Axovant Releases Positive Interim Updates for Parkinson's and Tay-Sachs Gene Therapy Trials

Biospace.com | March 11, 2019

Axovant has been busy, releasing interim results from two different clinical trials, one for Tay-Sachs, the other for Parkinson’s disease. For Parkinson’s disease, Axovant released positive interim results from the first cohort of its SUNRISE-PD Phase II clinical trial of AXO-Lenti-PD. AXO-Lenti-PD is a gene therapy that delivers three genes, tyrosine hydroxylase, cyclohydrolase 1, and aromatic L-amino acid decarboxylase, by way of a single lentiviral vector. These genes code for a group of enzymes required for dopamine synthesis. The cohort involved two patients with advanced Parkinson’s disease. They both received a single administration of the lowest dose of the therapy. It was well-tolerated, and no serious adverse events were observed. At three months after receiving the therapy, improvements of an average of 55 percent from baseline were observed, based on the Unified Parkinson’s Disease Rating Scale (UPDRS) Total OFF score. “Our focus in this first cohort of the SUNRISE-PD study was on the safety and tolerability of AXO-Lenti-PD, as well as the evaluation of efficacy using well-validated, objective measures,” stated Gavin Corcoran, Axovant’s executive vice president of Research and Development. “These early data support the safety of the lowest dose of AXO-Lenti-PD, similar to what was observed with the earlier generation construct, ProSavin, and also suggest substantially greater biological activity than the highest dose of ProSavin previously tested. These findings are highly encouraging, and we look forward to advancing to higher dose cohorts where we will explore the full clinical potential of AXO-Lenti-PD in patients with Parkinson’s disease.”

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BeiGene Announces Strategic Alliance with Ontada to Improve U.S. Community Oncology Care

BeiGene | August 16, 2022

BeiGene a global biotechnology company that is developing and commercializing innovative and affordable oncology medicines to improve treatment outcomes and access for far more patients worldwide, today announced a strategic agreement with Ontada®, a McKesson business with leading provider technology and actionable real-world research, education, and evidence in oncology, to improve U.S. community oncology care through the development of real-world evidence data, tools, and insights to help increase access to affordable, cutting-edge therapies. The partnership will be led by Ontada and focus on accelerating the development and implementation of RWE to improve community education on the financial impact and value of oncolytics as well as improve patient access to oncology medicines. “BeiGene is excited to work with Ontada to advance our shared vision of improving patient access, affordability, and value for community oncology stakeholders and key partners working at the intersection of clinical practice and real-world evidence. Working together, BeiGene and Ontada will generate novel oncology insights through real-world data, tools, and technologies that will facilitate the acceleration of precise decision-making in support of community oncology practices whose ultimate mission is to improve patient outcomes.” Josh Neiman, Chief Commercial Officer, North America and Europe, at BeiGene As part of the collaboration, the companies will optimize the use of RWE to communicate value across key stakeholders including patients, providers, and payers; develop timely education on emerging science and therapeutic differentiation; and collaborate to advance use of RWE in healthcare and regulatory decision making to increase timely and affordable patient access to life-saving oncology therapies. “BeiGene aims to bring 10 molecules per year to the clinic starting in 2023 and, as this research advances, we’re delighted to form this strategic partnership with McKesson, which shares the same vision as BeiGene for improving the care of cancer patients,” said Christiane Langer, M.D., Senior Vice President of Global Medical Affairs, Ex-China, at BeiGene. “This collaboration will leverage Ontada’s technology platform and deep understanding of the experiences of patients treated in community oncology clinics to help accelerate both the development of the BeiGene portfolio and expand access to treatment options.” “As a leader in real-world research, Ontada is uniquely positioned to support complex research that uncovers deeper patient insights and perspectives that advance cancer care,” said Susan Shiff, Ph.D., president of Ontada. “We are excited to announce this unique partnership with BeiGene and look forward to developing a stronger understanding of how to better treat oncology patients through the use of real-world data and evidence.” About Ontada® Ontada is an oncology technology and insights business dedicated to transforming the fight against cancer. Part of McKesson Corporation, Ontada was founded on the core belief that precise insights – delivered exactly at the point of need – can save more patients’ lives. We connect the full patient journey by combining technologies used by The US Oncology Network and other community oncology providers with real-world data and research relied on by all top 15 global life sciences companies. Our work helps accelerate innovation and power the future of cancer care. About McKesson Corporation McKesson Corporation is a diversified healthcare services leader dedicated to advancing health outcomes for patients everywhere. Our teams partner with biopharma companies, care providers, pharmacies, manufacturers, governments, and others to deliver insights, products and services to help make quality care more accessible and affordable. Learn more about how McKesson is impacting virtually every aspect of healthcare at McKesson.com and read Our Stories. BeiGene Oncology BeiGene is committed to advancing best- and first-in-class clinical candidates internally or with like-minded partners to develop impactful and affordable medicines for patients across the globe. We have a growing R&D and medical affairs team of approximately 2,900 colleagues dedicated to advancing more than 100 clinical trials that have involved more than 16,000 subjects. Our expansive portfolio is directed predominantly by our internal colleagues supporting clinical trials in more than 45 countries and regions. Hematology-oncology and solid tumor targeted therapies and immuno-oncology are key focus areas for the Company, with both mono- and combination therapies prioritized in our research and development. BeiGene currently has three approved medicines discovered and developed in our own labs: BTK inhibitor BRUKINSA® in the U.S., China, the European Union, Great Britain, Canada, Australia, and additional international markets; and the non-Fc-gamma receptor binding anti-PD-1 antibody, tislelizumab, as well as the PARP inhibitor, pamiparib, in China. BeiGene also partners with innovative companies who share our goal of developing therapies to address global health needs. We commercialize a range of oncology medicines in China licensed from Amgen, Bristol Myers Squibb, EUSA Pharma and Bio-Thera. We also plan to address greater areas of unmet need globally through our other collaborations including with Mirati Therapeutics, Seagen, and Zymeworks. In January 2021, BeiGene and Novartis announced a collaboration granting Novartis rights to co-develop, manufacture, and commercialize BeiGene’s anti-PD1 antibody, tislelizumab, in North America, Europe, and Japan. Building upon this productive collaboration, BeiGene and Novartis announced an option, collaboration, and license agreement in December 2021 for BeiGene’s TIGIT inhibitor, ociperlimab, that is in Phase 3 development. Novartis and BeiGene also entered into a strategic commercial agreement through which BeiGene will promote five approved Novartis Oncology products across designated regions of China. About BeiGene BeiGene is a global biotechnology company that is developing and commercializing innovative and affordable oncology medicines to improve treatment outcomes and access for far more patients worldwide. With a broad portfolio, we are expediting development of our diverse pipeline of novel therapeutics through our internal capabilities and collaborations. We are committed to radically improving access to medicines for far more patients who need them. Our growing global team of more than 8,500 colleagues spans five continents, with administrative offices in Beijing, China; Cambridge, U.S.; and Basel, Switzerland.

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CELL AND GENE THERAPY

CSL Plasma Reimagines Donor Experience With First Donations Completed on Innovative Plasma Collection Technology

CSL Plasma | August 08, 2022

CSL Plasma, the plasma collection business of global biotherapeutics leader CSL Behring, began a new era in plasma donations, collecting its first plasma donations using leading-edge plasma collection technology that is expected to significantly reduce average collection time – by approximately one-third – and improve the donor experience. The Rika Plasma Donation System, only available at CSL Plasma and developed by Terumo Blood and Cell Technologies, is designed to reduce collection time, and improve the donor and operator experience. When considering prior average CSL Plasma donation procedure times, this could represent about a 30% reduction in average donation time for donors. The new plasmapheresis system first launched today in the Aurora, Colorado, CSL Plasma location, as part of a phased rollout to CSL Plasma U.S. collection centers. Plasma collected at CSL Plasma facilities is used in the manufacturing of lifesaving plasma-derived therapies. "Today marks a threshold moment for plasma donors following years of dedication, innovation and collaboration – demonstrating how we are driven by our promise in delivering lifesaving and life-extending therapies to patients around the world. We look forward to the positive impact this new technology brings and are thankful for plasma donors who continue to do the amazing every day when they donate at CSL Plasma." CSL Chief Operating Officer Paul McKenzie CSL Plasma believes features of the new Rika system can enable the collection of more plasma, in shorter periods of time, supporting quality and safety, and, ultimately, better serving patients who rely on plasma-based therapies. One of the first plasma donors to experience the Rika system, Kelly Geiser is a resident of the Denver area. She has donated plasma since 2017, and is a Platinum level donor as part of CSL Plasma's donor loyalty program, iGive Rewards®. Geiser appreciated that she was able to complete the donation procedure with the new technology in under 35 minutes and in the fastest donation time she has experienced, while every plasma donor's experience will vary. Donating plasma is also personal for Geiser, who says that therapies made from plasma have helped her and her family, including a parent who recently underwent emergency surgery. "It feels good knowing I'm helping people and, who knows, I might've helped somebody I know," said Geiser. According to Terumo BCT, benefits of the Rika Plasma Donation System include Rika completes one plasma collection in 35 minutes or less on average. The proprietary design of the system's centrifuge maximizes the plasma yield per cycle. The Rika Plasma Donation System was designed to allow no more than 200 milliliters of blood cells outside the donor's body, resulting in a low extracorporeal volume, which may contribute to a more comfortable donor experience. With less than 200 milliliters of blood cells outside the donor's body at any given time, should the initial procedure not be completed, the donor can return and donate without being deferred. The device is designed with an advanced user interface to guide CSL Plasma front-line employees. Operators can quickly and confidently resolve alarms, thanks to guidance provided on the Rika system's touchscreen, which also alerts operators about situations that require attention and provides suggested actions to resolve the situation. The system allows for real-time data analytics to drive device maintenance and repairs. CSL Plasma is implementing the new devices at Colorado centers and then expanding to other U.S. locations, providing donors more opportunities to "do the amazing" and be rewarded for their generosity. "As a long-time leader in our industry, CSL Plasma looks forward to this new plasmapheresis technology to delight our plasma donors and attract new donors while supporting a better operating experience for our employees," said Walter Charles, CSL Plasma Senior Vice President and General Manager. "As we complete the implementation of new technology at U.S. centers, we continue to address the critical and ongoing need for human plasma to produce life-saving medicines for people with serious and rare diseases." About CSL Plasma CSL Plasma operates one of the world's largest and most sophisticated plasma collection networks, with more than 300 plasma collection centers in the U.S., Europe and China. Headquartered in Boca Raton, Florida, CSL Plasma is a subsidiary of CSL Behring, a global biotherapeutics company and a member of the CSL Group of companies. Plasma collected at CSL Plasma facilities is used by CSL Behring for the sole purpose of manufacturing lifesaving plasma-derived therapies for people in more than 100 countries. The parent company, CSL Limited headquartered in Melbourne, Australia, employs more than 25,000 people. About CSL Behring CSL Behring is a global biotherapeutics leader driven by our promise to save lives. Focused on serving patients' needs by using the latest technologies, we discover, develop and deliver innovative therapies for people living with conditions in the immunology, hematology, cardiovascular and metabolic, respiratory, and transplant therapeutic areas. We use three strategic scientific platforms of plasma fractionation, recombinant protein technology, and cell and gene therapy to support continued innovation and continually refine ways in which products can address unmet medical needs and help patients lead full lives.

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CELL AND GENE THERAPY

GenScript and Gladstone-UCSF Institute of Genomic Immunology Collaborate on Groundbreaking Non-Viral Cell Therapy Study

Genscript Biotech Corporation | September 23, 2022

GenScript USA Inc., the world's leading life-science research tools and services provider, is collaborating with researchers at the Gladstone-UCSF Institute of Genomic Immunology to advance the development of new, non-viral cell therapies that modify genomic sequences in the pursuit of more effective cell therapy products while limiting the cellular toxicity that is typically associated with previously available methods. A new study, published in Nature Biotechnology, details methods for achieving highly efficient non-viral knock-in using GenScript's GenExact™ single-strand DNA modified with Cas9-target sequences. This method achieved up to ˜40% knock-in efficiency in primary immune cell types. One powerful application of CRISPR/Cas genome editing technology involves the precise insertion of DNA sequences via the HDR pathway. Traditionally, researchers have relied on viral vectors to deliver DNA insertion templates used for gene therapy into cells. However, difficulties manufacturing large amounts of clinical-grade viral vectors have delayed getting cell therapies to patients. Additionally, viral vectors can insert genes at any location within the genome, leading to safety concerns. Previous research by the group at Gladstone and UCSF has shown that synthetic DNA templates can be delivered without the use of viral vectors, but high levels of double-stranded DNA can be toxic to cells, resulting in low efficiency. Efficiency can be improved using a modified version of the DNA templates that can bind to the Cas9 enzyme. However, additional work was required to improve the yield of successfully engineered cells and to make the process compatible with the manufacturing of future cell therapies. Those goals motivated the team's current study. In this study, the team tested modified GenExact ssDNA designed with Cas9-targeted sequences to determine if the gene-editing efficiency could be improved with lower toxicity. First, synthetic sgRNA was complexed with Cas9 protein to form a RNP complex. Then the RNP and ssDNA template were delivered into the cell via electroporation, enabling efficient gene editing. The team discovered that this combination of GenExact ssDNA with Cas9-targeted sequence offers up to 40% knock-in editing efficiency with minimal cellular toxicity. This approach can accelerate the development and manufacture of novel, high-yield non-viral gene therapies — and at lower cost. "We are very proud to have collaborated on this groundbreaking scale-up work by the talented team at Gladstone and UCSF. GenScript is excited for the opportunities that this high-yield cell-line engineering process will provide to our customers," said Ray Chen, Ph.D, president of GenScript Life Science Group. "We were very happy to partner with Genscript on critical experiments demonstrating high efficiency and yield of CAR knock-in cells at clinical scale. The long ssDNA produced by Genscript exceeded our expectations and helped us clearly demonstrate the potential for future therapeutic applications using these methods.", Brian Shy, MD, PhD, a former clinical fellow in the lab of Alex Marson, MD, PhD, director of the Gladstone-UCSF Institute of Genomic Immunology About GenScript Biotech Corporation GenScript Biotech Corporation is a global biotechnology group. Based on its leading gene synthesis technology, GenScript has developed four major platforms including the global cell therapy platform, the biologics contract development and manufacturing organization platform, the contract research organization platform, and the industrial synthesis product platform. GenScript was founded in New Jersey, USA in 2002 and listed on the Hong Kong Stock Exchange in 2015. GenScript's business operation spans over 100 countries and regions worldwide, with legal entities located in the USA, mainland China, Hong Kong, Japan, Singapore, the Netherlands, and Ireland. GenScript has provided premium, convenient, and reliable products and services for over 100,000 customers.

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