing supraphysiological enzyme activity and corresponding substrate reductions in the CSF and urine. The launch strategy for Libmeldy is also advancing in Europe with momentum building on reimbursement discussions and patient finding activities.

Recent Presentations and Business Updates

Data presentations at ESGCT

Clinical and pre-clinical data from across the companys investigational hematopoietic stem cell (HSC) gene therapy portfolio were featured in two oral and seven poster presentations at the European Society of Gene & Cell Therapy Congress (ESGCT) on October 19-22. Highlights from key presentations are summarized below:

OTL-201 for Mucopolysaccharidosis type IIIA (MPS-IIIA): A poster presentation featured supportive biomarker data from the first four patients with evaluable results, with duration of follow-up ranging from 6 to 12 months. The treatment has been generally well-tolerated in all enrolled patients (n=5) with no treatment-related serious adverse events (SAEs). Supraphysiological N-sulphoglucosamine sulphohydrolase ( SGSH) enzyme activity above the normal range was seen in leukocytes and plasma within one to three months in all evaluable patients (n=4).A greater than 90% reduction in urinary glycosaminoglycans (GAGs) was seen within three months in all evaluable patients (n=4).SGSH activity in the cerebrospinal fluid (CSF) increased from undetectable at baseline to within or above the normal range by six months in all patients with available data (n=3).CSF GAGs decreased from baseline in patients with available data (n=3).OTL-204 for Progranulin-mutated Frontotemporal Dementia (GRN-FTD): Preliminary in vivo data from the preclinical proof-of-concept study showed that murine GRN -/- HSPCs, transduced with an LV expressing progranulin under the control of a novel promoter, are able to engraft and repopulate the brain myeloid compartment of FTD mice and to locally deliver the GRN enzyme.

R&D Investor Event Summary

In September, Orchard hosted an R&D investor event highlighting its discovery and research engine in HSC gene therapy, including an update on the OTL-104 program in development for NOD2 Crohns disease (NOD2-CD) and potential new applications in HSC-generated antigen-specific regulatory T-cells (Tregs) and HSC-vectorization of monoclonal antibodies (mAbs).

The discussion also covered the differentiated profile of Orchards HSC gene therapy approach, which has exhibited favorable safety, long-term durability and broad treatment applicability.

In particular, Orchards lentiviral vector-based HSC gene therapy programs have shown no indication of insertional oncogenesis and no evidence of clonal dominance due to integration into oncogenes. Importantly, the promoters and regulatory elements of Orchard vectors are derived from human (not viral) sequences and are specifically designed to have limited enhancer activity on neighboring genes thereby mitigating the potential for safety concerns.In addition, because of the fundamental biological differences between the HSC and adeno-associated virus (AAV) gene therapy approaches, Orchards programs have not, to date, seen the safety and durability concerns experienced by the AAV gene therapy field.

Libmeldy (atidarsagene autotemcel) launch in Europe

Orchard is providing an update on the following key launch activities for Libmeldy in Europe:

Discussions with health authorities and payors are underway across Europe in key markets including Germany, the UK, France and Italy.Qualification of treatment centers is progressing with The University of Tbingen in Germany ready to treat commercial patients and other centers in the final stages of qualification and treatment readiness.Disease awareness and patient identification activities continue and have supported patient referrals in major European centers. Orchards partnerships in the Middle East and Turkey allow for opportunities to treat eligible patients from these territories at qualified European centers.Orchard is providing sponsorship for an ongoing newborn screening pilot in Germany and is working with laboratories to implement pilots in Italy, the UK, France and Spain.

Executive organizational update

The company also announced that Frank Thomas will step down from his role as president and chief operating officer, following a transition in 2022. A search for a chief financial officer is underway. Mr. Thomas other responsibilities will be assumed by existing members of the leadership team in commercial and corporate affairs. Orchard recently strengthened the executive team with the appointments of Nicoletta Loggia as chief technical officer and Fulvio Mavilio as chief scientific officer and the promotion of Leslie Meltzer to chief medical officer.

I want to extend my gratitude to Frank Thomas for his immense contributions to Orchard, said Gaspar. During his tenure, Frank oversaw the transition of the organization to a publicly traded company and has managed operations with a focus on cross-company innovation, including his role as a key architect in creating and executing the focused business plan we rolled out in 2020. Along with the entire board of directors and leadership team, I appreciate Franks commitment to facilitate a smooth transition during this time.

Gaspar continued, Our search is focused on a CFO to lead the broad strategic planning efforts necessary to capitalize on the full potential of our hematopoietic stem cell gene therapy platform. We have a strong team in place to aid Orchards success in this next phase of growth and are well capitalized through the anticipated completion of several value-creating milestones.

Upcoming Milestones

In June 2021, Orchard announced several portfolio updates following recent regulatory interactions for the companys investigational programs in metachromatic leukodystrophy (MLD), Mucopolysaccharidosis type I Hurler syndrome (MPS-IH) and Wiskott-Aldrich syndrome (WAS).

OTL-200 for MLD in the U.S: Based on feedback received from the U.S. Food and Drug Administration (FDA), the company is preparing for a Biologics License Application (BLA) filing for OTL-200 in pre-symptomatic, early-onset MLD in late 2022 or early 2023, using data from existing OTL-200 patients. This approach and timeline are subject to the successful completion of activities remaining in advance of an expected pre-BLA meeting with FDA, including future CMC regulatory interactions and demonstration of the natural history data as a representative comparator for the treated population.OTL-203 for MPS-IH: Orchard is incorporating feedback from FDA and the European Medicines Agency (EMA) into a revised global registrational study protocol, with study initiation expected to occur in 2022.OTL-201 for MPS-IIIA: Additional interim data from this proof-of-concept study are expected to be presented at medical meetings in 2022, including early clinical outcomes of cognitive function.OTL-103 for WAS: The company expects a MAA submission with EMA for OTL-103 in WAS in 2022, subject to the completion of work remaining on potency assay validation and further dialogue with EMA. The company will provide updated guidance for a BLA submission in the U.S. following additional FDA regulatory interactions.

Third Quarter 2021 Financial Results

Revenue from product sales of Strimvelis were $0.7 million for the third quarter of 2021 compared to $2.0 million in the same period in 2020, and cost of product sales were $0.2 million for the third quarter of 2021 compared to $0.7 million in the same period in 2020. Collaboration revenue was $0.5 million for the third quarter of 2021, resulting from the collaboration with Pharming Group N.V. entered into in July 2021. This revenue represents expected reimbursements for preclinical studies and a portion of the $17.5 million upfront consideration received by Orchard under the collaboration, which will be amortized over the expected duration of the agreement.

Research and development (R&D) expenses were $20.8 million for the third quarter of 2021, compared to $14.7 million in the same period in 2020. The increase was primarily due to higher manufacturing and process development costs for the companys neurometabolic programs and lower R&D tax credits as compared to the same period in 2020. R&D expenses include the costs of clinical trials and preclinical work on the companys portfolio of investigational gene therapies, as well as costs related to regulatory, manufacturing, license fees and development milestone payments under the companys agreements with third parties, and personnel costs to support these activities.

Selling, general and administrative (SG&A) expenses were $13.0 million for the third quarter of 2021, compared to $13.0 million in the same period in 2020. SG&A expenses are expected to increase in future periods as the company builds out its commercial infrastructure globally to support additional product launches following regulatory approvals.

Net loss was $36.4 million for the third quarter of 2021, compared to $20.3 million in the same period in 2020. The increase in net loss as compared to the prior year was primarily due to higher R&D expenses as well as the impact of foreign currency transaction gains and losses. The company had approximately 125.5 million ordinary shares outstanding as of September 30, 2021.

Cash, cash equivalents and investments as of September 30, 2021, were $254.1 million compared to $191.9 million as of December 31, 2020. The increase was primarily driven by net proceeds of $143.6 million from the February 2021 private placement and $17.5 million in upfront payments from the July 2021 collaboration with Pharming Group N.V., offset by cash used for operating activities and capital expenditures. The company expects that its cash, cash equivalents and investments as of September 30, 2021 will support its currently anticipated operating expenses and capital expenditure requirements into the first half of 2023. This cash runway excludes an additional $67 million that could become available under the companys credit facility and any non-dilutive capital received from potential future partnerships or priority review vouchers granted by the FDA following future U.S. approvals.

About Libmeldy / OTL-200 Libmeldy (atidarsagene autotemcel), also known as OTL-200, has been approved by the European Commission for the treatment of MLD in eligible early-onset patients characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity in children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. Libmeldy is the first therapy approved for eligible patients with early-onset MLD. The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies. In addition to the risks associated with the gene therapy, treatment with Libmeldy is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies, the safety profiles of these interventions were consistent with their known safety and tolerability. For more information about Libmeldy, please see the Summary of Product Characteristics (SmPC) available on the EMA website. Libmeldy is approved in the European Union, UK, Iceland, Liechtenstein and Norway. OTL-200 is an investigational therapy in the US.

Libmeldy was developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. About Orchard

At Orchard Therapeutics, our vision is to end the devastation caused by genetic and other severe diseases. We aim to do this by discovering, developing and commercializing new treatments that tap into the curative potential of hematopoietic stem cell (HSC) gene therapy. In this approach, a patients own blood stem cells are genetically modified outside of the body and then reinserted, with the goal of correcting the underlying cause of disease in a single treatment.

In 2018, the company acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Today, Orchard has a deep pipeline spanning pre-clinical, clinical and commercial stage HSC gene therapies designed to address serious diseases where the burden is immense for patients, families and society and current treatment options are limited or do not exist.

Orchard has its global headquarters inLondonandU.S. headquarters inBoston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website ( http://www.orchard-tx.com ), the investor relations website ( ir.orchard-tx.com ), and on social media ( Twitter and LinkedIn ), including but not limited to investor presentations and investor fact sheets,U.S. Securities and Exchange Commissionfilings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Forward-Looking Statements

This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, including its plans and expectations for the commercialization of Libmeldy, the therapeutic potential of Libmeldy (OTL-200) and Orchards product candidates, including the product candidates referred to in this release, Orchards expectations regarding its ongoing preclinical and clinical trials, including the timing of enrollment for clinical trials and release of additional preclinical and clinical data, the likelihood that data from clinical trials will be positive and support further clinical development and regulatory approval of Orchard's product candidates, and Orchards financial condition and cash runway into the first half of 2023. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation: the risk that prior results, such as signals of safety, activity or durability of effect, observed from clinical trials of Libmeldy will not continue or be repeated in our ongoing or planned clinical trials of Libmeldy, will be insufficient to support regulatory submissions or marketing approval in the US or to maintain marketing approval in the EU, or that long-term adverse safety findings may be discovered; the risk that any one or more of Orchards product candidates, including the product candidates referred to in this release, will not be approved, successfully developed or commercialized; the risk of cessation or delay of any of Orchards ongoing or planned clinical trials; the risk that Orchard may not successfully recruit or enroll a sufficient number of patients for its clinical trials; the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates; the delay of any of Orchards regulatory submissions; the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates or the receipt of restricted marketing approvals; the inability or risk of delays in Orchards ability to commercialize its product candidates, if approved, or Libmeldy, including the risk that Orchard may not secure adequate pricing or reimbursement to support continued development or commercialization of Libmeldy; the risk that the market opportunity for Libmeldy, or any of Orchards product candidates, may be lower than estimated; and the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development, its supply chain and commercial programs. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards quarterly report on Form 10-Q for the quarter endedSeptember 30, 2021, as filed with theU.S. Securities and Exchange Commission(SEC), as well as subsequent filings and reports filed with theSEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Contacts

Investors Renee Leck Director, Investor Relations +1 862-242-0764 Renee.Leck@orchard-tx.com

Media Benjamin Navon Director, Corporate Communications +1 857-248-9454 Benjamin.Navon@orchard-tx.com

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Orchard Therapeutics Reports Third Quarter 2021 Financial Results and Highlights Recent ... - KULR-TV

Bone Marrow Stem Cells Comments Off on Orchard Therapeutics Reports Third Quarter 2021 Financial Results and Highlights Recent … – KULR-TV | November 8th, 2021

SAN DIEGO, Oct. 27, 2021 (GLOBE NEWSWIRE) -- Sorrento Therapeutics, Inc. (Nasdaq: SRNE, "Sorrento") announced today positive preliminary results from two Phase 2 studies designed to identify the hospitalized patient population suffering from COVID-19-induced pneumonia and respiratory depression likely to respond to treatment with oral Abivertinib. Abivertinib is a novel small molecule tyrosine kinase inhibitor (TKI) that selectively targets both mutant forms of the epidermal growth factor receptor (EGFR) as well as Bruton's tyrosine kinase (BTK). Abivertinib irreversibly binds to the BTK receptor, preventing the phosphorylation of the receptor. Due to this effect, it has shown potent immunomodulatory activities in vitro with potent inhibition of key pro-inflammatory cytokine production, including IL-1 beta, IL-6 and TNF-alpha. These cytokines are associated with acute respiratory distress syndrome (ARDS), and with cytokine release syndrome (CRS) or cytokine storm, and COVID-19 disease progression with poor outcomes in patients.

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UPDATE  – Sorrento Announces Encouraging Results From Two Phase 2 Studies of Abivertinib For Treatment Of Hospitalized Severe COVID-19 Patients

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Daix (France), Long Island City (New York, United States), October 27, 2021 – Inventiva (Euronext Paris and Nasdaq: IVA), a clinical-stage biopharmaceutical company focused on the development of oral small molecule therapies for the treatment of NASH, mucopolysaccharidoses (MPS) and other diseases with significant unmet medical needs, today announced the design of a proof-of-concept Phase IIa combination trial with its lead drug candidate lanifibranor and the SGLT2 inhibitor empagliflozin in patients with T2D and non-cirrhotic NASH.

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Inventiva announces the design of LEGEND, a Phase IIa combination trial with lanifibranor and SGLT2 inhibitor empagliflozin in patients with NASH and...

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MALVERN, Pa., Oct. 27, 2021 (GLOBE NEWSWIRE) -- TELA Bio, Inc. ("TELA"), a commercial-stage medical technology company focused on designing, developing, and marketing innovative tissue reinforcement materials to address unmet needs in soft tissue reconstruction, today announced that the company will report third quarter 2021 financial results on Wednesday, November 10, 2021. TELA Bio’s management will host a conference call and webcast at 4:30 p.m. ET that day to discuss the financial results and provide a corporate update.

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TELA Bio to Announce Third Quarter 2021 Financial Results

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BOSTON and LAUSANNE, Switzerland, Oct. 27, 2021 (GLOBE NEWSWIRE) -- SOPHiA GENETICS SA (Nasdaq: SOPH) today announced it will report financial results for the third quarter of 2021 before market open on Wednesday, November 10, 2021. The company’s management will webcast a corresponding conference call beginning at 8:30 a.m. Eastern Time / 2:30 p.m. Central European Time to discuss its results, business developments, and outlook.

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SOPHiA GENETICS to Report Third Quarter 2021 Financial Results on November 10, 2021

Global News Feed Comments Off on SOPHiA GENETICS to Report Third Quarter 2021 Financial Results on November 10, 2021 | October 28th, 2021

CPI-818 clinical trial in China will be conducted by Angel Pharmaceuticals CPI-818 clinical trial in China will be conducted by Angel Pharmaceuticals

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Corvus Pharmaceuticals Announces Partner Angel Pharmaceuticals Received IND Approval for Phase 1/1b Clinical Trial of ITK Inhibitor CPI-818 in China

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DURHAM, N.C., Oct. 27, 2021 (GLOBE NEWSWIRE) -- Bioventus Inc. (Nasdaq: BVS) (“Bioventus” or the “Company”), a global leader in innovations for active healing, today announced that third quarter of fiscal year 2021 financial results will be released before the market opens on Tuesday, November 9, 2021.

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Bioventus to Release Third Quarter of Fiscal Year 2021 Financial Results on November 9, 2021

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Alan Fuhrman, experienced biotech executive and Checkmate board member, appointed as interim President and CEO Alan Fuhrman, experienced biotech executive and Checkmate board member, appointed as interim President and CEO

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Checkmate Pharmaceuticals Announces CEO Transition

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JUPITER, Fla., Oct. 27, 2021 (GLOBE NEWSWIRE) -- Dyadic International, Inc. (“Dyadic”, “we”, “us”, “our”, or the “Company”) (NASDAQ: DYAI), a global biotechnology company focused on further improving, applying and deploying its proprietary C1-cell protein production platform to accelerate development, lower production costs and improve the performance of biologic vaccines and drugs at flexible commercial scales, today announced that it will report its financial results for the third quarter ended September 30, 2021 and host a corporate update conference call on Wednesday, November 10, 2021.

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Dyadic to Report Third Quarter 2021 Financial Results on Wednesday, November 10, 2021

Global News Feed Comments Off on Dyadic to Report Third Quarter 2021 Financial Results on Wednesday, November 10, 2021 | October 28th, 2021

SALT LAKE CITY, Oct. 27, 2021 (GLOBE NEWSWIRE) -- Myriad Genetics, Inc. (NASDAQ: MYGN), a leader in genetic testing and precision medicine, today announced that it will hold its quarterly earnings conference call for the quarter ended September 30, 2021, at 4:30 p.m. EDT on Tuesday, November 2, 2021. The company’s quarterly earnings will be released the same day prior to the market opening. During the call, Paul J. Diaz, president and CEO, and R. Bryan Riggsbee, chief financial officer, will provide a financial overview and business update of Myriad Genetics’ performance for the third quarter of 2021.

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Myriad Genetics to Release Third Quarter Financial Results on November 2, 2021

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- All Stockholder Proposals Were Approved -

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Greenrose Acquisition Corp. Stockholders Approve Business Combination

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TORONTO, Oct. 27, 2021 (GLOBE NEWSWIRE) -- PharmaTher Holdings Ltd. (the “Company” or “PharmaTher”) (OTCQB: PHRRF) (CSE: PHRM), a clinical-stage psychedelics biotech company, today released its business update and financial results for the three months ended August 31, 2021.  All amounts are stated in Canadian dollars unless otherwise indicated.

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PharmaTher Provides Business Update and Releases Financial Results for the First Quarter of Fiscal 2022

Global News Feed Comments Off on PharmaTher Provides Business Update and Releases Financial Results for the First Quarter of Fiscal 2022 | October 28th, 2021

ROCKVILLE, Md., Oct. 27, 2021 (GLOBE NEWSWIRE) -- Supernus Pharmaceuticals, Inc. (Nasdaq: SUPN), a biopharmaceutical company focused on developing and commercializing products for the treatment of central nervous system (CNS) diseases, today announced that the Company expects to report financial and business results for the third quarter of 2021 after the market closes on Wednesday, November 3, 2021.

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Supernus to Announce Third Quarter 2021 Financial Results and Host Conference Call on November 3, 2021

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HONG KONG and SHANGHAI, China and FLORHAM PARK, N.J., Oct. 28, 2021 (GLOBE NEWSWIRE) -- HUTCHMED (China) Limited (“HUTCHMED”) (Nasdaq/AIM:HCM; HKEX:13) today announces that it has initiated a Phase III trial in China of HMPL-523, a novel, investigational spleen tyrosine kinase (“Syk”) inhibitor, in adult patients with primary immune thrombocytopenia (“ITP”), an autoimmune disorder that can lead to increased risk of bleeding. The first patient received their first dose on October 27, 2021.

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HUTCHMED Initiates ESLIM-01, a Phase III Trial of HMPL-523 in Patients with Immune Thrombocytopenia in China

Global News Feed Comments Off on HUTCHMED Initiates ESLIM-01, a Phase III Trial of HMPL-523 in Patients with Immune Thrombocytopenia in China | October 28th, 2021