DetailsCategory: DNA RNA and CellsPublished on Wednesday, 28 October 2020 18:51Hits: 331

NORWALK, CT & SAN FRANCISCO, CA, USA I October 28, 2020 I The Multiple Myeloma Research Foundation (MMRF) and Indapta Therapeutics announced a partnership to support the advancement of the companys universal G-NK cell therapy into clinical trials for the treatment of patients with multiple myeloma. Indapta Therapeutics, Inc., is a biotechnology company focused on developing and commercializing a proprietary, off-the-shelf, allogeneic FcR-deficient natural killer (G-NK) cell therapy for the treatment of multiple cancers. The collaboration is supported with an investment in Indapta by the MMRFs venture philanthropy arm, the Myeloma Investment Fund (MIF).

Our investment in Indaptas G-NK cell therapy is consistent with our strategy to fund the development of first-in-class, potentially transformative treatments, said Paul Giusti, President and Chief Executive Officer of the MMRF. Indaptas off-the-shelf cell therapy uses a promising new class of NK cells, which could provide a significant benefit for patients.

About Indaptas G-NK Cell Therapy

Indapta Therapeutics is developing a universal, allogeneic G-NK cell therapy designed to substantially improve the cytotoxicity of monoclonal antibody therapy in multiple cancers. G-NK cells are a specific and potent subset of NK (natural killer) cells with specialized anti-tumor activity resulting from an epigenetic change, rather than engineering. Indapta has further enhanced G-NK cells via specific G-NK cell subset selection and its proprietary manufacturing process which, when combined, produce a G-NK cell therapy that demonstrates higher efficacy, persistence, and enhanced cryopreservation than multiple mAb therapies alone or mAb therapies combined with conventional NK cells.

When a monoclonal antibody binds to the tumor target and to Indaptas G-NK cell therapy product, it initiates the release of dramatically more cancer killing compounds than conventional NK cells, allowing for increased efficacy and potentially less frequent dosing. Indaptas off-the-shelf G-NK cell therapy is further differentiated from other NK cell therapies in that it is a cell banked product with low variability. In vivo studies have demonstrated the safety of Indaptas G-NK cell therapy.

This collaboration with the MMRF and MIF will be invaluable in helping us advance the clinical development of our universal, allogeneic G-NK cell therapy, said Guy DiPierro, Founder, and Chief Executive Officer of Indapta Therapeutics. We look forward to tapping into MMRFs deep myeloma expertise and other critical resources, including genomic datasets. The Foundations insights into patient recruitment and study networks will help us reach multiple myeloma patients for our own clinical trials.

About Indapta Therapeutics

Indapta Therapeutics, Inc. is a biotechnology company focused on developing and commercializing a proprietary, first-in-class, off-the-shelf allogeneic cell therapy to treat multiple types of difficult-to-treat hematologic cancers and solid tumors. Headquartered in San Francisco, Indapta was founded in 2017 by Guy DiPierro along with Ronald Martell and scientists at the University of California, Davis, and Stanford University. The company has developed allogeneic FcRI-deficient natural killer cells, known as G-NK cells, and is working to bring this off-the-shelf cell therapy to patients to address the limitations of currently available autologous T-cell therapies.

About The Multiple Myeloma Research Foundation

A pioneer in precision medicine, the Multiple Myeloma Research Foundation (MMRF) seeks to find a cure for multiple myeloma by relentlessly pursuing innovation that accelerates the development of next-generation treatments to extend the lives of patients. Founded in 1998 by Kathy Giusti, a multiple myeloma patient, and her twin sister Karen Andrews, as a 501(c)(3) nonprofit organization, the MMRF is a world-recognized leader in cancer research, launching over 70 clinical trials and leading to 13 FDA-approved drugs. Together with its partners, the MMRF has created the only end-to-end solution in precision medicine. With this, the MMRF, an innovator in precision medicine, continues to disrupt the industry today, building the CoMMpass Study, the single largest genomic dataset for any cancer, and launching MyDRUG, the first platform trial in multiple myeloma. Since its inception, the organization has collected over 4,000 samples and tissues in its database, which is shared among 23 academic institutions. The MMRF has raised over $400 million and directs nearly 90% of the total funds to research and related programs. To learn more, visit http://www.themmrf.org.

About Myeloma Investment Fund

The Myeloma Investment Fund (MIF), a wholly-owned subsidiary of the Multiple Myeloma Research Foundation (MMRF), is a venture philanthropy fund that invests in promising companies, clinical assets, and technologies in oncology to accelerate the delivery of precision medicine approaches and a cure to every multiple myeloma patient. The MIF collaborates closely with portfolio companies to help them advance multiple myeloma research, providing access to longstanding relationships with pharmaceutical companies and academic medical centers, a clinical network of 23 sites, and data from the largest genomics data set of any cancer. This evergreen fund is supported entirely by philanthropy; all profits will be reinvested back into research for more effective treatments until there is a cure for every patient. For more information, visit MyelomaInvestmentFund.org.

SOURCE: Indapta Therapeutics

More here:
Multiple Myeloma Research Foundation (MMRF) and Indapta Therapeutics Announce Investment to Advance Clinical Development of Natural Killer Cell...

Regenerative medicine is a part of translational research in the fields of molecular biology and tissue engineering. This type of medicine involves replacing and regenerating human cells, organs, and tissues with the help of specific processes. Doing this may involve a partial or complete reengineering of human cells so that they start to function normally.

Get Exclusive PDF Sample Copy Of This Report:https://www.tmrresearch.com/sample/sample?flag=B&rep_id=1889

Regenerative medicine also involves the attempts to grow tissues and organs in a laboratory environment, wherein they can be put in a body that cannot heal a particular part. Such implants are mainly preferred to be derived from the patients own tissues and cells, particularly stem cells. Looking at the promising nature of stem cells to heal and regenerative various parts of the body, this field is certainly expected to see a bright future. Doing this can help avoid opting for organ donation, thus saving costs. Some healthcare centers might showcase a shortage of organ donations, and this is where tissues regenerated using patients own cells are highly helpful.

There are several source materials from which regeneration can be facilitated. Extracellular matrix materials are commonly used source substances all over the globe. They are mainly used for reconstructive surgery, chronic wound healing, and orthopedic surgeries. In recent times, these materials have also been used in heart surgeries, specifically aimed at repairing damaged portions.

Cells derived from the umbilical cord also have the potential to be used as source material for bringing about regeneration in a patient. A vast research has also been conducted in this context. Treatment of diabetes, organ failure, and other chronic diseases is highly possible by using cord blood cells. Apart from these cells, Whartons jelly and cord lining have also been shortlisted as possible sources for mesenchymal stem cells. Extensive research has conducted to study how these cells can be used to treat lung diseases, lung injury, leukemia, liver diseases, diabetes, and immunity-based disorders, among others.

Buy This Report @https://www.tmrresearch.com/checkout?rep_id=1889<ype=S

Global Regenerative Medicine Market: Overview

The global market for regenerative medicine market is expected to grow at a significant pace throughout the forecast period. The rising preference of patients for personalized medicines and the advancements in technology are estimated to accelerate the growth of the global regenerative medicine market in the next few years. As a result, this market is likely to witness a healthy growth and attract a large number of players in the next few years. The development of novel regenerative medicine is estimated to benefit the key players and supplement the markets growth in the near future.

Global Regenerative Medicine Market: Key Trends

The rising prevalence of chronic diseases and the rising focus on cell therapy products are the key factors that are estimated to fuel the growth of the global regenerative medicine market in the next few years. In addition, the increasing funding by government bodies and development of new and innovative products are anticipated to supplement the growth of the overall market in the next few years.

On the flip side, the ethical challenges in the stem cell research are likely to restrict the growth of the global regenerative medicine market throughout the forecast period. In addition, the stringent regulatory rules and regulations are predicted to impact the approvals of new products, thus hampering the growth of the overall market in the near future.

Global Regenerative Medicine Market: Market Potential

The growing demand for organ transplantation across the globe is anticipated to boost the demand for regenerative medicines in the next few years. In addition, the rapid growth in the geriatric population and the significant rise in the global healthcare expenditure is predicted to encourage the growth of the market. The presence of a strong pipeline is likely to contribute towards the markets growth in the near future.

Global Regenerative Medicine Market: Regional Outlook

In the past few years, North America led the global regenerative medicine market and is likely to remain in the topmost position throughout the forecast period. This region is expected to account for a massive share of the global market, owing to the rising prevalence of cancer, cardiac diseases, and autoimmunity. In addition, the rising demand for regenerative medicines from the U.S. and the rising government funding are some of the other key aspects that are likely to fuel the growth of the North America market in the near future.

Furthermore, Asia Pacific is expected to register a substantial growth rate in the next few years. The high growth of this region can be attributed to the availability of funding for research and the development of research centers. In addition, the increasing contribution from India, China, and Japan is likely to supplement the growth of the market in the near future.

Global Regenerative Medicine Market: Competitive Analysis

The global market for regenerative medicines is extremely fragmented and competitive in nature, thanks to the presence of a large number of players operating in it. In order to gain a competitive edge in the global market, the key players in the market are focusing on technological developments and research and development activities. In addition, the rising number of mergers and acquisitions and collaborations is likely to benefit the prominent players in the market and encourage the overall growth in the next few years.

Some of the key players operating in the regenerative medicine market across the globe are Vericel Corporation, Japan Tissue Engineering Co., Ltd., Stryker Corporation, Acelity L.P. Inc. (KCI Licensing), Organogenesis Inc., Medtronic PLC, Cook Biotech Incorporated, Osiris Therapeutics, Inc., Integra Lifesciences Corporation, and Nuvasive, Inc. A large number of players are anticipated to enter the global market throughout the forecast period.

To know more about the table of contents, you can click @https://www.tmrresearch.com/sample/sample?flag=T&rep_id=1889

About TMR Research

TMR Research is a premier provider of customized market research and consulting services to busi-ness entities keen on succeeding in todays supercharged economic climate. Armed with an experi-enced, dedicated, and dynamic team of analysts, we are redefining the way our clients conduct business by providing them with authoritative and trusted research studies in tune with the latest methodologies and market trends.

Originally posted here:
Regenerative Medicine Market Poised to Garner Maximum Revenues During 2025 - The Think Curiouser

Caron Jacobson, MD, discusses the significance of CD19-targeted CAR T-cell therapy in B-cell malignancies.

Caron Jacobson, MD, a senior physician and medical director of the Immune Effector Cell Therapy Program at Dana-Farber Cancer Institute, as well as an assistant professor of medicine at Harvard Medical School, discusses the significance of CD19-targeted CAR T-cell therapy in B-cell malignancies.

The introduction ofCD19-targeted CAR T-cell therapy has transformed the treatment of patients with CD19-positive cancers,says Jacobson, adding that the effects of CAR T-cell therapy have been felt across hematologic malignancies.

As these products continue in development, research efforts are seeking ways to apply this technology to other diseases that do not express CD19, Jacobson explains.

With a number of ongoing clinical trials underway, the field is hopeful that these products will receive new regulatoryindications sothat the benefits observed in B-cell malignancies can translate to other tumor types, Jacobson concludes.

Go here to read the rest:
Dr. Jacobson on the Significance of CD19-Targeted CAR T-Cell Therapy in B-Cell Malignancies - OncLive

Oct. 29, 2020 12:00 UTC

Rare disease and neurology expert Dr. Angela Genge to lead QurAlis clinical R&D for ALS and FTD

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- QurAlis Corporation, a biotech company focused on developing precision medicines for amyotrophic lateral sclerosis (ALS) and other neurologic diseases, today announced the appointment of Angela Genge, MD, FRCP(C), eMBA to the position of Chief Medical Officer (CMO). Dr. Genge is the Executive Director of the Montreal Neurological Institutes Clinical Research Unit and the Director of Montreal Neurological Hospitals ALS Global Center of Excellence.

The company also announced the formation of its Clinical Advisory Board, which will work closely with Dr. Genge on QurAlis clinical research and development programs in ALS and frontotemporal dementia (FTD) as the company prepares to move its pipeline to the clinical stage.

As QurAlis grows and advances quickly toward the clinic, we are proud to welcome to the team Dr. Genge, a world-renowned expert in ALS clinical drug development, and announce the highly esteemed group of ALS experts who will be forming our Clinical Advisory Board, said Kasper Roet, PhD, Chief Executive Officer of QurAlis. Dr. Genge has been treating patients and studying and developing therapeutics and clinical trials for ALS and other rare neurologic diseases for more than 25 years, diligently serving these vulnerable patient populations. Along with our newly formed Clinical Advisory Board, having a CMO with this extensive expertise, understanding and experience is invaluable to our success. Dr. Genge and our Board members are tremendous assets for our team who will undoubtedly help us advance on the best path toward the clinic, and we look forward to working with them to conquer ALS.

Previously, Dr. Genge directed other clinics at the Montreal Neurological Hospital including the Neuromuscular Disease Clinic and the Neuropathic Pain Clinic. In 2014, she was a Distinguished Clinical Investigator in Novartis Global Neuroscience Clinical Development Unit, and she has served as an independent consultant for dozens of companies developing and launching neurological therapeutics. Dr. Genge has served in professorial positions at McGill University since 1994.

At this pivotal period in its journey, QurAlis is equipped with a strong, committed leadership team and promising precision medicine preclinical assets, and I look forward to joining the company as CMO, said Dr. Genge. This is an exciting opportunity to further strengthen my work in ALS and other neurological diseases, and I intend to continue innovating and expanding possibilities for the treatment of rare neurological diseases alongside the dedicated QurAlis team.

QurAlis new Clinical Advisory Board Members are:

Dr. Al-Chalabi is a Professor of Neurology and Complex Disease Genetics at the Maurice Wohl Clinical Neuroscience Institute, Head of the Department of Basic and Clinical Neuroscience, and Director of the Kings Motor Neuron Disease Care and Research Centre. Dr. Al-Chalabi trained in medicine in Leicester and London, and subsequently became a consultant neurologist at Kings College Hospital.

Dr. Andrews is an Associate Professor of Neurology in the Division of Neuromuscular Medicine at Columbia University, and serves as the Universitys Director of Neuromuscular Clinical Trials. She currently oversees neuromuscular clinical trials and cares for patients with neuromuscular disease, primarily with ALS. Dr. Andrews is the elected co-chair of the Northeastern ALS (NEALS) Consortium and is also elected to the National Board of Trustees of the ALS Association.

Dr. Cudkowicz is the Julianne Dorn Professor of Neurology at Harvard Medical School and Chief of Neurology and Director of the Sean M. Healey & AMG Center for ALS at Mass General Hospital. As co-founder and former co-chair of the Northeast ALS Consortium, she accelerated the development of ALS treatments for people with ALS, leading pioneering trials using antisense oligonucleotides, new therapeutic treatments and adaptive trial designs. Through the Healey Center at Mass General, she is leading the first platform trial for people with ALS.

Dr. Shaw serves as Director of the Sheffield Institute for Translational Neuroscience, the NIHR Biomedical Research Centre Translational Neuroscience for Chronic Neurological Disorders, and the Sheffield Care and Research Centre for Motor Neuron Disorders. She also serves as Consultant Neurologist at the Sheffield Teaching Hospitals NHS Foundation Trust. Since 1991, she has led a major multidisciplinary program of research investigating genetic, molecular and neurochemical factors underlying neurodegenerative disorders of the human motor system.

Dr. Van Damme is a Professor of Neurology and director of the Neuromuscular Reference Center at the University Hospital Leuven in Belgium. He directs a multidisciplinary team for ALS care and clinical research that is actively involved in ALS clinical trials, but is also working on the genetics of ALS, biomarkers of ALS, and disease mechanisms using different disease models, including patient-derived induced pluripotent stem cells.

Dr. van den Berg is a professor of neurology who holds a chair in experimental neurology of motor neuron diseases at the University Medical Center Utrecht in the Netherlands. He also is director of the centers Laboratory for Neuromuscular Disease, director of the Netherlands ALS Center, chairman of the Neuromuscular Centre the Netherlands, and chairman of the European Network to Cure ALS (ENCALS), a network of the European ALS Centres.

About ALS

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrigs disease, is a progressive neurodegenerative disease impacting nerve cells in the brain and spinal cord. ALS breaks down nerve cells, reducing muscle function and causing loss of muscle control. ALS can be traced to mutations in over 25 different genes and is often caused by a combination of multiple sub-forms of the condition. Its average life expectancy is three years, and there is currently no cure for the disease.

About QurAlis Corporation

QurAlis is bringing hope to the ALS community by developing breakthrough precision medicines for this devastating disease. Our stem cell technologies generate proprietary human neuronal models that enable us to more effectively discover and develop innovative therapies for genetically validated targets. We are advancing three antisense and small molecule programs addressing sub-forms of the disease that account for the majority of patients. Together with a world-class network of thought leaders, drug developers and patient advocates, our team is rising to the challenge of conquering ALS. http://www.quralis.com

View source version on businesswire.com: https://www.businesswire.com/news/home/20201029005203/en/

The rest is here:
QurAlis Announces Appointment of New Chief Medical Officer and Formation of Clinical Advisory Board - BioSpace

Real-world effectiveness data on Oxbryta (voxelotor) in the treatment of sickle cell disease featured as oral presentation

SOUTH SAN FRANCISCO, Calif., Oct. 26, 2020 (GLOBE NEWSWIRE) -- Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT), today announced its participation in the 15th Annual Scientific Conference on Sickle Cell and Thalassemia (ASCAT)and 1stEHA European Sickle Cell Conference, taking place online on Oct. 26-31, 2020. Two abstracts have been accepted for presentation, including real-world effectiveness data of Oxbryta (voxelotor) tablets in the treatment of sickle cell disease (SCD).

GBT is focused on addressing sickle cell disease at its root cause to seek to modify the course of the disease, and, ultimately, mitigate the serious and life-threatening complications that often lead to long-term damage and early death, said Ted W. Love, M.D., president and CEO of GBT. Were pleased to be at ASCAT 2020, where we are sharing real-world effectiveness data demonstrating that the benefits of treatment of sickle cell patients with Oxbryta were consistent with the results of our Phase 3 HOPE Study.

The two abstracts presented at the conference provide greater insight into the safety and efficacy of Oxbryta:

Details of the ASCAT presentations are as follows:

Wednesday, Oct. 28Abstract Session: New TherapiesReal-World Effectiveness of Voxelotor for the Treatment of Sickle Cell Disease: A Chart Review StudyPresenter: Kenneth Bridges, M.D., GBTTime: 11 a.m. GMT

Virtual Presentationin Poster RoomSickle Cell Anemia and COVID-19: Use of Voxelotor to Avoid TransfusionPresenter: William B. Ershler, M.D., Inova Schar Cancer Institute

As part of its presence at ASCAT, GBT will host an educational online symposium, Getting to the Root Cause: Understanding the Devastating Impact of SCD on Patients, on Monday, Oct. 26, 7-8 p.m. GMT. The symposium will include presentations by Caterina Minniti, M.D., professor of clinical medicine and pediatrics at Einstein College of Medicine and director of the Sickle Cell Center for Adults at Montefiore Medical Center; and Bart Biemond, M.D., Ph.D., professor of internal medicine and hematologist at the Faculty of Medicine of the University of Amsterdam. More information about the symposium, which is available to registered attendees of the ASCAT meeting, can be found in the conference program.

Coinciding with ASCAT, today GBT released a comprehensive report, Overview of the Sickle Cell Disease Environment in Select European Countries, in order to help raise awareness of SCD in Europe. The report is based on research conducted in five European countries and explores the burden of the disease on patients, families and health care systems. In addition, the report examines existing public policy efforts to address and alleviate the challenges faced by those living with SCD. SCD is one of the most prevalent genetic rare diseases in Europe, and despite a clinical understanding of SCD, estimated life expectancy for those affected by the disease is significantly lower than the European average, indicating an urgent need to improve the quality of care for these patients.

About Sickle Cell DiseaseSickle cell disease (SCD) affects an estimated 100,000 people in the United States,1 an estimated 52,000 people in Europe2 and millions of people throughout the world, particularly among those whose ancestors are from sub-Saharan Africa.1 It also affects people of Hispanic, South Asian, Southern European and Middle Eastern ancestry.1 SCD is a lifelong inherited rare blood disorder that impacts hemoglobin, a protein carried by red blood cells that delivers oxygen to tissues and organs throughout the body.3 Due to a genetic mutation, individuals with SCD form abnormal hemoglobin known as sickle hemoglobin. Through a process called hemoglobin polymerization, red blood cells become sickled deoxygenated, crescent-shaped, and rigid.3-5 The sickling process causes hemolytic anemia (low hemoglobin due to red blood cell destruction) and blockages in capillaries and small blood vessels, which impede the flow of blood and oxygen throughout the body. The diminished oxygen delivery to tissues and organs can lead to life-threatening complications, including stroke and irreversible organ damage.4-7

About Oxbryta (voxelotor) TabletsOxbryta (voxelotor) is an oral, once-daily therapy for patients with sickle cell disease (SCD). Oxbryta works by increasing hemoglobins affinity for oxygen. Since oxygenated sickle hemoglobin does not polymerize, GBT believes Oxbryta blocks polymerization and the resultant sickling and destruction of red blood cells, which are primary pathologies faced by every single person living with SCD. With the potential to improve hemolytic anemia and oxygen delivery, GBT believes that Oxbryta has the potential to modify the course of SCD. On Nov. 25, 2019, Oxbryta received U.S. Food and Drug Administration (FDA) accelerated approval for the treatment of SCD in adults and children 12 years of age and older.8 As a condition of accelerated approval, GBT will continue to study Oxbryta in the HOPE-KIDS 2 Study, a post-approval confirmatory study using transcranial Doppler (TCD) flow velocity to assess the ability of the therapy to decrease stroke risk in children 2 to 15 years of age.

In recognition of the critical need for new SCD treatments, the FDA granted Oxbryta Breakthrough Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations for the treatment of patients with SCD. Additionally, Oxbryta has been granted Priority Medicines (PRIME) designation from the European Medicines Agency (EMA), and the European Commission (EC) has designated Oxbryta as an orphan medicinal product for the treatment of patients with SCD.

GBT plans to seek regulatory approvals to expand the potential use of Oxbryta in the United States for the treatment of SCD in children age 4 to 11 years and to treat hemolytic anemia in SCD in people age 12 years and older in Europe.

Important Safety InformationOxbryta should not be taken if the patient has had an allergic reaction to voxelotor or any of the ingredients in Oxbryta. See the end of the patient leaflet for a list of the ingredients in Oxbryta. Oxbryta can cause serious side effects, including serious allergic reactions. Patients should tell their health care provider or get emergency medical help right away if they get rash, hives, shortness of breath or swelling of the face.

Patients receiving exchange transfusions should talk to their health care provider about possible difficulties with the interpretation of certain blood tests when taking Oxbryta.

The most common side effects of Oxbryta include headache, diarrhea, stomach (abdominal) pain, nausea, tiredness, rash and fever. These are not all the possible side effects of Oxbryta. Before taking Oxbryta, patients should tell their health care provider about all medical conditions, including if they have liver problems; if they are pregnant or plan to become pregnant as it is not known if Oxbryta can harm an unborn baby; or if they are breastfeeding or plan to breastfeed as it is not known if Oxbryta can pass into breastmilk or if it can harm a baby. Patients should not breastfeed during treatment with Oxbryta and for at least two weeks after the last dose.

Patients should tell their health care provider about all the medicines they take, including prescription and over-the-counter medicines, vitamins and herbal supplements. Some medicines may affect how Oxbryta works. Oxbryta may also affect how other medicines work.

Patients are advised to call their doctor for medical advice about side effects. Side effects can be reported to the FDA at 1-800-FDA-1088. Side effects can also be reported to Global Blood Therapeutics at 1-833-428-4968 (1-833-GBT-4YOU).

Full Prescribing Information for Oxbryta is available at Oxbryta.com.

About Global Blood TherapeuticsGlobal Blood Therapeutics (GBT) is a biopharmaceutical company dedicated to the discovery, development and delivery of life-changing treatments that provide hope to underserved patient communities. Founded in 2011, GBT is delivering on its goal to transform the treatment and care of sickle cell disease (SCD), a lifelong, devastating inherited blood disorder. The company has introduced Oxbryta (voxelotor), the first FDA-approved treatment that directly inhibits sickle hemoglobin polymerization, the root cause of red blood cell sickling in SCD. GBT is also advancing its pipeline program in SCD with inclacumab, a p-selectin inhibitor in development to address pain crises associated with the disease. In addition, GBTs drug discovery teams are working on new targets to develop the next generation of treatments for SCD. To learn more, please visit http://www.gbt.com and follow the company on Twitter @GBT_news.

Forward-Looking StatementsCertain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995, including statements containing the words will, anticipates, plans, believes, forecast, estimates, expects, and intends, or similar expressions. These forward-looking statements are based on GBTs current expectations and actual results could differ materially. Statements in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. GBT intends these forward-looking statements, including statements regarding GBTs priorities, dedication, focus, goals and vision; the safety, efficacy and mechanism of action of Oxbryta and other product characteristics; the commercialization, delivery, availability, use, and commercial and medical potential of Oxbryta; ongoing and planned studies of Oxbryta and related protocols, activities and expectations; the potential expansion of the approved use of Oxbryta for more patients in the U.S., and potential regulatory approval for Oxbryta to treat patients in Europe; raising awareness of SCD in Europe; altering the treatment, course and care of SCD and mitigating related complications; need to improve the quality of care for SCD patients in Europe; the potential of inclacumab; and advancing GBTs pipeline, working on new targets and discovering, developing and delivering treatments, to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Securities Exchange Act, and GBT makes this statement for purposes of complying with those safe harbor provisions. These forward-looking statements reflect GBTs current views about its plans, intentions, expectations, strategies and prospects, which are based on the information currently available to the company and on assumptions the company has made. GBT can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved, and, furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a variety of risks and factors that are beyond GBTs control including, without limitation, risks and uncertainties relating to the COVID-19 pandemic, including the extent and duration of the impact on GBTs business, including commercialization activities, regulatory efforts, research and development, corporate development activities and operating results, which will depend on future developments that are highly uncertain and cannot be accurately predicted, such as the ultimate duration of the pandemic, travel restrictions, quarantines, social distancing and business closure requirements in the U.S. and in other countries, and the effectiveness of actions taken globally to contain and treat the disease; the risks that GBT has only recently established its commercialization capabilities and may not be able to successfully commercialize Oxbryta; risks associated with GBTs dependence on third parties for development, manufacture and commercialization activities related to Oxbryta; government and third-party payor actions, including those relating to reimbursement and pricing; risks and uncertainties relating to competitive products and other changes that may limit demand for Oxbryta; the risks regulatory authorities may require additional studies or data to support continued commercialization of Oxbryta; the risks that drug-related adverse events may be observed during commercialization or clinical development; data and results may not meet regulatory requirements or otherwise be sufficient for further development, regulatory review or approval; compliance with the funding and other obligations under the Pharmakon loan; and the timing and progress of GBTs and Syros research and development activities under their collaboration; along with those risks set forth in GBTs Annual Report on Form 10-K for the fiscal year ended December 31, 2019, and in GBTs most recent Quarterly Report on Form 10-Q filed with the U.S. Securities and Exchange Commission, as well as discussions of potential risks, uncertainties and other important factors in GBTs subsequent filings with the U.S. Securities and Exchange Commission. Except as required by law, GBT assumes no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

References

Contact: Steven ImmergutGBT650-410-3258media@gbt.com

See the rest here:
GBT Presents Data at 15th Annual Scientific Conference on Sickle Cell and Thalassemia - BioSpace

"Clinicians can now have greater confidence in the robustness, reliability and proven clinical utility of the cobas EGFR Mutation Test v2 when evaluating lung cancer patients who may benefit from targeted EGFR TKI therapies," said Neil Gunn, Head of Roche Sequencing Solutions. "By approving a single test for a broad group of therapies, this new and innovative approach by the FDA[3] can pave the way for future EGFR TKI therapies to utilise the cobas EGFR Mutation Test v2 to help identify patients for personalised medicine."

About the cobas EGFR Mutation Test v2Thecobas EGFR Mutation Test v2 is a real-time polymerase chain reaction (PCR) test for the qualitative detection of defined mutations of the epidermal growth factor receptor (EGFR) gene in non-small cell lung cancer (NSCLC) patients. Defined EGFR mutations are detected using DNA isolated from formalin-fixed paraffin-embedded tumor tissue (FFPET) or circulating tumour DNA (ctDNA) from plasma derived fromethylenediamine tetraacetic acid (EDTA) anti-coagulated peripheral whole blood.

About RocheRoche is a global pioneer in pharmaceuticals and diagnostics focused on advancing science to improve people's lives. The combined strengths of pharmaceuticals and diagnostics under one roof have made Roche the leader in personalised healthcare a strategy that aims to fit the right treatment to each patient in the best way possible.

Roche is the world's largest biotech company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology and diseases of the central nervous system. Roche is also the world leader in in vitro diagnostics and tissue-based cancer diagnostics, and a frontrunner in diabetes management.

Founded in 1896, Roche continues to search for better ways to prevent, diagnose and treat diseases and make a sustainable contribution to society. The company also aims to improve patient access to medical innovations by working with all relevant stakeholders. More than thirty medicines developed by Roche are included in the World Health Organization Model Lists of Essential Medicines, among them life-saving antibiotics, antimalarials and cancer medicines. Moreover, for the eleventh consecutive year, Roche has been recognised as one of the most sustainable companies in the Pharmaceuticals Industry by the Dow Jones Sustainability Indices (DJSI).

The Roche Group, headquartered in Basel, Switzerland, is active in over 100 countries and in 2019 employed about 98,000 people worldwide. In 2019, Roche invested CHF 11.7 billion in R&D and posted sales of CHF 61.5 billion. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. For more information, please visit http://www.roche.com.

All trademarks used or mentioned in this release are protected by law.

References

Roche Molecular Solutions Media RelationsElizabeth Baxter1.925.523.8812[emailprotected]

SOURCE Roche

https://www.roche.com/

Read more:
Roche receives FDA approval for the cobas EGFR Mutation Test v2 as the first companion diagnostic test for expanded EGFR TKI therapies in patients...

The report on Global Orthopedic Regenerative Medicine Market is a dependable point of reference heralding high accuracy business decisions on the basis of thorough research and observation by seasoned research professionals at CMI Research. The report on global Orthopedic Regenerative Medicine market evidently highlights the causal factors such as demand analysis, trend examination, and technological milestones besides manufacturing activities that have been systematically touched upon to instigate systematic growth projection.

This CMI Research report on global Orthopedic Regenerative Medicine market systematically studies and follows noteworthy progresses across growth trends, novel opportunities as well as drivers and restraints that impact growth prognosis.

Free Sample Report + All Related Graphs & Charts @:https://www.coherentmarketinsights.com/insight/request-sample/3566

Which market players and aspiring new entrants may witness seamless entry?

Curasan, Inc., Carmell Therapeutics Corporation, Anika Therapeutics, Inc., Conatus Pharmaceuticals Inc., Histogen Inc., Royal Biologics, Ortho Regenerative Technologies, Inc., Swiss Biomed Orthopaedics AG, Osiris Therapeutics, Inc., and Octane Medical Inc.

Predicting Scope: Global Orthopedic Regenerative Medicine Market, 2020-2027

Elaborate research proposes global Orthopedic Regenerative Medicine market is likely to experience an impressive growth through the forecast span, 2020-2027, ticking a robust CAGR of xx% USD. The Orthopedic Regenerative Medicine market is anticipated to demonstrate a whopping growth with impressive CAGR valuation. The Orthopedic Regenerative Medicine market is also likely to maintain the growth spurt showing signs of steady recovery.

For appropriate analysis of all the market relevant information as well emerging trends and historical developments in the Orthopedic Regenerative Medicine market, CMI Research has referred to various primary and secondary research practices and contributing factors.

Regional Overview: Global Orthopedic Regenerative Medicine Market

The report specifically sheds light upon note-worthy business discretion, popular trends investment probabilities aligning with budding opportunities as well as breakthrough developments in policies and monetary inclination echoing investor preferences in Orthopedic Regenerative Medicine space.

Competitive Landscape: Global Orthopedic Regenerative Medicine Market

Further in the report, readers are presented with minute details pertaining to significant company profiles, product development, on pricing, production and vital information on raw material and equipment developments also form crucial report contents in this CMI Research report.

Have Any Query? Ask Our Expert @: https://www.coherentmarketinsights.com/insight/request-customization/3566

Segmentation Based on Orthopedic Regenerative Medicine Market Types:

By Procedure Cell TherapyTissue EngineeringBy Cell TypeInduced Pluripotent Stem Cells (iPSCs)Adult Stem CellsTissue Specific Progenitor Stem Cells (TSPSCs),Mesenchymal Stem Cells (MSCs)Umbilical Cord Stem Cells (UCSCs)Bone Marrow Stem Cells (BMSCs)By SourceBone MarrowUmbilical Cord BloodAdipose TissueAllograftsAmniotic FluidBy ApplicationsTendons RepairCartilage RepairBone RepairLigament RepairSpine RepairOthers

Global Orthopedic Regenerative Medicine Market Size & Share, By Regions and Countries/Sub-regions:

Asia Pacific: China, Japan, India, and Rest of Asia Pacific

Europe: Germany, the UK, France, and Rest of Europe

North America: the US, Mexico, and Canada

Latin America: Brazil and Rest of Latin America

Middle East & Africa: GCC Countries and Rest of Middle East & Africa

The regional analysis segment is a highly comprehensive part of the report on the global Orthopedic Regenerative Medicine market. This section offers information on the sales growth in these regions on a country-level Orthopedic Regenerative Medicine market.

The historical and forecast information provided in the report span between2020 and 2027. The report provides detailed volume analysis and region-wise market size analysis of the market.

Report Investment, a Priority: Explains CMI Research

This report also helps market participants to organize R&D activities aligning with exact market requirements

The report resonates critical findings on decisive factors such as downstream needs and requirement specifications as well as upstream product and service development

The report aids in reader comprehension of the market based on dual parameters of value and volume.

This CMI Research initiated research output on Orthopedic Regenerative Medicine market is a ready-to-refer handbook of noteworthy cues for easy adoption by market players and stakeholders

CMI Research skillfully underpins a vivid segment analysis of the global Orthopedic Regenerative Medicine market, rendering appropriate inputs about the revenue generation capabilities of each segment.

Buy this research report @: https://www.coherentmarketinsights.com/insight/buy-now/3566

Thanks for reading our report. If you have any further questions, please contact us. Our team will provide you with the report tailored to your needs.

About Us:

Coherent Market Insightsis a prominent market research and consulting firm offering action-ready syndicated research reports, custom market analysis, consulting services, and competitive analysis through various recommendations related to emerging market trends, technologies, and potential absolute dollar opportunity.

Contacts Us: +1-206-701-6702Email:[emailprotected]Web:https://www.coherentmarketinsights.com/

Go here to see the original:
Covid-19 Impact On Orthopedic Regenerative Medicine Market 2020 Future Development, Manufacturers, Trends, Share, Size And Forecast to 2027 |...

News

New medicines for multiple sclerosis and various cancers will be available on the Pharmaceutical Benefits Scheme from 1 November.

Under recommendation by the Pharmaceutical Benefits Advisory Committee, the Pharmaceutical Benefits Scheme (PBS) will introduce and expand a number of listings from 1 November.The listings will provide greater access to potentially life-saving medicine treatments for people with multiple sclerosis (MS), as well as various forms of cancer.MS medicineMS is the most commonly acquired neurological disease in younger adults, with most people diagnosed between the ages of 2040. More than 25,600 Australians currently live with MS, and 75% of those are female.Although there are a number oftreatmentoptions available, there is currently no known cure for MS.Federal Health Minister Greg Hunt says there is a high clinical need for effective treatments for patients with progressive forms of relapse onset MS.Siponimod (sold as Mayzent) will be listed on the PBS for the first time to treat patients with secondary progressive MS.With average out-of-pocket costs of more than $25,000 per year, the PBS estimates around 800 patients will benefit from this listing each year.With PBS subsidy, patients will pay $41 per prescription of siponimod, or $6.60 with a concession card.Lymphoma, carcinoma and reproductive cancer medicinesMethoxsalen (sold as Uvadex) will be listed on the PBS for the first time for treatment of patients with erythrodermic cutaneous T-cell lymphoma (CTCL) who have not responded to other treatments.CTCL is a rare type of non-Hodgkins lymphoma that affects the skin and causes an accumulation of malignant T-cells in the skin. It most common in adults aged 4060 years.Around 75 Australians per year will receive PBS-subsidised access to the new medication and specialised treatment for CTCL, which would otherwise cost them more than $3400 a year.Uvadex will be introduced alongside two new items on the Medicare Benefits Schedule (MBS) from 1 November for the use of extracorporeal photopheresis (ECP) in combination with the medicine to treat the condition, as recommended by the Medical Services Advisory Committee.These include:

cancer medicines multiple sclerosis PBS

Read more from the original source:
newsGP - PBS to provide access to life-saving MS and cancer medicines - RACGP

News Release

Tuesday, October 27, 2020

Researchers from the National Institutes of Health (NIH) have discovered a new inflammatory disorder called vacuoles, E1 enzyme, X-linked, autoinflammatory and somatic syndrome (VEXAS), which is caused by mutations in the UBA1 gene. VEXAS causes symptoms that included blood clots in veins, recurrent fevers, pulmonary abnormalities and vacuoles (unusual cavity-like structures) in myeloid cells. The scientists reported their findings in the New England Journal of Medicine.

Nearly 125 million people in the U.S. live with some form of a chronic inflammatory disease. Many of these diseases have overlapping symptoms, which often make it difficult for researchers to diagnose the specific inflammatory disease in a given patient.

Researchers at the National Human Genome Research Institute (NHGRI), part of the NIH, and collaborators from other NIH Institutes took a unique approach to address this challenge. They studied the genome sequences from more than 2,500 individuals with undiagnosed inflammatory diseases, paying particular attention to a set of over 800 genes related to the process of ubiquitylation, which helps regulate both various protein functions inside a cell and the immune system overall. By doing so, they found a gene that is intricately linked to VEXAS, a disease which can be life-threatening. So far, 40% of VEXAS patients who the team studied have died, revealing the devastating consequences of the severe condition.

Usually, researchers discover a previously unknown disease by studying several patients with similar symptoms, then searching for a gene or multiple genes that may play a role in causing the disease. However, this was not a viable option for the NIH research team.

We had many patients with undiagnosed inflammatory conditions who were coming to the NIH Clinical Center, and we were just unable to diagnose them, said David B. Beck, M.D., Ph.D., clinical fellow at NHGRI and lead author of the paper. Thats when we had the idea of doing it the opposite way. Instead of starting with symptoms, start with a list of genes. Then, study the genomes of undiagnosed individuals and see where it takes us.

Out of the genome sequences of 2,560 patients with undiagnosed inflammatory conditions, over 1,000 patients had undiagnosed recurrent fevers and body-wide inflammation. The rest, part of the NIH Undiagnosed Diseases Network, had unusual and unclassified disorders.

Our objective was to see if any of the 2,560 patients shared variations in the same gene, said Daniel Kastner, M.D., Ph.D., scientific director of the Intramural Research Program at NHGRI and a senior author of the paper. Instead of looking at clinical similarities, we were instead taking advantage of shared genomic similarities that could help us discover a completely new disease.

Out of the 800 genes, one stood out. Three middle-aged males had rare and potentially damaging genomic variants in the UBA1 gene, but each of the three males appeared to have two copies of the UBA1 gene with one copy harboring the mutation, which was not unexpected because humans usually have two copies of every gene. However, the UBA1 gene resides in the X chromosome, and males have only one X chromosome (and one Y chromosome).

We were amazed to see this and wondered what it could mean. And thats when it clickedthis was only possible if there was mosaicism in these men, said Dr. Beck.

Mosaicism occurs when some people have groups of cells with mutations that are different from the rest of the body. The team predicted that there were specific cells in the patients bodies that carried the UBA1 gene in its normal form while other cells carried the gene in its mutated form.

Using DNA-sequencing methodologies, the researchers found that the mosaicism was indeed present in the patients myeloid cells, which are responsible for systemic inflammation and act as the first line of defense against infections.

The researchers then analyzed the genome sequences of additional individuals from various NIH cohorts and databases, which led to the discovery of an additional 22 adult males with the UBA1 gene mutations. Most of the individuals had symptoms that included blood clots in veins, recurrent fevers, pulmonary abnormalities and vacuoles (unusual cavity-like structures) in the myeloid cells.

Out of the combined 25 individuals, researchers were able to find a link between the various clinical rheumatologic and blood-related diagnoses made for the patients. Because these conditions exist in people with UBA1 mutations, the team grouped the various conditions into a new disease: VEXAS.

By using this genome-first approach, we have managed to find a thread that ties together patients carrying all of these seemingly unrelated, disparate diagnoses, Dr. Kastner said.

The researchers hope that this new genome-first strategy will help healthcare professionals improve disease assessments and provide appropriate treatments for thousands of patients who have various inflammation-related conditions. The study may also pave the way for a new and more appropriate classification of inflammatory diseases.

Additional research support for this study was provided by the National Institute of Arthritis and Musculoskeletal and Skin Diseases, the National Institute of Dental and Craniofacial Research, the National Heart, Lung, and Blood Institute, National Institute of Allergy and Infectious Diseases, the National Cancer Institute and the NIH Clinical Center.

NHGRI is one of the 27 institutes and centers at the National Institutes of Health. The NHGRI Extramural Research Program supports grants for research, and training and career development at sites nationwide. Additional information about NHGRI can be found athttps://www.genome.gov.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

###

See the article here:
Scientists use clues in the human genome to discover new inflammatory syndrome - National Institutes of Health

Find out which biopharma companies are raking in the cash this week, as companies from around the globe provide updates on their financing rounds and IPOs.

Foghorn Therapeutics

Foghorn boomed onto the NASDAQ with a $120 million IPO, surpassing its original expectations by $20 million. The Massachusetts-based biotech is investing in their proprietary Gene Traffic Control system, which tells the cells when to express which genes where and in what order within the chromatin regulatory system. The company currently has over 10 programs in its pipeline. Foghorn is anticipating filing an IND later this year for its lead candidate to begin a Phase I trial for the treatment of uveal melanoma, a cancer of the eye.

Scorpion Therapeutics

Scorpion Therapeutics aims to put the sting in cancer with a $108 million launch to pursue what they call precision oncology 2.0. The company plans to tackle the existing challenges of precision medicine with a three-prong approach developing better medicines for known cancer-driving mutations, developing drugs for targets previously thought to be undruggable, and finding new, untapped targets that could lead to even better drugs. Scorpion is focused on advancing several pipeline programs toward the clinic, while working on identifying new targets. We envision this being a robust pipelineits not about one or two programs, said Lina Gugucheva, Scorpion's chief business officer. Once were in the clinic, we will continue to add multiple programs into the clinic with no rate abatement, or speed abatement, in the next five- to 10-year horizon.

AavantiBio

Former Sarepta Therapeutics executive Bo Cumbo left to launch his new gene therapy company, AavantiBio, with a $107 million Series A. AavantiBios strategic partnership with University of Floridas Powell Gene Therapy Center provide their foundational research in rare genetic disorders. The company's lead program is aimed at Friedrichs Ataxia, a rare inherited genetic disease that causes cardiac and central nervous system dysfunction. AavantiBio has a unique opportunity to change the lives of those living with FA and other rare diseases, Cumbo said. Central to our mission is building on the advancements in gene transfer therapies and harnessing these revolutionary technologies to realize their potential as life-altering medicines.

Sirnaomics

An oversubscribed Series D will help biopharma company Sirnaomics' continued development of novel RNAi therapeutics to treat a range of disorders including cancers, fibrosis, metabolic diseases and viral infections. The $105 million round was co-led by existing investor Rotating Boulder Fund, and new investors Walvax Biotechnology and Sunshine Riverhead Capital. Sirnaomics is the only biopharma conducting R&D and clinical development in the field of RNAi therapeutics in both the U.S. and China. In April they saw positive results for their Phase IIa test of candidate STP705 in squamous cell carcinoma. They are preparing for an IPO in the near future.

Be Biopharma

Be Bio is a leader in developing B cells as medicines. B cells are prolific protein producers that can be collected from peripheral blood, have a programmable lifetime that could last decades, can target specific tissues, and have customizable functionality. Be Bio plans to use their $52 million Series A to precisely engineer B cells to treat a range of diseases. Our mission is to develop what we see as a new class of cell medicines that have a broad new pharmacology, Aleks Radovic-Moreno, Ph.D., said of B cells potential. We think it's a big new white space that's enabled by the rich biology of these cells.

Orbus Therapeutics

An expanded Series A will support Orbus ongoing STELLAR study, a Phase III clinical trial studying eflornithine in patients with anaplastic astrocytoma, a type of brain tumor, whose cancer has recurred following radiation and adjuvant chemotherapy. The $71 million funding includes an initial financing of $32.5 million in 2015. In the U.S., more than 3,600 new cases of anaplastic glioma are diagnosed each year with a median survival of just over three years despite treatment. The STELLAR studys primary endpoint is duration of overall survival in the 340 patients they plan to enroll.

Primmune Therapeutics

Primmune aims to advance the development of their novel orally-administered, small molecule toll-like receptor 7 agonists as therapeutic-adjuvants for acute viral diseases and cancer. The company's $27.4 million Series A gives it the capital to take their PRTX007 candidate into a short-term pharmacokinetic, pharmacodynamic and tolerability study, followed by a clinical proof-of-concept in an acute viral disease setting. Primmune's early data shows the potential for PRTX007 to drive the natural innate immune response to combat systemic diseases.

Prokarium

Privately-held Prokarium closed a $21 million Series B led by Korea Investment Partners with participation from the UK governments Future Fund, Flerie Invest and Riyadh Valley Company. The funds will be utilized to support the clinical development of Prokariums microbial immunotherapy for non-muscle invasive bladder cancer as well as expansion of its pipeline across multiple solid tumors. The bladder cancer therapy is expected to be in the clinic by 2022. Prokarium added Hyam Levitsky, MD, to its Board of Directors and strengthened the executive team with the following promotions: Kristen Albright, PharmD to COO, Livija Deban, Ph.D., to VP of Research and Peter McGowan, FCCA, as CFO.

Read the original post:
Biopharma Money on the Move: October 21-27 - BioSpace