Patients with Leber hereditary optic neuropathy who received bilateral injections of Gensight Biologics SA’s Lumevoq (lenadogene nolparvovec) are continuing to see statistically significant visual improvements three years into the phase III REFLECT trial, but the missed primary endpoint at 1.5 years, along with a series of manufacturing mishaps, have left the gene therapy’s approval prospects uncertain.
Abeona Therapeutics Inc. has announced three investigational preclinical gene therapy product candidates from its ophthalmology program. The new AAV-based therapies use novel AAV capsids from Abeona's in-licensed AIM capsid library. Abeona intends to submit its first pre-IND application meeting request this month.
The U.S. FDA marked the 40th anniversary of the Orphan Drug Act with Rare Disease Day 2023 as Robert Califf, the agency’s commissioner of food and drugs, opened the day by expressing his wonder and accompanying concern regarding gene editing and gene therapy.
Genascence Corp. has been awarded US$11.6 million over 4 years from the California Institute for Regenerative Medicine (CIRM) to help advance the company's GNSC-001 gene therapy for knee osteoarthritis (OA). The funding will support a phase Ib trial and manufacturing activities.
Otoferlin is a calcium sensor protein critical for the transmission of the signal from inner hair cells (IHCs) to the spiral ganglion neurons (SGNs), and it is encoded by the OTOF gene. Pathogenic biallelic loss of function variations in OTOF result in failure of synaptic transmission, causing autosomal recessive deafness 9 (DFNB9), which is a congenital severe-to-profound auditory neuropathy.
A trio of proposed Medicare drug payment models that made a Feb. 14 debut in the U.S. is playing to mixed reviews. Two of the models to be tested by the U.S. Centers for Medicare & Medicaid Services (CMS) Innovation Center seem to “address the real problems underlying prescription drug pricing – patient out-of-pocket expenses and better payment systems that reward the value a medicine brings to the patient and the overall health care system,” said John Murphy, chief policy officer for the Biotechnology Innovation Organization. But he called the third model, which is expected to restrict Medicare payment for some Part B drugs that have indications with accelerated approval, “an attack on the accelerated approval pathway,” which Congress mandated to spur investment and innovation in areas of unmet medical need.
A phase IIb clinical study has Frequency Therapeutics Inc. reeling and making big changes, including layoffs. The placebo-controlled phase IIb study of FX-322 in treating acquired sensorineural hearing loss by regenerating hair cells in the cochlea missed its primary efficacy endpoint, so the company is discontinuing the program. It’s also shutting down a phase Ib study of FX-345, a program for treating the same indication.
Taysha Gene Therapies Inc.’s stock (NASDAQ:TSHA) struggled Feb. 1 following the U.S. FDA’s recommendation that the company dose more patients in a double-blind, placebo-controlled study of TSHA-120 for the ultra-rare indication giant axonal neuropathy.
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