gene therapy
-
A One-Time Treatment for NASH? Kriya Strikes Deal for Liver Disease Gene Therapy
Gene therapy biotech Kriya Therapeutics has acquired Tramontane Therapeutics, a startup focused on metabolic diseases. Tramontane brings a gene therapy that’s a potential one-time treatment for the fatty liver disease NASH.
-
FDA Approves BioMarin Pharma’s Gene Therapy, the First for Hemophilia A
The FDA approved Roctavian for treating hemophilia A. The regulatory decision makes the BioMarin Pharmaceutical product the first gene therapy for this inherited bleeding disorder.
-
Payer’s Place: Dawn Maroney
Dawn Maroney, President, Markets of Alignment Health and CEO of Alignment Health Plan, to discuss how they are using technology to provide better service and care to consumers.
-
FDA Approves First Gene Therapy for Duchenne Muscular Dystrophy
Sarepta Therapeutics’ Elevidys is now the first FDA-approved gene therapy for Duchenne muscular dystrophy. Elevidys is an engineered version of a gene intended to restore function lost to the mutation at the root of the inherited muscle disease.
-
Where Cell Therapies Are Going Next, and How Insurers Are Getting Ready
Multiple efforts are underway to improve cell therapies for cancer. During the MedCity News INVEST conference in Chicago, a panel discussed the future of cell therapies and what the industry doing to manage the financial risks of these new treatments.
-
BioPharma, Sponsored Post, Startups
The Biopharma Startups Presenting at MedCity INVEST
Companies presenting their technology at the conference seek to support a diverse patient population spanning conditions such as inflammatory bowel disease, food allergies, and cancer. Cell, gene therapy and immunotherapies also form some of their treatment approaches in development.
-
Biotech uniQure Cashes In on Hemophilia Gene Therapy With $400M Royalty Deal
By selling partial royalties to approved hemophilia gene therapy Hemgenix, uniQure gains immediate cash to support its pipeline, including a Huntington’s disease gene therapy already in the clinic . The royalty deal extends uniQure’s cash runway into 2026.
-
Sponsored Post, BioPharma, Payers
What Do Payers Need to Help Value-Based Contracting Deals for Novel Therapies Succeed?
A panel discussion at the annual Abarca Forward conference in Puerto Rico last month sought to identify some of the innovative financial approaches that can be applied to managing the high price of novel therapies. Conversations also addressed what payers need for these financing models to be sustainable.
-
Cell Therapy Pioneers Team Up to Found Viral Vector CDMO Backed by $64M
VintaBio manufactures AAV and lentiviral vectors for entities pursuing clinical development of cell and gene therapies. The contract development manufacturing organization’s launch comes as demand for viral vectors continues to outpace the supply of these crucial components of the therapies.
-
New Hemophilia Data Lead FDA to Delay Decision for BioMarin Gene Therapy
BioMarin Pharmaceutical’s submission of additional data for patients treated with its hemophilia A gene therapy, Roctavian, mean that the FDA will push out a regulatory decision to early summer. But it could be worth the wait as analysts anticipate FDA approval and project Roctavian becoming one of BioMarin’s biggest products.
-
Applying Remote Patient Monitoring to Surgery Prep and Recovery, Oncology and Women’s Health
Join us to learn about the latest trends in remote monitoring and how to extend its benefits beyond chronic conditions to more patients – all while using fewer staff resources.
-
AbbVie Pays Capsida $70M to Expand Gene Therapy Alliance to Eye Diseases
AbbVie and Capsida Therapeutics are expanding their gene therapy R&D alliance to the eyes. Capsida is in line to receive $70 million now and up to $595 million later, depending on the progress of the eye programs.
-
What Will Value-Based Drug Pricing Look Like By 2030? Experts Share 4 Predictions
Panelists at a recent pharmacy conference were optimistic about the future of value-based drug contracting. For example, one said that healthcare will be close to establishing a value-based formulary by 2030, and another said that longitudinal patient tracking will become a more important part the drug pricing process.
-
FDA Asks Gene Therapy Biotech to Run Clinical Trial That Might Be Impossible
Taysha Gene Therapies has encouraging data from an open-label, Phase 1/2 study in the rare disease giant axonal neuropathy. It might not be enough. The FDA recommended the biotech conduct a randomized and placebo-controlled study—a challenge in any ultra-rare disease.
-
CDMO Startup Vector BioMed Launches to Fill Lentiviral Gap for Cell & Gene Therapies
Vector BioMed has developed a technology platform for designing and manufacturing the lentiviruses used to deliver cell and gene therapies. The startup is launching as the field continues to experience shortages of these viral vectors.
-
Astellas Plots Near-Term Moves to Execute Long-Term Vision in Gene Therapy
Astellas has become an active player in gene therapy. MedCity News caught up with company executive Richard Wilson, who explained Astellas’s strategy, the clinical holds on its two most advanced programs, and the rationale behind its recent gene therapy deals.
-
Neurocrine Commits $175M to Voyager in New Neuro Gene Therapy Alliance
Neurocrine Biosciences is paying $175 million up front for rights to a preclinical Voyager Therapeutics gene therapy that could put the biotech in competition with Eli Lilly among other companies. Voyager could earn up to $4.2 billion in milestone payments.
Promoted
Payer’s Place: Dr. Anil Singh
Dr. Anil Singh shares his insights into the strategies employed by the organization to identify the most effective digital solutions for their members.
Promoted
Discover the Next-Gen Platform for Integrated Collaborative Care
Beyond EHRs and digital front doors, reducing the gaps in patient care journeys.
