At the recent ASGCT meeting, researchers from Exegenesis Bio Inc. presented preclinical data for EXG-102-031, a novel recombinant adeno-associated virus (rAAV)-gene therapy being developed for the treatment of neovascular age-related macular degeneration (AMD), also called wet AMD (wAMD).

Usher syndrome is the most common cause of deaf-blindness. Mutations in the protocadherin-15 (PCDH15) gene cause Usher syndrome type 1 F (USH1F), which makes up about 3-11% of all Usher syndrome cases. As a component of tip links of the inner ear hair cells, PCDH15 binds to cadherin-23 (CDH23) to convey force from sound stimuli to the mechanosensory transduction channels.

Facioscapulohumeral muscular dystrophy (FSHD) is a skeletal muscular dystrophy characterized by DNA hypomethylation of D4Z4 repeat units of a macrosatellite array found at the distal end of chromosome region 4q35, which causes a myotoxic expression of DUX4. Researchers from Epic-Bio presented the discovery of EPI-321, a novel gene therapy candidate for the treatment of FSHD.

Sarepta Therapeutics Inc. CEO Doug Ingram said the U.S. FDA has promised to schedule “expeditiously” an advisory committee meeting on the BLA related to SRP-9001 (delandistrogene moxeparvovec) for Duchenne muscular dystrophy (DMD).

The U.S. FDA has lifted the clinical hold on Astellas Pharma Inc.’s Fortis phase I/II trial evaluating AAV gene replacement therapy AT-845 in adults with late-onset Pompe disease.

The U.S. FDA has lifted the clinical hold on Astellas Pharma Inc.’s Fortis phase I/II trial evaluating adeno-associated virus (AAV) gene replacement therapy AT-845 in adults with late-onset Pompe disease.

After long years of painstaking work, the commercialization of cell and gene therapies picked up pace in 2022, with multiple approvals. More progress is expected in 2023, with several firsts in the offing and products for larger patient populations reaching the market.

It has been shown that site-specific phosphorylation of tau at threonine 205 (T205) by the kinase p38γ disengages tau from toxic pathways, thus acting as a neuroprotective function in Alzheimer’s disease. Researchers in Australia have designed a novel AAV-based gene therapy approach targeting p38γ, DBA/2.p38γCA, in murine models of epilepsy in which tau is known to play a neurotoxic effect.

Sonothera Inc. reeled in $60.75 million in a series A financing led by Arch Venture Partners. The funds are earmarked for the continued development of Sonothera’s ultrasound-guided, nonviral, gene therapy platform and treatments.