A Pfizer gene therapy for Duchenne muscular dystrophy failed its Phase 3 clinical trial. But analysts draw distinctions between that therapy and Sarepta Therapeutics’ Elevidys, which awaits an FDA decision that could grant it full regulatory approval.

The post Pfizer & Sarepta Gene Therapies Both Failed Phase 3, But Analysts Expect Sarepta Will Win Approval appeared first on MedCity News.

Orchard Therapeutics gene therapy Lenmeldy won FDA approval for treating the rare enzyme deficiency metachromatic leukodystrophy. In January, Kyowa Kirin completed its acquisition of Orchard, which now operates as a subsidiary of the Japanese drugmaker.

The post Gene Therapy Is Now First FDA-Approved Treatment for Rare & Fatal CNS Disorder appeared first on MedCity News.

The preliminary results from three clinical trials administering gene therapy to address deafness from otoferlin mutations mark inspiring and incredible progress in hearing healthcare.

The post Overcoming Hearing Healthcare Barriers to Realize Gene Therapy Promise appeared first on MedCity News.

Fractyl Health will use the IPO proceeds for pivotal testing of a medical device and surgical procedure intended to address root causes of diabetes and obesity. The Fractyl pipeline also includes a gene therapy for these conditions.

The post Metabolic Disorder-Focused Fractyl Health ‘GUTS’ Out a $110 Million IPO appeared first on MedCity News.

Eli Lilly said its experimental gene therapy restored hearing in a boy born with profound hearing loss. The results for this experimental treatment come three months after Regeneron Pharmaceuticals reported early but encouraging data for its hearing loss gene therapy.

The post Eli Lilly Sounds Off as Early Data Show How Gene Therapy Can Restore Hearing appeared first on MedCity News.

The FDA approved Vertex Pharmaceuticals’ gene therapy Casgevy for treating beta thalassemia, an inherited blood disease that leads to low levels of functioning hemoglobin. Last month, the one-time treatment won its first FDA nod for treating sickle cell disease.

The post Vertex’s CRISPR Gene Therapy Lands Another FDA Nod in a Rare Blood Disease appeared first on MedCity News.

Novartis is paying Voyager Therapeutics $100 million up front to collaborate on gene therapies for Huntington’s disease and spinal muscular atrophy. The deal builds on a relationship the companies started in 2022.

The post Novartis Returns to Voyager Therapeutics to Reach New Gene Therapy Destinations appeared first on MedCity News.

The biggest trend in gene therapy development is the potential of several treatments for rare neurological diseases to move from the bench to the bedside.

The post Are Rare Brain Diseases the Next Commercial Frontier for Gene Therapy? appeared first on MedCity News.

It’s time to build the infrastructure needed to scale up inhaled gene therapies targeting cystic fibrosis, and to bolster investment that supports several key program components.

The post Gene Therapy and a New Medicine Delivery Approach Signal Progress for Cystic Fibrosis Patients appeared first on MedCity News.

The first gene therapies for sickle cell disease are now approved: Casgevy from Vertex Pharmaceuticals Casgevy and Lyfgenia from Bluebird Bio. Despite the same-day approval, key differences give one of these therapies commercialization advantages over the other.

The post FDA Approves Not One, But Two Sickle Cell Disease Gene Therapies appeared first on MedCity News.

Ajinomoto’s Forge Biologics acquisition brings contract development and manufacturing organization capabilities in gene therapy. Forge specializes in manufacturing AAV vectors used to deliver these therapies.

The post Ajinomoto Adds Gene Therapy to CDMO Scope via $554M Forge Bio Buyout appeared first on MedCity News.

The gene therapy programs of Lexeo Therapeutics span rare forms of cardiomyopathy and certain types of Alzheimer’s disease. But Lexeo’s IPO comes as a rival gene therapy developer sues the biotech, claiming its work employs stolen trade secrets.

The post Lexeo’s IPO Raises $100M for Gene Therapies That Treat the Heart, Protect the Brain appeared first on MedCity News.

BioMarin Pharmaceutical CEO Jean-Jacques Bienaimé will retire in December, when he will be succeeded by Genentech CEO Alexander Hardy. The transition comes with BioMarin in the early stages of the commercial launch of Roctavian, the first FDA-approved hemophilia A gene therapy.

The post Genentech Veteran Named New BioMarin CEO as Gene Therapy Launch Ramps Up appeared first on MedCity News.

AstraZeneca starts the Cellectis partnership with $105 million in upfront cash and an equity investment. The pharmaceutical giant will also cover research costs for up to 10 cell therapy programs in oncology, immunology, and rare diseases.

The post New AstraZeneca, Cellectis Alliance Spans Up to 10 Cell & Gene Therapies appeared first on MedCity News.

Sarepta Therapeutics’ Duchenne muscular dystrophy gene therapy fell short of its main Phase 3 goal, but the firm contends the full body of evidence supports expanding the therapy’s label to all patients who have the muscle-wasting disease. Analysts say prospects of the therapy, Elevidys, rest on FDA willingness to exercise flexibility it has already shown to rare disease drugmakers, including Sarepta.

The post Sarepta Gene Therapy Misses in Phase 3; Prospects Now Rely on FDA Flexibility appeared first on MedCity News.

Bayer opens its new cell therapy manufacturing facility as it prepares a Parkinson’s disease cell therapy for Phase 2 testing. The new 100,000 square foot facility also has space for manufacturing other cell therapies in the pharma giant’s pipeline.

The post Bayer’s New $250M Cell & Gene Therapy Site Signals More Investments to Come appeared first on MedCity News.

The Orchard Therapeutics acquisition comes as the biotech’s gene therapy for metachromatic leukodystrophy approaches a March 2024 FDA decision. The ex vivo gene therapy is already commercialized in Europe, marketed as Libmeldy.

The post Kyowa Kirin Buys Into Gene Therapy With Orchard Therapeutics Acquisition appeared first on MedCity News.

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.

The post A One-Time Treatment for NASH? Kriya Strikes Deal for Liver Disease Gene Therapy appeared first on MedCity News.

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.

The post FDA Approves BioMarin Pharma’s Gene Therapy, the First for Hemophilia A appeared first on MedCity News.

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.

The post FDA Approves First Gene Therapy for Duchenne Muscular Dystrophy appeared first on MedCity News.

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.

The post Where Cell Therapies Are Going Next, and How Insurers Are Getting Ready appeared first on MedCity News.

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.

The post The Biopharma Startups Presenting at MedCity INVEST appeared first on MedCity News.

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.

The post Biotech uniQure Cashes In on Hemophilia Gene Therapy With $400M Royalty Deal appeared first on MedCity News.

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.

The post What Do Payers Need to Help Value-Based Contracting Deals for Novel Therapies Succeed? appeared first on MedCity News.

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.

The post Cell Therapy Pioneers Team Up to Found Viral Vector CDMO Backed by $64M appeared first on MedCity News.

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.

The post New Hemophilia Data Lead FDA to Delay Decision for BioMarin Gene Therapy appeared first on MedCity News.

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.

The post AbbVie Pays Capsida $70M to Expand Gene Therapy Alliance to Eye Diseases appeared first on MedCity News.

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.

The post What Will Value-Based Drug Pricing Look Like By 2030? Experts Share 4 Predictions appeared first on MedCity News.

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.

The post FDA Asks Gene Therapy Biotech to Run Clinical Trial That Might Be Impossible appeared first on MedCity News.

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.

The post CDMO Startup Vector BioMed Launches to Fill Lentiviral Gap for Cell & Gene Therapies appeared first on MedCity News.