A patient death in a clinical trial usually prompts the FDA to impose a clinical hold. But Neurogene is able to avoid a lengthy clinical trial pause largely because of its participation in an FDA pilot program intended to speed up the development of therapies for rare diseases.
The PTC Therapeutics gene therapy, Kebilidi, treats an enzyme deficiency that affects the body’s ability to produce dopamine, a neurotransmitter important for motor control. FDA approval for the product comes two years after it won its first regulatory approvals in Europe.
Closing cancer health equity gaps require medical breakthroughs made possible by new funding approaches.
Neurogene’s Rett syndrome gene therapy has preliminary data supporting safety and efficacy of the one-time treatment. But a late-breaking report of a serious complication in a patient who received the high dose sent shares of the biotech downward.
The death wasn’t due to Beam Therapeutics’ sickle cell disease therapy, BEAM-101, but was instead attributed to the preconditioning treatment needed before the genetic medicine is infused. A different Beam program in preclinical development could avoid the need for toxic preconditioning; data from both programs will be presented during the upcoming American Society of Hematology annual meeting.
In addition to meeting the main safety and tolerability goal, MieraGTx Holdings’ gene therapy for Parkinson's disease also showed signs of efficacy. The one-time treatment is intended to reprogram dysfunctional brain circuits.
Astellas secured an exclusive option to license global rights to an AviadoBio gene therapy in development as a one-time treatment for a certain type of frontotemporal dementia. A Phase 1/2 test of the AviadoBio gene therapy is ongoing.
Hear executives from Quantum Health, Surescripts, EY, Clinical Architecture and Personify Health share their views on digital transformation in healthcare.
Pfizer’s hemophilia A gene therapy reduced annualized bleeding rates in a Phase 3 clinical trial, setting the stage for discussions with regulators. However, the market for such one-time treatments is uncertain, as uptake of commercialized hemophilia gene therapies remains slow.
A uniQure gene therapy for Huntington’s disease has interim clinical data showing an 80% slowing of disease progression. An accelerated approval pathway is one of the topics the company wants to discuss with the FDA.
FDA reviewers concluded the data for Sarepta Therapeutics’ Elevidys were insufficient to show efficacy in the rare muscle disorder Duchenne muscular dystrophy. Peter Marks, the agency’s top biologics official, reviewed the same data and reached a different conclusion.
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 progress in artificial intelligence (AI) is reshaping patient care, across various dimensions, from facilitating faster discharge to curating treatment plans and suggesting lifestyle changes.
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 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.