The Foundation for the National Institutes of Health (FNIH) has announced the online publication of the first playbook designed to help accelerate the development of adeno-associated virus (AAV) gene therapies for rare diseases.
Invitae Corp. has entered into a partnership with Bridgebio Pharma Inc. to advance genetics-based drug discovery for rare diseases. The goal of the collaboration is to generate new insights focused on genetic modifiers and the discovery of novel therapeutic targets for rare diseases and other unmet medical needs.
The U.S. FDA is promising to make 2024 a “breakout” 12 months for gene therapies, with a number of initiatives to promote clinical development, approvals and uptake. “This is a great year to focus on gene therapy,” said Peter Marks, director of the Center for Biologics Evaluation and Research at the FDA. “I just want to focus on moving ahead gene therapy,” he told attendees of the J. P. Morgan Healthcare Conference on Jan. 8.
Drug guidances are still pouring forth from the U.S. FDA as 2023 winds to an end. The latest batch deals with issues as varied as the reformulation of drug products that use carbomers manufactured with benzene, potency assurance for cellular and gene therapies, the quality of topical eye treatments, and the development of drugs and biologics for rare diseases.
Reneo Pharmaceuticals Inc. is halting development of its only asset, mavodelpar, after a phase IIb study missed its primary and secondary efficacy endpoints. The selective peroxisome proliferator-activated receptor (PPAR)-delta agonist was being developed to treat to rare genetic mitochondrial diseases.
The U.S. FDA has approved Novartis AG’s Fabhalta (iptacopan) as the first oral monotherapy for adults with paroxysmal nocturnal hemoglobinuria, a rare blood disease that impairs blood cell production. This is the only factor B inhibitor of the immune system’s complement pathway and is expected to be on the market before December ends. Fabhalta has plenty of competition from already-approved therapies and more treatments are in development.
Astrazeneca plc is making a $220 million equity investment and tossing in $25 million up front to Cellectis SA as part of a new collaboration agreement. The deal is part of Astrazeneca’s efforts, including a July licensing agreement worth about $1 billion with Pfizer Inc., to delve deeper into gene therapy for treating cancer and rare diseases.
In a show of bipartisan solidarity, members of the U.S. Senate Special Committee on Aging voiced their support Oct. 26 for a new regulatory pathway to quicken access to new drugs for rare diseases that have no approved treatments.
Japanese specialty global pharma Kyowa Kirin Co. Ltd. has agreed to buy out Orchard Therapeutics plc in a $387.4 million cash takeover that could jump $90 million to reach $477.6 million, contingent on the pending U.S. FDA approval of its EU-approved gene therapy, Libmeldy (atidarsagene autotemcel).
Japanese specialty global pharma Kyowa Kirin Co. Ltd. has agreed to buy out Orchard Therapeutics plc in a $387.4 million cash takeover that could jump $90 million to reach $477.6 million, contingent on the pending U.S. FDA approval of its EU-approved gene therapy, Libmeldy (atidarsagene autotemcel).
