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.
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.
At the Payer Insights sessions on Day 1 of ViVE 2024, a panel on prior authorization offered compelling insights from speakers who shared the positive developments in this area after years of mounting frustration. Speakers also shared challenges as they work with providers to figure out how policy developments and technology will work in practice.
The preliminary results from three clinical trials administering gene therapy to address deafness from otoferlin mutations mark inspiring and incredible progress in hearing healthcare.
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.
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 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.
Canada has a proud history of achievement in the areas of science and technology, and the field of biomanufacturing and life sciences is no exception.
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 biggest trend in gene therapy development is the potential of several treatments for rare neurological diseases to move from the bench to the bedside.
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 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.
This eBook, in collaboration with Care Logistics, details how hospitals and health systems can facilitate more effective decision-making by operationalizing elevated awareness.
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 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.
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.
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.
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.