Spur Therapeutics Ltd., formerly Freeline Therapeutics, has announced new data from its GBA1 Parkinson’s disease research program. In a subset of Parkinson’s disease patients with mutations in the GBA1 gene, such mutations lead to a deficiency of the glucocerebrosidase (GCase) enzyme and the accumulation of harmful substrates.

CSL Behring’s expensive hemophilia B gene therapy is to be reimbursed by the U.K. National Health Service, after the company agreed to an outcomes-based payment scheme. The therapy, Hemgenix (etranacogene dezaparvovec), which has a U.K. list price of £2.6 million (US$3.3 million), was approved under a managed access scheme, in which data will be collected over five years to enable both the long-term effectiveness, and any adverse liver toxicity caused by the transgene, to be monitored.

Two days before the PDUFA date, the U.S. FDA handed down a complete response letter (CRL) for Rocket Pharmaceuticals Inc.’s Kresladi (marnetegragene autotemcel), delaying potential approval of the lentiviral-based gene therapy as the first therapeutic option for leukocyte adhesion deficiency type I, a rare, inherited immune disorder. But the Cranbury, N.J.-based company has suggested that delay won’t be long, as the CRL requests only “limited” chemistry manufacturing and controls (CMC) information – additional CMC data were also cited as the reason for the three-month review extension earlier this year.

Exsilio Therapeutics emerged from stealth mode on June 25, 2024, with $82 million from a series A financing that was co-led by Novartis Venture Fund and Delos Capital. The company plans to use naturally occurring, mobile genetic elements to integrate therapeutic genes at a defined location in the genome, making it safer than random integration, which can cause tumor formation.

4D Molecular Therapeutics Inc. has obtained IND clearance by the FDA for 4D-175, an R100 vector-based intravitreal genetic medicine, for the treatment of patients with geographic atrophy.

Scientists at Recode Therapeutics Inc. have developed an optimized lipid nanoparticle (LNP) to act on specific tissues.

Sarepta Therapeutics Inc. CEO Douglas Ingram said he expects “ferocious” demand for gene therapy Elevidys (delandistrogene moxeparvovec), granted full approval by the U.S. FDA for Duchenne muscular dystrophy (DMD). Shares of the Cambridge, Mass.-based firm closed June 21 at $16.72, up $37.22, or about 30% on the news.