First Gene Therapy Clinical Trial to Address Genetic Blindness

PharmaNewsIntelligence | March 11, 2020

Oregon Health & Science University (OHSU) held the first-ever gene therapy clinical trial, BRILLIANCE, to address blindness-causing gene mutation, LCA10, according to a recent press release The clinical trial, sponsored by Allergan plc and Editas Medicine, is one of 14 different clinical trials investigating new genetic treatments for ophthalmic conditions and nearly 50 vision-related clinical trials. The procedure uses gene-editing tool CRISPR to edit human genes, also known as in vivo gene editing. Previously, gene-editing methods have edited genetic material after it was removed from the human body. Being able to edit genes inside the human body is incredibly profound,” said Mark Pennesi, MD, PhD, the Kenneth C. Swan, associate professor of ophthalmology in the OHDU School of Medicine and chief of the OSHU Casey Eye Institute’s Paul H. Casey Ophthalmic Genetics Division.

Spotlight

Finite Element Analysis (FEA) has been widely used to analyze the in vivo structural response of implantable medical devices and assess device performance, reliability and durability. In order to obtain a robust design, parameter variations and their influence on device performance need to be understood. Probabilistic analysis techniques like Monte Carlo sampling combined with FEA clarify these stochastic processes and in determining probability of failure and design reliability.

Spotlight

Finite Element Analysis (FEA) has been widely used to analyze the in vivo structural response of implantable medical devices and assess device performance, reliability and durability. In order to obtain a robust design, parameter variations and their influence on device performance need to be understood. Probabilistic analysis techniques like Monte Carlo sampling combined with FEA clarify these stochastic processes and in determining probability of failure and design reliability.

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BUSINESS INSIGHTS

Astellas and Sutro Biopharma Announce Worldwide Strategic Collaboration to Advance Novel Immunostimulatory Antibody-Drug Conjugates (iADCs)

Sutro Biopharma, Inc.; Astellas Pharma Inc. | June 28, 2022

Astellas Pharma Inc. and Sutro Biopharma, Inc. announced a worldwide, strategic collaboration and licensing agreement focused on the discovery and development of novel immunostimulatory antibody-drug conjugates. The collaboration leverages the unique cancer-fighting potential of iADCs as a novel modality, enabled by Sutro’s ability to engineer complex conjugated antibodies, and Astellas’ global oncology R&D expertise. “We are delighted to work with Astellas, a premier biopharmaceutical company with substantial expertise in immuno-oncology, on this novel modality. iADCs hold promise well beyond the existing success of ADCs. Sutro’s unique conjugation technology enables dual conjugations that site-specifically incorporate a potent cytotoxin that can directly kill tumor cells together with an immunostimulatory component that has the potential to locally prime an immune response to the patient’s particular tumor cells. We look forward to collaboratively exploring the potential of this approach to treat cold tumors and bring new drug therapies to patients who do not respond to existing immunotherapies.” William J. Newell, Sutro’s Chief Executive Officer Naoki Okamura, Chief Strategy Officer, at Astellas commented, "Astellas considers Immuno-Oncology as one of the Primary Focuses of its R&D strategy; our goal is to bring effective drugs to patients who do not respond to existing immune checkpoint inhibitors. Sutro is a leading company in the area of iADCs, a new modality, and has its own original iADC technologies. The strategic partnership with Sutro will help us expand our pipeline and widen the choice of cancer immunotherapies.” A key challenge with cancer immunotherapies, including immune checkpoint inhibitors, is the tumor microenvironment*1 within cold tumors creating barriers to immune cell infiltration and thus preventing the cure. The immune checkpoint inhibitors approved to date are efficacious as a monotherapy in only about 20% of cancer types, which vary widely*1. This strategic partnership will engage in the development of iADCs, a next generation modality with the potential for effective and efficient approaches for treatment of cold tumors so as to bring new drug therapies to patients who do not respond to existing therapies. An iADC, which combines an antibody with a small molecule compound that induces immunogenic cell death*2 in addition to an immune activating molecule, has the potential to boost the anti-cancer action. This partnership will enable Astellas and Sutro to mutually leverage strengths in their respective fields to accelerate iADC development for three distinct biological targets; Sutro will engage in research and preclinical studies to identify candidate compounds and then Astellas will pursue clinical development. Sutro has advanced technologies for linking drugs to antibodies and proprietary component parts, including candidate antibodies and linkable cytotoxins and immunostimulatory molecules. For development of iADCs, Astellas will utilize the strength of its global R&D and commercialization capabilities in the area of antibodies and the small molecular components. These iADCs may have the potential to provide new therapeutic options for treatment of cancers for which no broadly effective therapy is currently available. Under the terms of the agreement, Sutro will receive an upfront cash payment of US$90 million to develop iADCs for three biological targets and may be eligible to receive up to US$422.5 million in development, regulatory and commercial milestones for each product candidate, and tiered royalties ranging from low double-digit to mid-teens on worldwide sales of any commercial products that may result from the collaboration, subject to Sutro’s cost and profit sharing option for the United States. Sutro has the option to share in the costs and profits for developing and commercializing product candidates in the United States. If Sutro exercises this option for a particular product candidate, Astellas and Sutro will equally share the costs of such co-development and co-commercialization, with the resulting profits/losses from co-commercialization also shared equally in the United States.

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BUSINESS INSIGHTS

Amylyx Pharmaceuticals Announces ALBRIOZA™ is Now Available in Canada for the Treatment of ALS

Amylyx Pharmaceuticals, Inc | August 01, 2022

Amylyx Pharmaceuticals, Inc. announced that ALBRIOZA™ is now commercially available in Canada for people living with amyotrophic lateral sclerosis. ALBRIOZA is an oral fixed-dose combination therapy that may reduce neuronal cell death as a stand-alone therapy or when added to existing treatments. In a clinical trial, ALBRIOZA significantly slowed disease progression and loss of functional decline in people living with ALS. “We have remained steadfast in our commitment to ensure ALBRIOZA would be available in Canada as quickly as possible following Health Canada’s decision to approve ALBRIOZA, with conditions, in June,” said Margaret Olinger, Chief Commercial Officer at Amylyx. “We are pleased that starting this week, we are ready to fill prescriptions for ALBRIOZA.” Amylyx filed reimbursement submissions to the Canadian Agency for Drugs and Technologies and Institut national d’excellence en santé et en services sociaux in Quebec as part of an aligned Health Technology Assessments (HTA) review with Health Canada in parallel with the regulatory process to reduce the timelines required for patient access. The Company is continuing to work with the pan-Canadian Pharmaceutical Alliance, Federal, Provincial, and Territorial public drug plans, and private insurers to expeditiously achieve listing and reimbursement, so that eligible Canadians living with ALS can have access to ALBRIOZA as quickly and efficiently as possible. “In Canada, prescription drugs undergo a thorough and often long review and approval process across regulatory, HTA, pricing negotiations, and formulary listing with public drug plans. In addition, private insurers have their own review and listing processes. We continue to work diligently to navigate reimbursement processes to pursue broad and equitable access to ALBRIOZA across all provinces in Canada.” Chris Aiello, General Manager and Head of Canada at Amylyx “Market availability is an important milestone in the available treatment options for people living with ALS,” said Tammy Moore, CEO of the ALS Society of Canada. “As we look forward, we do not want to see coverage decisions become a barrier to access, and we urge the decision-makers throughout the drug access and reimbursement process to work expeditiously to provide equitable access for all eligible Canadians living with ALS who are in desperate need.” As part of Amylyx’ continued commitment to the ALS community, through the Amylyx Care Team Support Program, Amylyx provides insurance navigation, treatment coordination and educational support for people living with ALS who have been prescribed ALBRIOZA in Canada and their caregivers. Eligibility and enrollment into the ACT Support Program can be discussed with prescribing health care professionals. About ALBRIOZA™ ALBRIOZA™ is an oral fixed-dose medication approved with conditions to treat amyotrophic lateral sclerosis (ALS) in Canada and with marketing applications pending in the United States and European Union. The combination of sodium phenylbutyrate and taurursodiol may reduce neuronal cell death, hypothesized to occur by simultaneously mitigating endoplasmic reticulum stress and mitochondrial dysfunction. ALBRIOZA is also being explored for the potential treatment of other neurodegenerative diseases. About Amylyx Pharmaceuticals Amylyx Pharmaceuticals, Inc. is committed to supporting and creating more moments for the neurodegenerative community through the discovery and development of innovative new treatments. Amylyx is headquartered in Cambridge, Massachusetts and has operations in Canada and EMEA.

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PHARMACY MARKET

GluCare.Health Partners with Novo Nordisk for Innovative Weight Management Patient Program

Glucare.Health and NovoNordisk: | June 09, 2022

GluCare.Health, one of the Middle East's leading health tech startups, has partnered with global pharmaceutical company Novo Nordisk, market leader in Diabetes & Obesity, to introduce a blueprint for the future of healthcare. The partnership will see a 150-patient weight management pilot program that will combine new digital bio-markers, using wearable technology and devices for continuous data monitoring, with existing therapeutics. It introduces the Middle East's first value-based reimbursement model, and will be the first relationship of its kind for Novo Nordisk globally. This innovative partnership will put accountability for outcomes vs. costs on the provider, rather than on the insurers or patients. The 6-month program, which begins in September, is open to people with obesity who meet certain criteria. Patients will receive an unparalleled level of in-patient and data-driven remote care, with a multidisciplinary clinic team having continuous access to all patient data through an intelligent analytical platform. If patients do not lose at least 10% of their body weight by the end of the program, GluCare.Health and Novo Nordisk will cover all or a portion of the cost under the value-based reimbursement pilot. GluCare.Health is also committed to transparency when it comes to reporting results of the program. With one in three adults affected by obesity in the UAE[1], GluCare.Health's Co-Founder and Managing Director, Dr. Ihsan AlMarzooqi, says that the blueprint for care has the potential to not only save many lives but the UAE economy billions of dollars in costs and lost productivity. "When patients with chronic conditions are not supported properly, it can have a devastating impact on those individuals and their families. Additionally, the cost of treating the health consequences of obesity to the UAE economy alone is approximately $3.6 billion annually[2]. Traditional models of care remain woefully limited when it comes to outcomes. This partnership allows us to embed Novo Nordisk's advanced therapeutics into a companion platform like GluCare.Health, which enhances its efficacy dramatically." About Glucare.Health The first hybrid Digital Therapeutics (DTx)+Human vertically integrated diabetes platform of its kind in the world, GluCare.Health is reinventing metabolic care and transforming lives. Conceptualized in the UAE and opening the doors to its state-of-the-art 10,000 sq foot Dubai clinic in September 2020, it is the world's first healthcare provider to empower both clinicians and patients through Remote Continuous Data Monitoring as part of its standard model of care, an innovative and highly personalized 'continuous healthcare' approach that provides a comprehensive and real-time view of patients. As part of its unique model, GluCare is also an early adopter of - and the region's first clinic to use - Digital Therapeutics (DTx), combining it with wearable and smart technology, data analytics, unique in-clinic workflows, and a caring and connected expert care team. Applying technology as a humanizing force, GluCare.Health's team partners with patients and their families, giving them the tools, knowledge, and continuous support to live longer, healthier, and more fulfilling lives. About NovoNordisk Novo Nordisk is a leading global healthcare company, founded in 1923 and headquartered in Denmark. Our purpose is to drive change to defeat diabetes and other serious chronic diseases such as obesity and rare blood and endocrine disorders. We do so by pioneering scientific breakthroughs, expanding access to our medicines, and working to prevent and ultimately cure disease. Novo Nordisk employs about 49,300 people in 80 countries and markets its products in around 170 countries. For more information, visit Novo Nordisk UAE.

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