The full rights to Taysha Gene Therapies Inc.’s lead gene therapy candidate are coming back to the company. In 2022, Astellas Pharma Inc. invested $50 million in Taysha for 15% of the company and the exclusive option to license TSHA-102 for treating the rare disease Rett syndrome. That option has expired, according to Taysha.

The U.S. FDA named the first nine recipients of the recently unveiled commissioner’s national priority voucher (CNPV) program aimed at addressing unmet public health needs by shortening regulatory review times to as little as one to two months. For one of those firms, Disc Medicine Inc., which submitted an NDA for bitopertin for rare genetic disorder erythropoietic protoporphyria in September, that could mean a potential approval before the end of 2025.

While recent advances in gene therapy have offered unprecedented options for patients with hemophilia, new data presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, revealed persistent concerns regarding the durability of these treatments and their potential liver toxicity.

A yearslong bipartisan effort to end the patent-eligibility chaos the U.S. Supreme Court created more than a decade ago could finally come to fruition with the current Congress.

Lexeo Therapeutics Inc. feels like it’s in a faster lane to a BLA for its Friedreich ataxia cardiomyopathy gene therapy after talking with the U.S. FDA. The agency told Lexeo that LX-2006 could be on the accelerated approval path if there is a mingling of the company’s data and studies.

Newco Trogenix Ltd. has emerged from incubation and raised £70 million (US$94.1 million) in a series A, as it prepares the ground for a U.S/U.K. clinical trial of a novel gene therapy construct in glioblastoma multiforme that is due to start at the beginning of 2026.

In a deal that could bring more than $2.1 billion in payments to Arbor Biotechnologies Inc., 90-year-old Chiesi Group gained exclusive and global rights to develop and commercialize ABO-101 for primary hyperoxaluria type 1, an ultra-rare disease caused by a mutation in the AGXT gene, as well as an option to go after a limited number of additional targets.

Opus Genetics Inc. will be sitting down with the U.S. FDA to talk about positive three-month data from the pediatric cohort of its ongoing phase I/II trial called OPGx-LCA5-1001 – partially funded by the agency – evaluating OPGx-LCA5, a gene augmentation therapy for ultra-rare Leber congenital amaurosis type 5 (LCA5). The affliction is a severe form of retinal dystrophy that renders babies blind in the first year of life.

The U.S. FDA released a trio of draft guidances to help sponsors in developing and monitoring cell and gene therapies, as well as other regenerative medicine therapies.

An ongoing concern for scientists is that there will be across-the-board funding cuts. This is already happening in mRNA research, where reductions affected coronavirus-related projects. During the pandemic, efforts focused on this pathogen, and once the health emergency was over, grants for antivirals were eliminated. However, these drugs could stem future outbreaks. Despite the cuts, recent research continues to demonstrate the potential of mRNA, not only for the development of antivirals, but also for obtaining more effective and longer-lasting vaccines.