CSL’s Broad Response to COVID-19 and Advancements across the Company’s Strategic Scientific Platforms are the Focus of Annual R&D Day – PRNewswire

MELBOURNE, Australia and KING OF PRUSSIA, Pa., Oct. 20, 2020 /PRNewswire/ -- In its annual R&D briefing to investors today, CSL Limited (ASX:CSL; USOTC:CSLLY) demonstrated how the company is advancing a novel research portfolio across four strategic scientific platforms (Plasma Fractionation, Recombinant Technology, Cell and Gene Therapy, Adjuvanted Cell and Egg-based Vaccines) across six therapeutic areas (Immunology, Hematology, Respiratory, Cardiovascular and Metabolic, Transplant, Influenza Vaccines) and two businesses (CSL Behring and Seqirus) to help patients lead full lives, protect public health and drive future business growth. It's this scientific capability and diverse portfolio that positioned the company to quickly respond to the COVID-19 public health crisis.

"CSL's acumen in vaccines, monoclonal antibodies, recombinant technologies, manufacturing capabilities and external partnerships, along with a therapeutic area focus and insight that includes Immunology and Respiratory, has supported the growth and progress of our pipeline and has also enabled us to respond quickly to the need for potential solutions in the world's fight against COVID-19," said Bill Mezzanotte, MD, MPH,Executive Vice President, Head of Research and Development and Chief Medical Officer. "Right across the spectrum of infection and disease, CSL has taken on projects we think make sense both scientifically and that fit our capabilities."

The company's efforts in the fight against COVID-19 include:

"The same collaborative mindset with which CSL has responded to the COVID-19 pandemic is also reflected in how we are advancing our pipeline," said Andrew Nash, PhD, Senior Vice President, Head of Research and Chief Scientific Officer. "Across therapeutic areas, strategic platforms and geographies we continue to grow our footprint and capabilities so that we can deliver a significant benefit to patients around the world."

Other advancements in CSL's R&D pipeline in the last year include:

Setting the pace in Plasma Product development:CSL is continuously improving current plasma fractionation processes and developing new therapeutic options, which has shown major relevance in the fight against COVID-19 and is also evident in other therapeutic areas. For example:

Advancing its Cell and Gene Therapy research by:

Building momentum in Recombinant Technology:CSL has established a highly differentiated preclinical and clinical stage pipeline of recombinant proteins and monoclonal antibodies from its proficiency in protein biology and innate cell immunity. In addition to using this technology against COVID-19, the company is studying:

About CSL CSL (ASX:CSL) is a leading global biotechnology company with a dynamic portfolio of life-saving medicines, including those that treat hemophilia and immune deficiencies, as well as vaccines to prevent influenza. Since our start in 1916, we have been driven by our promise to save lives using the latest technologies. Today, CSL including our two businesses, CSL Behring and Seqirus - provides life-saving products to more than 70 countries and employs 27,000 people. Our unique combination of commercial strength, R&D focus and operational excellence enables us to identify, develop and deliver innovations so our patients can live life to the fullest. For more information about CSL Limited, visitwww.csl.com.

SOURCE CSL

http://www.csl.com

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CSL's Broad Response to COVID-19 and Advancements across the Company's Strategic Scientific Platforms are the Focus of Annual R&D Day - PRNewswire

Stem Cell Therapy Market Research Report Forecast to 2029 (Includes Business Impact of COVID-19) – Eurowire

Trusted Business Insights answers what are the scenarios for growth and recovery and whether there will be any lasting structural impact from the unfolding crisis for the Stem Cell Therapy market.

Trusted Business Insights presents an updated and Latest Study on Stem Cell Therapy Market. The report contains market predictions related to market size, revenue, production, CAGR, Consumption, gross margin, price, and other substantial factors. While emphasizing the key driving and restraining forces for this market, the report also offers a complete study of the future trends and developments of the market.The report further elaborates on the micro and macroeconomic aspects including the socio-political landscape that is anticipated to shape the demand of the Stem Cell Therapy market during the forecast period. It also examines the role of the leading market players involved in the industry including their corporate overview, financial summary, and SWOT analysis.

Get Sample Copy of this Report @ Stem Cell Therapy Market Research Report Forecast to 2029 (Includes Business Impact of COVID-19)

Abstract, Snapshot, Market Analysis & Market Definition: Stem Cell Therapy Market Industry / Sector Trends

Stem Cell Therapy Market size was valued at USD 7.8 billion in 2018 and is expected to witness 10.2% CAGR from 2021 to 2026.

U.S. Stem Cell Therapy Market Size, By Type, 2018 & 2025 (USD Million)

Rising prevalence of chronic diseases will positively impact the stem cell therapy market growth. Cardiovascular diseases, neurological disorders and other chronic conditions have resulted in high mortality over past few years. Conventional therapeutic methods and treatments are currently replaced due to lack of efficiency and efficacy. Recently developed stem cell therapies are capable of replacing defective cells to treat diseases that has reduced morbidity drastically. Therefore, people have now started relying on stem cell therapy that has long term positive effects.

Advancements in stem cell therapy in developed regions such as North America and Europe have boosted the industry growth. Since past few years, there have been several researches carried out for stem cell therapy. Currently developed stem cell therapies have shown positive outcomes in treatment of leukemia. Similarly, due to advancements in regenerative medicine, several other chronic conditions such as muscular dystrophy and cardiovascular diseases also have been cured. Aforementioned factors have surged the industry growth. However, high cost of allogenic stem cell therapy may hamper the industry growth to some extent.

Market Segmentation, Outlook & Regional Insights: Stem Cell Therapy Market

Stem Cell Therapy Market, By Type

Allogenic stem cell therapy segment held around 39% revenue share in 2018 and it is anticipated to grow substantially during the analysis timeframe. Allogenic stem cell is available as off the shelf therapy and it is easily scalable that helps in providing treatment without delay. Moreover, the procedure includes culturing donor-derived immunocompetent cells that are highly effective in treatment of several diseases. Stem cells obtained in allogenic therapy are free of contaminating tumor cells. This reduces risk for disease recurrence that will surge its demand thereby, stimulating segment growth.

Autologous stem cell therapy segment is estimated to witness 10.1% growth over the forthcoming years. People usually prefer autologous stem cell therapy as it has minimum risk of immunological rejection. However, on introduction of allogenic stem cell therapy, demand for autologous stem cell therapy has declined as it is difficult to scale up. However, there are concerns regarding risk of cross contamination during large scale manufacturing of autologous stem cell lines that will impede segmental growth to some extent.

Stem Cell Therapy Market, By Application

The neurology segment was valued at around USD 1.6 billion in 2018 and it is estimated that it will witness significant growth over the forthcoming years. Stem cells are used to replenish the disrupted neurological cells that help in quick patient recovery. Pluripotent stem cells provide a replacement for cells and tissues to treat Alzheimers, Parkinsons disease, cerebral palsy, amyotrophic lateral sclerosis, and other neurodegenerative diseases. Thus, the pivotal role of stem cells in treating the life-threatening neurological condition will escalate segment growth.

The cardiovascular segment will witness 10% growth over the analysis timeframe. Considerable segmental growth can be attributed to development in stem cell therapies that have enhanced recovery pace in patients suffering from cardiovascular diseases. Recently developed allogeneic stem cell therapies are efficient and easily available that have reduced the mortality rates in cardiovascular patients. Above mentioned factors will propel cardiovascular segment growth in near future.

Stem Cell Therapy Market, By End-users

The hospital segment held over 56% revenue share in 2018 and it is anticipated to grow significantly in near future. The rising preference for stem cell therapies offered by hospitals proves beneficial for business growth. Hospitals have affiliations with research laboratories and academic institutes that carry out research activities for developing stem cell therapies. On the introduction and approval of any novel stem therapy, hospitals implement it immediately. Associations with research and academic institutes further help hospitals to upgrade its stem cell treatment offerings that positively impact the segmental growth.

The clinics segment is expected to grow at around 10% during the forecast timeframe. Clinics specializing in providing stem cell therapies are well-equipped with advanced medical devices and superior quality reagents required for imparting stem cell therapies. However, as clinics offer specialized stem cell therapies, their treatment cost is much higher as compared to hospitals that may reduce its preference.

Stem Cell Therapy Market, By Region

North America stem cell therapy market held around 41.5% revenue share in 2018 and it is estimated to grow substantially in near future. Increasing the adoption of novel stem cell therapies will prove beneficial for regional market growth. Moreover, favorable government initiatives have a positive impact on regional market growth. For instance, the government of Canada has initiated Strategic Innovation Fund Program that invests in research activities carried out for stem cell therapies enabling development in stem cell therapy. Above mentioned factors are expected to drive the North America market growth.

Asia Pacific stem cell therapy market is anticipated to witness 10.8% growth in the near future owing to increasing awareness amongst people pertaining to the benefits of advanced stem cell therapies. Additionally, favorable initiatives undertaken by several organizations will promote industry players to come up with innovative solutions. For instance, according to Pharma Focus Asia, members of the Asia-Pacific Economic Cooperation collaborated with Life Sciences Innovation Forum to involve professionals having expertise in stem cell therapies from academia and research centers to promote developments in stem cell research. Thus, growing initiatives by organizations ensuring the availability of new stem cell therapies will foster regional market growth.

Latin America Stem Cell Therapy Market Size, By Country, 2025 (USD Million)

Key Players, Recent Developments & Sector Viewpoints: Stem Cell Therapy Market

Key industry players in the stem cell therapy market include Astellas Pharma Inc, Cellectis, Celyad, Novadip Biosciences, Gamida Cell, Capricor Therapeutics, Cellular Dynamics, CESCA Therapeutics, DiscGenics, OxStem, Mesoblast Ltd, ReNeuron Group, and Takeda Pharmaceuticals. Chief industry players implement several initiatives such as mergers and acquisitions to sustain market competition. Also, receiving approvals for stem cell therapy products from regulatory authorities fosters the companys growth. For instance, in March 2018, the European Commission approved Takedas Alofisel that is off-the-shelf stem cell therapy. Product approval will help the company to gain a competitive advantage and capture market share.

Stem Cell Therapy Industry Viewpoint

The stem cells industry can be traced back to the 1950s. In 1959 first animals were made by in-vitro fertilization by preserving the stem cells. Till 2000, research was being carried out on stem cells to study its therapeutic effect. In 2000, fund allocations were made to research on cells derived from aborted human fetuses. In the same year, scientists derived human embryonic stem cells from the inner cell mass of blastocytes. Later, in 2010, clinical trials for human embryonic stem cell-based therapy were initiated. As technology progressed, stem cell therapy for treating cancer was developed. However, due to ethical issues, the use of stem cells for curing diseases witnessed slow growth for a few years. But as the regulatory scenario changed, people started preferring stem cell therapies due to its better efficacy. Stem cell therapy is in the developing stage and has numerous growth opportunities in developing economies with a high prevalence of chronic diseases.

Key Industry Development

In September 2020, Takeda Pharmaceutical Company Limited announced the expansion of its cell therapy manufacturing capabilities with the opening of a new 24,000 square-foot R&D cell therapy manufacturing facility at its R&D headquarters in Boston, Massachusetts. The facility provides end-to-end research and development capabilities and will accelerate Takedas efforts to develop next-generation cell therapies, initially focused on oncology with the potential to expand into other therapeutic areas.

The R&D cell therapy manufacturing facility will produce cell therapies for clinical evaluation from discovery through pivotal Phase 2b trials. The current Good Manufacturing Practices (cGMP) facility is designed to meet all U.S., E.U., and Japanese regulatory requirements for cell therapy manufacturing to support Takeda clinical trials around the world.

The proximity and structure of Takedas cell therapy teams allow them to quickly apply what they learn across a diverse portfolio of next-generation cell therapies including CAR NKs, armored CAR-Ts, and gamma delta T cells. Insights gained in manufacturing and clinical development can be quickly shared across global research, manufacturing, and quality teams, a critical ability in their effort to deliver potentially transformative treatments to patients as fast as possible.

Takeda and MD Anderson are developing a potential best-in-class allogeneic cell therapy product (TAK-007), a Phase 1/2 CD19-targeted chimeric antigen receptor-directed natural killer (CAR-NK) cell therapy with the potential for off-the-shelf use being studied in patients with relapsed or refractory non-Hodgkins lymphoma (NHL) and chronic lymphocytic leukemia (CLL). Two additional Phase 1 studies of Takeda cell therapy programs were also recently initiated: 19(T2)28z1xx CAR T cells (TAK-940), a next-generation CAR-T signaling domain developed in partnership with Memorial Sloan Kettering Cancer Center (MSK) to treat relapsed/refractory B-cell cancers, and a cytokine and chemokine armored CAR-T (TAK-102) developed in partnership with Noile-Immune Biotech to treat GPC3-expressing previously treated solid tumors.

Takedas Cell Therapy Translational Engine (CTTE) connects clinical translational science, product design, development, and manufacturing through each phase of research, development, and commercialization. It provides bioengineering, chemistry, manufacturing, and control (CMC), data management, analytical, and clinical and translational capabilities in a single footprint to overcome many of the manufacturing challenges experienced in cell therapy development.

Key Insights Covered: Exhaustive Stem Cell Therapy Market 1. Market size (sales, revenue and growth rate) of Stem Cell Therapy industry. 2. Global major manufacturers operating situation (sales, revenue, growth rate and gross margin) of Stem Cell Therapy industry. 3. SWOT analysis, New Project Investment Feasibility Analysis, Upstream raw materials and manufacturing equipment & Industry chain analysis of Stem Cell Therapy industry. 4. Market size (sales, revenue) forecast by regions and countries from 2019 to 2025 of Stem Cell Therapy industry.

Research Methodology: Stem Cell Therapy Market

Looking for more? Check out our repository for all available reports on Stem Cell Therapy in related sectors.

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Stem Cell Therapy Market Research Report Forecast to 2029 (Includes Business Impact of COVID-19) - Eurowire

Stem Cell Therapy Market Trends, Opportunities, New Technologies and Developments, Competitor analysis and Market Sizing from 2020 to 2026 – PRnews…

The Stem Cell Therapy Market study analysis offers a complete evaluation of the Stem Cell Therapy Market which contains facts, thoughtful insights, historical data, and statistically supported and industry-validated market information. Stem Cell Therapy Market analyses also contain forecasts that are derived from an appropriate set of conventions and practices.

This research report is involved with the widespread usage of both primary & secondary data sources. The research report includes the analysis of several factors that are affecting the industry, along with the government policy, competitive landscape, and market environment, present trends in the market, technological development, upcoming technologies, and technical progress in related industries, and market risks, opportunities, market barriers, and challenges.

Get a Sample Copy of the Stem Cell Therapy Market Report with Latest Industry Trends @ https://www.adroitmarketresearch.com/contacts/request-sample/691?utm_source=Pallavi

Stem Cell Therapy Market 2020 Industry Research Report is a professional and in-depth study on the current state of the Global Stem Cell Therapy industry. Moreover, research report categorizes the global Stem Cell Therapy market by top players/brands, region, type and end user. Stem Cell Therapy Market report also tracks the latest market dynamics, such as driving factors, restraining factors, and industry news like mergers, acquisitions, and investments. It provides market size (value and volume), Stem Cell Therapy market share, growth rate by types, applications, and combines both qualitative and quantitative methods to make micro and macro forecasts in different regions or countries.

Click to access the full report and Table of Content at https://www.adroitmarketresearch.com/industry-reports/stem-cell-therapy-market?utm_source=Pallavi

Global Stem Cell Therapy Market: Competitive Analysis

This section of the report identifies various key manufacturers of the market. It helps the reader understand the strategies and collaborations that players are focusing on combat competition in the market. The comprehensive report provides a significant microscopic look at the market. The reader can identify the footprints of the manufacturers by knowing about the global revenue of manufacturers, the global price of manufacturers, and sales by manufacturers during the forecast period of 2019 to 2015.

Stem Cell Therapy Market Segmentation

Type Analysis of Stem Cell Therapy Market:

Based on cell source, the market has been segmented into,

Adipose Tissue-Derived Mesenchymal SCs Bone Marrow-Derived Mesenchymal SCs Embryonic SCs Other Sources

Applications Analysis of Stem Cell Therapy Market:

Based on therapeutic application, the market has been segmented into,

Musculoskeletal Disorders Wounds & Injuries Cardiovascular Diseases Gastrointestinal Diseases Immune System Diseases Other Applications

The Stem Cell Therapy market is analysed and market size information is provided by regions (countries). The report includes country-wise and region-wise market size for the period 2015-2026. It also includes market size and forecast by Type and by Application segment in terms of sales and revenue for the period 2015-2026.

The key regions covered in the Stem Cell Therapy market report are:

1. North America (U.S., Canada) 2. Europe (Germany, France, U.K., Italy, Russia) 3. Asia-Pacific (China, Japan, South Korea, India, Australia, Taiwan, Indonesia, Thailand, Malaysia, Philippines, Vietnam) 4. Latin America (Mexico, Brazil, Argentina) 5. Middle East & Africa (Turkey, Saudi Arabia, U.A.E.)

Some of the major objectives of this report:

1. To provide a detailed analysis of the market structure along with the forecast of the various segments and sub-segments of the global Stem Cell Therapy Market. 2. To provide insights about factors affecting market growth. To analyze the Stem Cell Therapy Market based on various factors- price analysis, supply chain analysis, porter five force analyses, etc. 3. To provide historical and forecast revenue of the Stem Cell Therapy Market segments and sub-segments with respect to four main geographies and their countries- North America, Europe, Asia, and the Rest of the World. 4. Country-level analysis of the market with respect to the current market size and future prospective. 5. To provide country-level analysis of the market for segment by application, product type, and sub-segments. 6. To provide strategic profiling of key players in the market, comprehensively analyzing their core competencies, and drawing a competitive landscape for the market. 7. Track and analyze competitive developments such as joint ventures, strategic alliances, mergers and acquisitions, new product developments, and research and developments in the Stem Cell Therapy Market.

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CIRM Bridges to Stem Cell Research & Therapy The Bridge to Everywhere (in biomedicine) OP-ED – The Silicon Valley Voice

In a recent clinical trial for an immune cell therapy for lymphoma, 62% of patients experienced complete cancer clearance in spite of the fact that some of them were on their 5th line of treatment. Stem cell therapies have the potential to enact more of these paradigm-shifting treatments. Proposition 14 will continue to advance these therapies and bring them to full development as available cures.

The vision of stem cell therapy is that a physician can just as easily grab an IV bag full of therapeutic cells as they might draw a drug into a syringe. Conceived through Proposition 71 in 2004, the California Institute for Regenerative Medicine (CIRM) serves as a vehicle to support all aspects of stem cell research. Medical progress requires not just well-designed clinical studies but also a well-trained workforce, educated at the intersection of stem cell biology, engineering, and chemistry.

Since 2008, CIRM has supported the training of nearly 1300 Community College and California State University students for the emerging field of Regenerative Medicine through the Bridges to Stem Cell Research and Therapy Program. The Bridges Training Program has functioned as a pathway for first-generation and underrepresented students from Humboldt to San Diego, to all of the biomedical sectors startup and cell therapy companies, academic research institutes, graduate and medical school, and more. Exposure to hands-on labs, advanced seminar discussions, and a required paid internship fully prepares these students for entering the stem cell workforce. Over 80% of Bridges alumni have either advanced to graduate school or joined the biomedical workforce in industry or academic institutions. These Programs bring a greater return than the initial cost of training.

SPONSORED

Consider Vahid Hamzeinejad, a bright high school student, headed to UC Berkeley to begin his college career. Enter the Great Recession; Vahid found himself back at home, working non-stop to help keep his parents restaurant afloat. Not giving up on his commitment to an education, he enrolled at the College of the Canyons. After completing an Associates degree, Vahid transferred to Cal Poly, hoping to join the Bridges Program. After receiving the Bridges core training, Vahid started his internship at ViaCyte, where he continues to work today, as a critical member of the team supporting ViaCytes clinical development of a functional cure for diabetes. The nearly $30 billion that California currently spends on diabetes treatments could be significantly reduced, in no small part due to the efforts of a student that cost taxpayers $36,000 to educate. That is before considering the benefit to patients quality of life that would occur by replacing insulin pumps, glucose monitors, and constant vigilance with a stem-cell-derived tissue that regulates blood sugar levels biologically making and secreting its own insulin.

Passing Proposition 14 will enable this and other unparalleled treatments for diabetes, heart disease, cancer, and neurological disorders.

Signed,

Robert Kam and the CIRM Bridges Program

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Orchard Therapeutics Receives Positive CHMP Opinion for Libmeldy for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD) | DNA RNA and…

Details Category: DNA RNA and Cells Published on Friday, 16 October 2020 14:20 Hits: 121

First therapy recommended for full marketing authorization in the EU for eligible patients with confirmed diagnosis of late infantile or early juvenile MLD variants

One-time treatment with Libmeldy has been shown to preserve cognitive and motor function in most patients

Libmeldy is backed by data across 35 patients with follow-up of up to 8 years post-treatment, demonstrating the potential durability of HSC gene therapy

BOSTON, MA, USA and LONDON, UK I October 16, 2020 I Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending full, or standard, marketing authorization for Libmeldy (cryopreserved autologous CD34+ cells encoding the arylsulfatase-A, or ARSA, gene), an investigational gene therapy for the treatment of metachromatic leukodystrophy (MLD), 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.

The CHMPs positive opinion will now be reviewed by theEuropean Commission(EC), which has the authority to grant marketing authorization for Libmeldy in theEuropean Union(EU). A final decision by the EC for Libmeldy is anticipated before the end of 2020. If approved, Libmeldy would be the first commercial therapy and first gene therapy for eligible patients with early-onset MLD.

MLD is a very rare, severe genetic condition caused by mutations in the ARSA gene which lead to neurological damage and developmental regression. In its most severe and common forms, young children rapidly lose the ability to walk, talk and interact with the world around them. A majority of these patients pass away in childhood, with palliative care often as their only option.

Todays positive CHMP opinion for marketing authorization of Libmeldy is a remarkable achievement that we share with the MLD community, as it brings us closer to delivering a one-time, potentially transformative therapy for eligible children suffering from this devastating disease, said Bobby Gaspar, M.D., Ph.D., chief executive officer, Orchard Therapeutics. Data from the Libmeldy clinical program have demonstrated the potential for long-term positive effects on cognitive development and maintenance of motor function, translating to individual preservation of motor milestones such as the ability to sit, stand and/or walk without support, as well as attainment of cognitive skills like social interactions and school attendance, at ages at which untreated patients show severe motor and cognitive impairments.

Libmeldy is designed as a one-time gene therapy, developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy, in which the patients own hematopoietic stem cells (HSCs) are selected, and functional copies of the ARSA gene are inserted into the genome of the HSCs using a lentiviral vector before these genetically modified cells are infused back into the patient. The ability of the gene-corrected HSCs to migrate across the blood-brain barrier into the brain, engraft, and express the functional enzyme has the potential to persistently correct the underlying genetic condition with a single treatment.

This is an important milestone toward making the availability of HSC gene therapy a reality for more patients, and it also is extremely rewarding for our multi-disciplinary team at SR-Tiget who has worked relentlessly along this 15-year journey to move the seminal proof of principle studies to the first in-human testing of this therapy, said SR-Tiget director Luigi Naldini, M.D, Ph.D. The robust and durable clinical benefits observed in early-onset MLD patients who received HSC gene therapy are compelling, especially when compared to the natural history of the disease. These results also further illustrate our view that the HSC gene therapy approach has the potential to deliver transformative effects in other storage diseases as well, especially when the cells are designed to overexpress the functional enzyme and provide an enhanced supply of it to the affected tissues.

As a parent, watching your child start down a seemingly normal developmental path only to suddenly and rapidly lose some or all of his or her abilities is heart-wrenching, and the agony is even more acute knowing no approved therapies currently exist for MLD, said Georgina Morton, Chair of ArchAngel MLD Trust. Todays decision to advance Libmeldy to the final EC approval stage represents a huge step forward for the parents of these young children and for all of us in the MLD community.

We are extremely appreciative of the EMAs expedited and thorough review of Libmeldys marketing authorization application, considering the severity of MLD coupled with the limited treatment options available today for young patients, said Anne Dupraz, chief regulatory officer, Orchard Therapeutics. The Agencys collaboration on this assessment is a testament to their broader public health commitment to ensure timely evaluation of new medicines for diseases where a pressing unmet need exists.

Data Supporting the Clinical Profile of Libmeldy

The positive CHMP opinion is supported by clinical studies of Libmeldy in both pre- and early- symptomatic, early-onset MLD patients. Early-onset MLD encompasses the disease variants traditionally referred to as late infantile (LI) and early juvenile (EJ).

Clinical efficacy was based on the integrated analysis of results from 29 patients with early-onset MLD who were all treated with Libmeldy prepared as a fresh (non-cryopreserved) formulation:

Clinical safety was evaluated in 35 patients with early-onset MLD:

Co-primary endpoints The co-primary endpoints of the integrated efficacy analysis were Gross Motor Function Measure (GMFM) total score and ARSA activity, both evaluated at 2 years post-treatment. Results of this analysis indicate that a single-dose intravenous administration of Libmeldy is effective in modifying the disease course of early-onset MLD in most patients.

Pre-symptomatic LI and EJ patients treated with Libmeldy experienced significantly less deterioration in motor function at 2 years and 3 years post-treatment, as measured by GMFM total score, compared to age and disease subtype-matched untreated patients (p0.008). The mean difference between treated pre-symptomatic LI patients and age-matched untreated LI patients was 71.0% at year 2 and 79.8% at year 3. Similarly, the mean difference between treated pre-symptomatic EJ patients and age-matched untreated EJ patients was 52.4% at year 2 and 74.9% at year 3. Although not statistically significant, a clear difference in GMFM total score was also noted between treated early-symptomatic EJ patients and age-matched untreated EJ patients (28.7% at year 2; p=0.350 and 43.9% at year 3; p=0.054).

A statistically significant increase in ARSA activity in peripheral blood mononuclear cells was observed at 2 years post-treatment compared to pre-treatment in both pre-symptomatic patients (20.0-fold increase; p<0.001) and early-symptomatic patients (4.2-fold increase; p=0.004).

At the time of the integrated data analysis, all treated LI patients were alive with a follow-up post-treatment up to 7.5 years and 10 out of 13 treated EJ patients were alive with a follow-up post-treatment of up to 6.5 years. No treatment-related mortality has been reported in patients treated with Libmeldy.

Key secondary endpoints For EJ patients who were early-symptomatic when treated with Libmeldy, meaningful effects on motor development were demonstrated when these patients were treated before entering the rapidly progressive phase of the disease (IQ85 and Gross Motor Function Classification (GMFC)1). By 4 years post-disease onset, an estimated 62.5% of treated, early-symptomatic EJ MLD patients survived and maintained locomotion and ability to sit without support compared with 26.3% of untreated early-symptomatic EJ MLD patients, representing a delay in disease progression following treatment with Libmeldy.

A secondary efficacy endpoint that measured cognitive and language abilities as quantified by Intelligence Quotient/Development Quotient (IQ/DQ) found:

Clinical safety Safety data indicate that Libmeldy was generally well-tolerated. The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies (AAA) reported in 5 out of 35 patients. Antibody titers in all 5 patients were generally low and no negative effects were observed in post-treatment ARSA activity in the peripheral blood or bone marrow cellular subpopulations, nor in the ARSA activity within the cerebrospinal fluid. 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.

About MLD and Investigational Libmeldy

Metachromatic leukodystrophy (MLD) is a rare and life-threatening inherited disease of the bodys metabolic system occurring in approximately one in every 100,000 live births. MLD is caused by a mutation in thearylsulfatase-A(ARSA) gene that results in the accumulation of sulfatides in the brain and other areas of the body, including the liver, gallbladder, kidneys, and/or spleen. Over time, the nervous system is damaged, leading to neurological problems such as motor, behavioral and cognitive regression, severe spasticity and seizures. Patients with MLD gradually lose the ability to move, talk, swallow, eat and see. Currently, there are no approved treatments for MLD. In its late infantile form, mortality at 5 years from onset is estimated at 50% and 44% at 10 years for juvenile patients.1Libmeldy (autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase-A (ARSA) gene), formerly OTL-200, is being studied for the treatment of MLD in certain patients. Libmeldy was acquired from GSK inApril 2018and originated from a pioneering collaboration between GSK and the Hospital San Raffaele and Fondazione Telethon, acting through their jointSan Raffaele-Telethon Institute for Gene TherapyinMilan, initiated in 2010.

About Orchard

Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and theSan Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

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

1Mahmood et al. Metachromatic Leukodystrophy: A Case of Triplets with the Late Infantile Variant and a Systematic Review of the Literature.Journal of Child Neurology2010, DOI:http://doi.org/10.1177/0883073809341669

SOURCE: Orchard Therapeutics

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Orchard Therapeutics Receives Positive CHMP Opinion for Libmeldy for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD) | DNA RNA and...

COVID-19 and the trials of treatment | News, Sports, Jobs – Escanaba Daily Press

Features

Oct 16, 2020

Karen Wils photo Shown at right is Karen Wils, the Chemo Beagler or the COVID Beagler.

ESCANABA The year 2020 has put me and most everybody else in a very unusual position.

We all feel a little trapped and uneasy thrown into a situation we have never faced before.

I always kind of thought I would need to have a stem cell transplant someday after I was diagnosed with multiple myeloma in 2008.

I often referred to this blood cancer as my pesky woodtick. Fortunately, I have been blessed with good control of the disease until this year.

Leaving home, going to the Mayo Clinic and going through transplant is enough excitement for one person, but to be doing that while COVID-19 plagues our country is almost too much.

To say the last few months have been an interesting journey would be putting it mildly.

As the summer drew to a close, many changes were closing in on me, too. My son went off to a new job, and my daughter left for college. The very next week, my husband and I left for Minnesota for an unknown amount of weeks for my stem cell transplant.

For three-and-a-half weeks we were at Mayo. My husband was my excellent care giver through the strong chemo, and the transplant of my own stem cells and the long road to recovery.

Doing all of this during a pandemic is really interesting. The Mayo Clinic takes COVID-19 very seriously. So with much testing, temperature taking, sanitizing and constant mask wearing, we maneuvered through the treatment.

Social distancing when you are staying at the Gift of Life Transplant House and so far from home is very difficult. Phone calls, Zoom and Facebook became my life link to the kids, family and friends.

After going through some pretty weak days, finally my new immune system was strong enough for me to go home.

Oh, the great joy of heading down those last miles from Menominee to Escanaba back into the land of trees and water!

Sleeping in my own bed, seeing my pets, and being able to stand in my own garden felt so wonderful, but the house was so quiet without the kids.

The COVID-19 cautions had to continue, too.

I wear my mask and feel a little bit trapped, like everybody else these days. I pray that I am making all healthy decisions when I encourage my children to go to church or my husband to go to the grocery store.

The COVID-19 virus threatens big things and little things like family traditions that have to be rethought. With great sadness, I postponed until next year our annual camp gathering/harvest festival. So many folks have been through canceled or postponed parties, birthdays, anniversaries and funerals.

As hard and as disappointing as this is, the important things remain. The trees still are changing to awesome autumn colors. The harvest season is in full swing. The wild animals are still thriving outside our windows, and our families are together even if we cant do everything we want to do right now.

As I get stronger every day, I thank God. I pray for a cure for COVID-19. Yoopers are tough and our traditions will thrive long after this pandemic!

Karen (Rose) Wils is a lifelong north Escanaba resident. Her folksy columns appear weekly in Lifestyles.

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Gamida Cell and Be The Match BioTherapies Expand Strategic Collaboration – BioSpace

Oct. 13, 2020 12:01 UTC

Organizations to work together to integrate Be The Match BioTherapies existing cell therapy infrastructure to manage the supply chain for potential commercialization of omidubicel

BOSTON & MINNEAPOLIS--(BUSINESS WIRE)-- Gamida Cell Ltd.. (Nasdaq: GMDA), a leading cellular and immune therapeutics company, and Be The Match BioTherapies, an organization offering solutions for companies developing and commercializing cell and gene therapies, today announced an expansion of their existing strategic collaboration for omidubicel, Gamida Cells advanced cell therapy in Phase 3 clinical development as a potentially life-saving treatment option for patients in need of an allogeneic hematopoietic stem cell (bone marrow) transplant. The broadened agreement represents an important step in both organizations patient access efforts and in Gamida Cells preparation for potential approval by the U.S. Food and Drug Administration (FDA).

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20201013005030/en/

The original partnership agreement between the organizations focused on the omidubicel development program and leveraged a wide range of Be The Match BioTherapies capabilities and services. This included providing cellular source material from the Be The Match Registry, which offers the most ethnically diverse listing of potential donors and umbilical cord blood units in the world, with 22 million potential donors and more than 300,000 umbilical cord blood units, as well as cell therapy supply chain and logistics management services. In building upon the existing collaboration, Gamida Cell will work through Be The Match BioTherapies for the ordering and supply of cord blood units, which serve as the starting material for omidubicel. The expanded agreement is designed to provide a smooth process throughout the omidubicel therapy supply chain.

Gamida Cells work to bring a new stem cell graft source to patients aligns with our core mission to help organizations deliver cellular therapies that can save more lives and improve the quality of life for patients, said Amy Ronneberg, chief executive officer of the National Marrow Donor Program/Be The Match and Be The Match BioTherapies. Were delighted to expand upon our collaboration to more fully leverage our infrastructure and technology to support the advancement of Gamida Cells efforts to seamlessly bring omidubicel to patients in clinical and commercial settings. Gamida Cells efforts to make stem cell transplant more accessible to patients could be particularly impactful for patients who do not have a matched donor of suitable age.

Be The Match BioTherapies is a respected leader in cell therapy and has an extensive history of assuring broad transplant access through the delivery of source material, globally, for patients in need of a transplant, stated Michele Korfin, chief operating and commercial officer of Gamida Cell. Deepening our collaboration represents an important step for Gamida Cell as the company increases its focus on potentially bringing omidubicel to patients in the commercial setting after reporting that omidubicel met its primary endpoint and all three secondary endpoints in our randomized, multi-center Phase 3 study. We look forward to our continued collaboration with Be The Match BioTherapies to ensure that we have an efficient and reliable cell therapy supply chain that can provide a positive experience for transplant teams and their patients.

In May, Gamida Cell reported that its Phase 3 study of omidubicel met its primary endpoint, demonstrating a highly statistically significant reduction in time to neutrophil engraftment, a key milestone in recovery from a stem cell transplant. Additionally, in October, Gamida Cell reported that all three secondary endpoints for the study related to platelet engraftment, infections and hospitalizations demonstrated statistical significance. Gamida Cell expects to begin submitting the biologics license application for omidubicel to the FDA on a rolling basis in the fourth quarter of 2020.

Despite the curative potential of bone marrow transplants, it is estimated that more than 40 percent of eligible patients in the U.S. do not receive one for various reasons, including difficulty in finding a matched donor. Omidubicel is designed to potentially serve as a universal alternative to existing donor sources for bone marrow transplant.

About Omidubicel Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers). In clinical studies (NCT01816230 and NCT02730299), omidubicel demonstrated rapid and durable time to engraftment and was generally well tolerated. Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia (NCT03173937). The aplastic anemia investigational new drug application is currently filed with the FDA under the brand name CordIn, which is the same investigational development candidate as omidubicel. For more information on clinical trials of omidubicel, please visit http://www.clinicaltrials.gov.

Omidubicel is an investigational therapy, and its safety and efficacy have not been evaluated by the U.S. Food and Drug Administration or any other health authority.

About Gamida Cell Gamida Cell is an advanced cell therapy company committed to cures for patients with blood cancers and serious blood diseases. We harness our cell expansion platform to create therapies with the potential to redefine standards of care in areas of serious medical need. For additional information, please visit http://www.gamida-cell.com or follow Gamida Cell on LinkedIn or Twitter at @GamidaCellTx.

About Be The Match BioTherapies Be The Match BioTherapies is the only cell and gene therapy solutions provider with customizable services to support the end-to-end cell therapy supply chain. Backed by the industry-leading experience of the National Marrow Donor Program (NMDP)/Be The Match, and a research partnership with the CIBMTR (Center for International Blood and Marrow Transplant Research), the organization designs solutions that advance the development of cell and gene therapies across the globe.

Be The Match BioTherapies is dedicated to accelerating patient access to life-saving cell and gene therapies by providing high-quality cellular source material from the Be The Match Registry, the worlds largest and most diverse registry of more than 22 million potential blood stem cell donors and more than 300,000 umbilical cord blood units. Through established relationships with apheresis, marrow collection and transplant centers worldwide, the organization develops, onboards, trains and manages expansive collection networks to advance cell therapies. Be The Match BioTherapies uses a proven integrated model of both cell therapy supply chain and logistics managers, complimented by regulatory compliance experts to successfully transport and deliver life-saving therapies across the globe. Through the CIBMTR, Be The Match BioTherapies extends services beyond the cell therapy supply chain to include long-term follow-up tracking for the first two FDA-approved CAR-T therapies.

For more information, visit http://www.BeTheMatchBioTherapies.com or follow Be The Match BioTherapies on LinkedIn or Twitter at @BTMBioTherapies.

Gamida Cell Forward Looking Statements This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including with respect to the effect on any cell therapy supply chain or Gamida Cells anticipated timing regulatory filing submissions for omidubicel, which statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to the ongoing global COVID-19 pandemic and manufacturing, clinical, scientific, regulatory and technical developments. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section and other sections of Gamida Cells Annual Report on Form 20-F, filed with the Securities and Exchange Commission (SEC) on February 26, 2020, and other filings that Gamida Cell makes with the SEC from time to time (which are available at http://www.sec.gov), the events and circumstances discussed in such forward-looking statements may not occur, and Gamida Cells actual results could differ materially and adversely from those anticipated or implied thereby. Any forward-looking statements speak only as of the date of this press release and are based on information available to Gamida Cell as of the date of this release.

View source version on businesswire.com: https://www.businesswire.com/news/home/20201013005030/en/

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Gamida Cell and Be The Match BioTherapies Expand Strategic Collaboration - BioSpace

UofL-born company secures an additional $115 million to advance cell therapy – uoflnews.com

A company born at the University of Louisville has raised an additional $115 million to further develop a therapy that could improve the lives of kidney transplant recipients.

The company, Talaris Therapeutics Inc., plans to use the series B funding to support a Phase 3 clinical trial the last step before applying for FDA approval. This follows a $100 million series A funding round announced last year to hire staff and initiate the trial.

Talaris recently began dosing patients in that trial of the unique cell therapy, called FCR001, which allows living-donor kidney transplant recipients to stay off immunosuppression drugs they would otherwise need for the rest of their lives.

Immunosuppressants help prevent rejection of the transplanted organ, but can cause side effects, including high blood pressure, diabetes, high cholesterol, neurological effects, increased risk of infection and decline in kidney function over time.

This technology could help transplant patients live fuller, healthier lives,said UofL Executive Vice President for Research and Innovation, Kevin Gardner.This UofL research, in the hands of strong industry partners and backed by investors, is a big step in advancing our health.

FCR001 consists of stem cells taken from the organ donor and processed at the Talaris facility, then infused into the organ transplant recipient. The goal is to create a durable dual immune system (part donor-derived and part recipient) in the transplant recipient. These two immune systems coexist, recognizing both the recipients own body as well as the donated organ as self.

The new funding also will advance aplanned Phase 2 trial of FCR001 in diffuse systemic sclerosis, a severe form of the rare autoimmune disease scleroderma.

Talaris, formerly known as Regenerex LLC, was founded by UofL researcher and innovator Suzanne Ildstad to commercialize the pioneering work of her team at the university. She now serves as Talaris chief scientific officer.

This financing moves us one step closer to helping organ transplant recipients no longer be dependent on immunosuppressive drugs, resulting in a greatly improved quality of life, Ildstad said of the earlier Phase 3 trial financing. The support and research infrastructure at UofL have been invaluable in our journey to this important juncture.

In an earlier Phase 2 trial, FCR001 allowed 70% of living donor kidney transplant patients durably to be weaned off all of their immunosuppression treatments.

The Phase 3 trial is expected to enroll 120 adult living donor kidney transplant recipients at multiple sites across the U.S. To date, the trial has been initiated at five clinical sites around the country.

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UofL-born company secures an additional $115 million to advance cell therapy - uoflnews.com

Houghton Regis dad appeals for help for life-saving treatment abroad – Dunstable Today

A father from Houghton Regis who was diagnosed with liver cancer is hoping to raise money for life-saving treatment in Holland.

Stuart Finlay (Finn), 36, was diagnosed with 10 weeks ago and was told the life expectancy was ten years with 44 per cent of the cases, and five years with 60 per cent of the cases, but after doing some research he found studies of treatment using different drugs that have seen people at stage 3 cancer making a full recovery.

He is now planning to get treatment that is not available in the UK , so he will be travelling to Holland and Frankfurt for two different procedure that he believes will help him more.

He said: "Fortunately, I was diagnosed early so the chances of survival are better than they normally would be .

"The treatment from the nurses and doctors with the NHS has been amazing but with Covid19 here it has been made harder then normal, so thank you all for your help and everything you have done for me.

"So far I have started chemotherapy and had cryotherapy which has freezed the tumor to kill any cancer cells and the tumor itself and then I will be starting a course of chemotherapy at the end of Oct, this is all that has been offered to me and my life expectancy with this treatment is 5 years.

"I will be getting targeted therapy in the Holland from UMC, VUmc Holland and then in Frankfurt, Germany, Stem cell Therapy."

The former army soldier, who has a three-year-old son and 14-year-old daughter, is now appealing for the public's help to raise the money for the treatment.

Finn said: "Having already put all the money I can into the treatment, my friend suggested making a page and seeing if any friends or family could help, and it started from there.

"The main thing for me is to raise awareness of this type of treatment and remove the stigma that is attached to CBD - which has really helped me - and stem cell treatment.

"If I don't survive this I want to be able to help someone else. I want to raise awareness of these treatments.

"I will be making videos of the treatment that I will be taking and of each of the doctors as well about this so that if anyone else thinks there is no hope trust me there is.

"I will be asking questions about it and how it works and if you have any questions please send them to me and I will ask them.

"You will be able to watch my Vlogs of everything I do and everywhere I go and what happens when you get these treatments."

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Houghton Regis dad appeals for help for life-saving treatment abroad - Dunstable Today

VERIFY: Was the antibody cocktail used to treat President Trump developed using human embryonic stem cells? – CBS News 8

Social media is buzzing with the claim President Trump, who is pro-life, used an antibody cocktail developed using human embryonic stem cells.

The antibody cocktail used to treat President Donald Trump for COVID-19 is getting a lot of attention on social media.

Some users are claiming Regeneron - the company that developed the treatment - used human embryonic stem cells to create it, but is this true?

News 8 reached out to Regeneron for comment.

"This particular discovery program (regn-cov2) did not involve human stem cells or embryonic stem cells," wrote Regeneron spokesperson Alexandra Bowie in a statement.

So, where did that claim about human embryonic stem cells come from?

It appears to have developed from this statement Regeneron issued back in April 2020 regarding stem cell research:

"As is the case with many other science-focused biotechnology companies, Regeneron uses a wide variety of research tools and technologies to help discover and develop new therapeutics. stem cells are one such tool. the stem cells most commonly used at Regeneron are mouse embryonic stem cells and human blood stem cells. currently, there are limited research efforts employing human-induced pluripotent stem cell lines derived from adult human cells and human embryonic stem cells that are approved for research use by the national institutes of health and created solely through in vitro fertilization."

According to the American Association for the Advancement of Science, here's what the antibody cocktail used to treat the president is made of:

"One antibody comes from a human who had recovered from a SARS-COV-2 infection; a B cell that makes the antibody was harvested from the person's blood and the genes for the immune protein isolated and copied. The other antibody is from a mouse, which was engineered to have a human immune system, that had the spike protein injected into it."

Bowie also told News 8 the statement about stem cell research on its website reflects the company's general position on stem cell research, but does not mean human embryonic stem cells were used in creating the antibody cocktail used to treat the president.

Nevertheless, some said the company's position on using stem cells in general contradicts President Trump's pro-life stance and that of Supreme Court Nominee Amy Coney Barrett.

But the bottom line, were human embryonic stem cells used in Regeneron's antibody cocktail to treat the president? News 8 can verify the answer is no.

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VERIFY: Was the antibody cocktail used to treat President Trump developed using human embryonic stem cells? - CBS News 8