– Treatment with Mitapivat Induced Hemoglobin Increase of ?1.0 g/dL in 6 of 11 (55%) Efficacy Evaluable Patients, Decreased Markers of Hemolysis, Reduced 2,3-DPG and Increased ATP –

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Agios Announces Updated Data from Phase 1 Study of Mitapivat, First-in-Class PKR Activator, in Sickle Cell Disease

BASKING RIDGE, N.J., Dec. 07, 2020 (GLOBE NEWSWIRE) -- electroCore, Inc. (Nasdaq: ECOR), a commercial-stage bioelectronic medicine company, announced today that management is scheduled to present at the 13th Annual LD Micro Main Event Conference.

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electroCore to Present at the 13th Annual LD Micro Main Event Conference

Unique Product Development Model Delivers Accelerated Growth

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Syneos Health to Outline Value Creation Plan at 2020 Analyst and Investor Event

Initial data demonstrate early signs of safety, tolerability and robust immune responses in acute myeloid leukemia (AML) patients with relapsed disease after allogeneic hematopoietic cell transplantation (allo-HSCT) Initial data demonstrate early signs of safety, tolerability and robust immune responses in acute myeloid leukemia (AML) patients with relapsed disease after allogeneic hematopoietic cell transplantation (allo-HSCT)

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Preliminary Results from NexImmune’s Phase 1/2 Trial of NEXI-001 in AML Presented at 62nd ASH Annual Meeting and Exposition

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Roche presents new data from its bispecific antibody portfolio across a range of blood cancers

CAMBRIDGE, Mass. and ROSTOCK, Germany, and BERLIN and MUNICH, Germany, Dec. 08, 2020 (GLOBE NEWSWIRE) -- Centogene N.V. (Nasdaq: CNTG), a commercial-stage company focused on rare diseases that transforms real-world clinical and genetic data into actionable information for patients, physicians and pharmaceutical companies, and Alnylam Pharmaceuticals, Inc. (“Alnylam”), the leading RNAi therapeutics company, announced today the launch of a new joint clinical screening program: the genetic screening of the at-risk population for hereditary TransthyRetin-related AMyloidosis and longitudinal monitoring of TTR positive subjects (the “TRAMmoniTTR Study”).

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CENTOGENE and Alnylam Pharmaceuticals Launch a New Clinical Program Aimed at Revolutionizing the Diagnosis of Hereditary Transthyretin-Related...

Editor's note: SpaceX has successfully launched the Dragon CRS-21 cargo mission for NASA and landed its Falcon 9 rocket. Read our launch wrap story here.

CAPE CANAVERAL, Fla. The next SpaceX resupply launch to the International Space Station, scheduled for Sunday (Dec. 6), will carry a host of science gear to the astronauts living and working on the orbiting laboratory.

The robotic flight, called CRS-21, marks the 21st mission for SpaceX under its commercial cargo resupply services contract with NASA. Launch is scheduled for 11:17 a.m. EST (1617 GMT) on Sunday from NASA's Kennedy Space Center in Florida, and you can watch the action live here at Space.com, courtesy of NASA. You can also watch directly via NASA TV or SpaceX.

SpaceX initially aimed to launch the CRS-21 cargo mission for NASA on Saturday (Dec. 5), but foul weather prompted a delay. "Due to poor weather in the recovery area for todays attempt, now targeting Sunday, December 6 at 11:17 a.m. EST for launch of CRS-21," SpaceX wrote in an update early Saturday morning. SpaceX plans to recover the mission's Falcon 9 booster for later reuse.

The upgraded Dragon cargo capsule that will launch atop a veteran SpaceX Falcon 9 rocket is filled with 6,400 lbs. (2,903 kilograms) of supplies and science investigations. The research gear will support a variety of experiments in the life sciences, regenerative medicine and many other fields.

Related: How SpaceX's Dragon space capsule works (infographic)

Saturday's flight will mark the first time SpaceXs upgraded Dragon spacecraft will carry cargo. (Up until now, the advanced Dragon variant has solely carried astronauts.) The vehicle is a modified version of the Crew Dragon spacecraft that lacks the systems necessary for human missions, such as seats, cockpit controls and a life-support system, as well as the SuperDraco thrusters that provide a special emergency escape system that's only used if a problem occurs during launch.

This new Dragon allows more science to ride skyward. Costello explained that the interior of Dragon can now support more powered payloads, which is a huge benefit for the life sciences as it allows for more cold storage and other types of investigations. It also allows for the crew to store some of the powered payloads onboard Dragon while the craft is on orbit.

Several of the payloads on Dragon feature a unique piece of hardware called a tissue chip. Human cells and tissue grow on the chip scaffold, creating a 3D structure in microgravity that researchers can observe to learn more about how fundamental processes work in space, including aging and bone and muscle loss.

One such investigation, run by the University of Florida, will study how muscles atrophy in space. Sixteen samples of skeletal muscle will be sent to the space station, where the bundles of muscle tissue will be observed in microgravity. Half of the muscle samples were donated by younger, active individuals while the other half are from older, more sedentary volunteers.

Half of the samples in each group will be subjected to electric stimuli to see how the muscles contract in the absence of gravity. Researchers will use this experiment as a starting point for future research that will eventually test therapies to see if muscle degradation can be prevented.

Another payload will look at brain organoids created using stem cell technology. This investigation seeks to understand how microgravity affects the survival and function of brain cells, which could lead to advances in treatments for autism and Alzheimers disease, researchers said.

"Space travel mimics the effects of aging we see on Earth, only in a much shorter time span, making it easier to examine the processes that are taking place," Bill McLamb, chief scientist at Kentucky-based company Space Tango, told Space.com. "Its hard to study human brains in space, which is why these types of experiments are so beneficial."

The investigation will take stem cells and convert them into brain cells that will form three-dimensional structures called brain organoids. Stored in a special container called a well, these types of mini organs are able to mimic both the cellular variety and the function of the developing human brain.

This type of research could help NASA and its partners prepare for crewed missions to distant destinations such as Mars, which will expose astronauts to the rigors of space for long stretches, and also help combat degenerative brain disease here on Earth, researchers said.

A team of researchers from Stanford University will be looking at how engineered heart tissue behaves in microgravity. The Cardinal Heart investigation will send tissue samples that consist of cardiomyocytes, endothelial cells and cardiac fibroblasts to study how changes in gravity affect the heart at the cellular level.

Researchers know that microgravity causes changes in the workload and shape of the human heart, but it's still unknown if these changes could become permanent if a person lived for long periods of time in space.

The project's tissue bundles will be affixed to tissue chips. The experiment's results could help identify new treatments and support development of screening measures to predict cardiovascular risk prior to spaceflight, team members said. Follow-on investigations will include therapies that could treat heart disease.

The HemoCue investigation will look at how white blood cells react in space. Here on Earth, doctors use the total number of white blood cells, as well as the various types observed, to diagnose illness. HemoCue will debut a new type of technology that will allow users to do white blood cell counts on orbit.

The goal is to test how well the device works in microgravity. If effective, it could be a valuable tool in an astronauts medical kit, researchers said.

Another payload called Micro-14 looks at how yeast, in particular Candida albicans, responds to the space environment. C. albicans is an opportunistic pathogen, capable of causing severe and even life-threatening illness in immunocompromised hosts. Micro-14 will evaluate how the yeast responds to microgravity, looking for changes at the cellular and molecular levels.

Since astronauts can become immunocompromised during spaceflight, researchers are especially interested in how best to predict the health risks from this organism. Previous research has shown that many microbes exhibit increased virulence in a microgravity environment, but more research is needed on this particular pathogen.

NASAs Jet Propulsion Laboratory in Southern California is spearheading a project that will take swab samples from various locations within the station to look at the relationship between bacteria and their metabolites (chemicals produced by bacterial growth). The project will help researchers better understand the distribution of microbes and metabolites within closed environments and how this distribution affects human health. The research could aid administrators of hospitals and nursing homes, where residents are often immunocompromised.

Related: SpaceX rocket launches for record 7th time, nails landing at sea

Sunday's launch marks the 101st flight overall for SpaceXs workhorse two-stage Falcon 9 rocket. The liftoff is expected to feature a veteran Falcon 9 first stage, designated B1058, that already has three flights under its belt. This frequent flyer previously launched SpaceX's Demo-2 mission, which sent two NASA astronauts to the space station this past summer, well as a communications satellite for the South Korean military and a batch of the companys own Starlink satellites.

Flying previously flown boosters has become commonplace for SpaceX, as the company continues to prove the Falcon 9's reliability. In fact, CRS-21 marks the 24th flight of 2020 for SpaceX, with the majority of those missions having flown on veteran rockets rather than brand-new ones.

To date, SpaceX has successfully landed its first-stage boosters 67 times. Now that the company has two fully operational drone-ship landing platforms "Of Course I Still Love You" and "Just Read the Instructions" in Florida, its able to launch (and land) more rockets. "Of Course I Still Love You" is already at the recovery zone waiting for its turn to catch B1058 when it returns to Earth shortly after liftoff.

Weather was a concern for SpaceX going into the weekend. Forecasts predicted iffy weather for a Saturday launch attempt, with the 45th Weather Squadron predicting a 50% chance of favorable conditions for liftoff. The primary concerns were thick clouds and cumulus clouds. The backup attempt on Sunday looks much better, with the forecast improving to 70% favorable on that day.

If all goes as planned, the Dragon will arrive at the station and dock at the Harmony modules space-facing port just over 24 hours after it blasts off.

Editor's note: This story was updated at 8:22 a.m. EST to include SpaceX's launch delay to Sunday, Dec. 6, due to bad weather.

Follow Amy Thompson on Twitter @astrogingersnap. Follow us on Twitter @Spacedotcom or Facebook.

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Tissue chips and organoids: SpaceX is launching lots of science to space for NASA on Sunday - Space.com

The Autologous Stem Cell Based Therapies Market was valued at US$ XX million in 2019 and is projected to reach US$ XX million by 2025, at a CAGR of XX percentage during the forecast period. In this study, 2019 has been considered as the base and 2020 to 2025 as the forecast period to estimate the market size for Autologous Stem Cell Based Therapies Market

Deep analysis about market status (2016-2019), competition pattern, advantages and disadvantages of products, industry development trends (2019-2025), regional industrial layout characteristics and macroeconomic policies, industrial policy has also been included. From raw materials to downstream buyers of this industry have been analysed scientifically. This report will help you to establish comprehensive overview of the Autologous Stem Cell Based Therapies Market

Get a Sample Copy of the Report at: https://i2iresearch.com/report/global-autologous-stem-cell-based-therapies-market-2020-market-size-share-growth-trends-forecast-2025/

The Autologous Stem Cell Based Therapies Market is analysed based on product types, major applications and key players

Key product type:Embryonic Stem CellResident Cardiac Stem CellsUmbilical Cord Blood Stem Cells

Key applications:Neurodegenerative DisordersAutoimmune DiseasesCardiovascular Diseases

Key players or companies covered are:RegeneusMesoblastPluristem Therapeutics IncU.S. STEM CELL, INC.Brainstorm Cell TherapeuticsTigenixMed cell Europe

The report provides analysis & data at a regional level (North America, Europe, Asia Pacific, Middle East & Africa , Rest of the world) & Country level (13 key countries The U.S, Canada, Germany, France, UK, Italy, China, Japan, India, Middle East, Africa, South America)

Inquire or share your questions, if any: https://i2iresearch.com/report/global-autologous-stem-cell-based-therapies-market-2020-market-size-share-growth-trends-forecast-2025/

Key questions answered in the report:1. What is the current size of the Autologous Stem Cell Based Therapies Market, at a global, regional & country level?2. How is the market segmented, who are the key end user segments?3. What are the key drivers, challenges & trends that is likely to impact businesses in the Autologous Stem Cell Based Therapies Market?4. What is the likely market forecast & how will be Autologous Stem Cell Based Therapies Market impacted?5. What is the competitive landscape, who are the key players?6. What are some of the recent M&A, PE / VC deals that have happened in the Autologous Stem Cell Based Therapies Market?

The report also analysis the impact of COVID 19 based on a scenario-based modelling. This provides a clear view of how has COVID impacted the growth cycle & when is the likely recovery of the industry is expected to pre-covid levels.

Contact us:i2iResearch info to intelligenceLocational Office: *India, *United State, *GermanyEmail: [emailprotected]Toll-free: +1-800-419-8865 | Phone: +91 98801 53667

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Autologous Stem Cell Based Therapies Market Research Report 2020: Market Competition Trend and Price by Manufacturers till 2026 - Factory Maintenance

ESA astronaut Tim Peake during his 4 hour 43 minute spacewalk to replace a failed power regulator and install cabling on the ISS. Credit: ESA/NASA

Tim Peake is the first official British astronaut to walk in space. The former Army Air Corps officer has spent six months in space, after blasting off on a Russian Soyuz rocket to the International Space Station on December 15, 2016, but the spacewalk doubtless was his most gruelling test.

But what exactly did he go through, during his remarkable spell aboard the space station? Space travel leads to many changes in the human body, many of which have been investigated since Yuri Gargarin made the first manned spaceflight in 1961 and an extensive team provides guidance and preparation for astronauts before, during and after any spaceflight. But if youre planning an out-of-this-world trip, here are some of the things to expect.

The skeletal muscle system is the largest organ system of the human body. Hundreds of muscles are used for maintaining posture sitting, standing and performing a wide range of movements, with different loading conditions imposed by the forces of gravity on Earth.

Skeletal muscles have the ability to adapt to different purposes and the different loads placed on them, a quality known as plasticity. But like inactivity, space flight leads to loss of both skeletal muscle mass (atrophy) and strength.

During long spaceflights on the ISS, research found that 37 crew members experienced a decrease in mean isokinetic strength of between 8% and 17%. Men and women were similarly affected. In fact, this degradation occurs even when astronauts follow a strict exercise regime, meaning that it has profound implications for humans embarking on even longer journeys, such as to Mars. Data suggests that around 30% of muscle strength is lost after spending 110 to 237 days in microgravity.

Many parts of the cardiovascular system (including the heart) are influenced by gravity. On Earth, for example, the veins in our legs work against gravity to get blood back to the heart. Without gravity, however, the heart and blood vessels change and the longer the flight, the more severe the changes.

The size and shape of the heart, for example, changes with microgravity and the right and left ventricles decrease in mass. This may be because of a decrease in fluid volume (blood) and changes in myocardial mass. A human heart rate (number of beats per minute) is lower in space than on Earth, too. In fact, it has been found that the heart rate of individuals standing upright on the ISS is similar to their rate while lying down pre-flight on Earth. Blood pressure is also lower in space than on Earth.

The cardiac output of the heart the amount of blood pumped out of the heart each minute decreases in space, too. Without gravity, there is also a redistribution of the blood more blood stays in the legs and less blood is returned to the heart, which leads to less blood being pumped out of the heart. Muscle atrophy also contributes to reduced blood flow to the lower limbs.

This reduced blood flow to the muscles, combined with the loss of muscle mass, impacts aerobic capacity (below).

Aerobic capacity is a measure of aerobic fitness the maximum amount of oxygen that the body can use during exercise. This can be measured by VO2max and VO2peak tests. Changes to both the muscles and cardiovascular system caused by spaceflight contribute to reduced aerobic fitness.

After nine to 14 days of spaceflight, for example, research shows that aerobic capacity (VO2peak) is reduced by 20%-25%. But the trends are interesting. During longer spells in space say, five to six months after the initial reduction in aerobic capacity, the body appears to compensate and the numbers begin improving although they never return to pre-trip levels.

On Earth, the effects of gravity and mechanical loading are needed to maintain our bones. In space, this doesnt happen. Bone normally undergoes continual remodelling and two types of cells are involved: osteoblasts (these make and regulate the bone matrix) and osteoclasts (these absorb bone matrix). During spaceflight, however, the balance of these two processes is altered which leads to reduced bone mineral density. Research shows that a 3.5% loss of bone occurs after 16 to 28 weeks of spaceflight, 97% of which is in weight-bearing bones, such as the pelvis and legs.

The immune system, which protects the body against disease, is also affected. There are a number of variables which contribute to this, including radiation, microgravity, stress, isolation and alterations in the circadian rhythm, the 24-hour cycle of sleep and wakefulness that we follow on Earth. Also, while in space, astronauts will interact with microbes from themselves, other crew members, their food, their environment and these can alter their immune response, which may lead to challenging situations and increase the potential for infections among the crew as well as contamination of extraterrestrial sites.

This article is republished from The Conversation under a Creative Commons license.

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Five things that happen to your body in space - RocketSTEM

Data support novel approach to develop and manufacture a best-in-class, durable medicine for people living with hemoglobinopathies

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Editas Medicine Announces Preclinical Data and Large-Scale Manufacturing Process for EDIT-301, in Development for the Treatment of Sickle Cell Disease...

CAMBRIDGE, Mass., Dec. 05, 2020 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), is presenting new preclinical data in support of NTLA-5001, the company’s wholly owned Wilms’ Tumor 1 (WT1)-directed T cell receptor (TCR)-T cell therapy candidate for the treatment of acute myeloid leukemia (AML), at the 62nd American Society of Hematology (ASH) Annual Meeting, taking place virtually from December 5-8, 2020. NTLA-5001 capitalizes on how natural T cells recognize and respond to tumors. The target, WT1, is highly overexpressed in AML, a cancer of the blood and bone marrow that is often fatal despite existing treatments (NIH SEER Cancer Stat Facts: Leukemia – AML). The new preclinical data being presented today highlight the faster expansion and superior function of T cells manufactured by Intellia’s proprietary approach, compared to a standard genome editing process. Specifically, NTLA-5001’s lead TCR-T cells resulted in significantly higher anti-tumor activity in mouse models of acute leukemias than that observed in mice treated with cells engineered using the standard process.

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Intellia Therapeutics Presents New Preclinical Data Supporting Its CRISPR/Cas9-Engineered TCR-T Cell Treatment for Acute Myeloid Leukemia at the...

SOUTH SAN FRANCISCO, Calif., Dec. 05, 2020 (GLOBE NEWSWIRE) -- Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T™) therapies for cancer, today announced positive initial results from the Phase 1 UNIVERSAL study of ALLO-715 in relapsed/refractory multiple myeloma (MM). Data were presented at an oral session of the American Society of Hematology (ASH) annual meeting. This study utilizes ALLO-647, Allogene's anti-CD52 monoclonal antibody (mAb), as a part of its differentiated lymphodepletion regimen.

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Allogene Therapeutics Reports Positive Initial Results from Phase 1 UNIVERSAL Study of ALLO-715 AlloCAR T™ Cell Therapy in Relapsed/Refractory...

- Beta thalassemia: All seven patients were transfusion independent with 3 to 18 months of follow-up after CTX001 infusion -

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CRISPR Therapeutics and Vertex Present New Data for Investigational CRISPR/Cas9 Gene-Editing Therapy, CTX001™ at American Society of Hematology...

– Evidence of biologic activity observed in each dose-escalation cohort treated to date –

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Kura Oncology Presents First Clinical Data for Menin Inhibitor KO-539 at American Society of Hematology Annual Meeting

Basel, 5 December 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that new data from the pivotal phase III MURANO and CLL14 studies support the efficacy of fixed-duration, chemotherapy-free Venclexta®/Venclyxto® (venetoclax)-based combinations in certain people with chronic lymphocytic leukaemia (CLL) and provide more evidence on the potential value of minimal residual disease (MRD). Data were presented at the all-virtual 62nd American Society of Hematology (ASH) Annual Meeting and Exposition on Saturday 5 December 2020. “These results reinforce the long-term value of fixed-duration, chemotherapy-free Venclexta/Venclyxto-based combinations in CLL, potentially offering patients a significant period of time without treatment following initial therapy,” said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. “These data also reflect our ongoing commitment to accelerating clinical advancements for patients by exploring the novel endpoint minimal residual disease as a potential predictor of patient outcomes.” Five-year data from the pivotal phase III MURANO trial continue to show sustained investigator-assessed progression-free survival (PFS) with Venclexta/Venclyxto plus MabThera®/Rituxan® (rituximab). Data, presented in an oral session, showed:

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Roche announces new data reinforcing the long-term benefit of Venclexta/Venclyxto-based combination for people with relapsed or refractory chronic...

- 9 of 14 Patients Showed Reduction in Tumor Size, Including Two Recently Reported Complete Responses -

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IGM Biosciences Presents First Clinical Data from IGM-2323 in Non-Hodgkin’s Lymphoma at 2020 ASH Annual Meeting

Updated data from the ALLCAR study suggests AUTO1’s potential for transformational activity in adult patients with r/r ALL

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Autolus Therapeutics presents compelling AUTO1 data from ALLCAR Phase 1 study in Adult Acute Lymphoblastic Leukemia (ALL) during the 62nd ASH Annual...

NEW YORK, Dec. 05, 2020 (GLOBE NEWSWIRE) -- Cellectis (Euronext Growth: ALCLS - Nasdaq: CLLS), a clinical-stage biopharmaceutical company focused on developing immunotherapies based on gene-edited allogeneic CAR T-cells (UCART), announced preliminary results from Cellectis’ dose escalation Phase 1 BALLI-01 study of UCART22 product candidate in relapsed/refractory B-cell Acute Lymphoblastic Leukemia (B-ALL) were presented at the American Society of Hematology (ASH) Annual Meeting. This is the first publicly released data from Cellectis’ BALLI-01 clinical trial.

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Cellectis Reports Preliminary Results from its Phase 1 BALLI-01 Study of UCART22 in R/R Adult B-ALL at American Society of Hematology (ASH) Annual...

~Five presentations related to Kiadis’ K-NK cell therapy platform will be presented

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Kiadis announces new data at the 2020 ASH Annual Meeting and Exposition

CAMBRIDGE, Mass., Dec. 06, 2020 (GLOBE NEWSWIRE) -- Constellation Pharmaceuticals, Inc. (Nasdaq: CNST) today announced that two oral presentations and three posters relating to the Phase 2 MANIFEST and the Phase 3 MANIFEST-2 clinical trials of CPI-0610 in myelofibrosis (MF) were presented at the American Society of Hematology (ASH) Annual Meeting and Exposition. The preliminary data in these presentations are based on a data cutoff of September 29, 2020, and reflect an analysis of clinical activity in 63 first-line (1L) and 94 second-line (2L) or later patients.

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Constellation Pharmaceuticals Provides Update of MANIFEST Study for CPI-0610 at ASH Meeting