Another data cut from Ultragenyx Pharmaceutical Inc. regarding UX-111 (rebisufligene etisparvovec), an AAV9 gene therapy for type A Sanfilippo syndrome, continued to brighten the picture for the compound, recently taken under review again by the U.S. FDA.
DFNB16, a recessive form of mild-to-moderate human deafness, is caused by mutations in the STRC gene, which encodes stereocilin, a protein essential for the effective function of outer hair cells as cochlear amplifiers. Researchers from the Institut Pasteur and Sorbonne Université in Paris, France, have developed and evaluated the efficacy of an Strc gene therapy in a mouse model of DFNB16.
Seamless Therapeutics has received big pharma endorsement of its proprietary recombinase gene editing platform, sealing a potential $1.12 billion deal with Eli Lilly and Co. to apply the technology in hearing loss.
Siren Biotechnology Inc. has obtained IND approval from the FDA enabling the initiation of its first-in-human trial for its lead investigational program SRN-101 in adult patients with recurrent high-grade glioma.
With the PDUFA date fast approaching for Regenxbio Inc.’s gene therapy RGX-121, the U.S. FDA placed the drug on clinical hold along with another, RGX-111, after preliminary analysis of a single case of neoplasm (specifically, an intraventricular central nervous system tumor) in a participant treated in the phase I/II study with the latter treatment.
Sensorion SA has announced a €60 million (US$72 million) financing, with a €20 million (US$24 million) strategic investment from Sanofi SA, as it advances its pipeline of therapies to restore, treat and prevent hearing loss disorders.
Following a clinical hold last October of Intellia Therapeutics Inc.’s Magnitude and Magnitude-2 phase III trials of CRISPR/Cas9 gene editing therapy nexiguran ziclumeran (nex-z) to treat transthyretin amyloidosis with cardiomyopathy (ATTR-CM) and polyneuropathy (ATTR-PN), respectively, the U.S. FDA lifted the hold on Magnitude-2, pushing the company’s shares up by 22% in early trading Jan. 27.
After a roller coaster of a year for Duchenne muscular dystrophy (DMD) gene therapy Elevidys (delandistrogene moxeparvovec), Sarepta Therapeutics Inc. looks to focus on the efficacy narrative in 2026, starting with newly unveiled three-year data showing durable efficacy across all key motor function assessments for treated DMD patients vs. external controls.
Scientists from the Smidt Heart Institute, Cedars-Sinai Medical Center (Los Angeles, CA, USA) recently explored adeno-associated virus (AAV) vectors carrying the T-box transcription factor 18 (TBX18) transgene to produce a physiologically responsive biological pacemaker activity for the treatment of bradycardia.
Adolore Biotherapeutics Inc. has announced that the FDA has granted orphan drug designation to the company’s Kv7-activating rdHSV-CA8* gene therapy for treatment of primary and secondary erythromelalgia.
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