Neurogene

Gene Therapy Net serves as a key resource for gene therapy information, including research, clinical trials, and regulations. Recent highlights include Novartis' promising SMA gene therapy, India's successful haemophilia-A gene therapy, Neurogene's FDA-permitted Rett syndrome trial continuation, a novel prenatal gene therapy technique, concerns over Bluebird bio's Skysona side effects, and ISCT's ANAB accreditation for workforce development in cell and gene therapy.

Roche partners with Innovent in a $1B deal. Tenaya's shares drop due to trial results. Incyte and iTeos halt drug developments. BioNTech's BNT327 could challenge Keytruda. AstraZeneca, Daiichi Sankyo, BioNTech, and Merus present cancer drug data. Merck and others target glioblastoma. BridgeBio's Attruby approved, competing with Pfizer and Alnylam. Neurogene continues Rett syndrome trial with adjusted dose.

In 2024, significant progress in gene therapy for medical disorders was noted, including neurology. Highlights include a DMD patient's death in Pfizer’s trial, FDA clearance for Jaguar’s autism gene therapy, a critical condition in Neurogene’s Rett syndrome trial, Genethon’s DMD therapy success, and FDA approval of PTC’s AADC deficiency therapy.

FDA investigates bluebird bio's Skysona gene therapy after reports of secondary blood cancers in treated patients, linked to clonal vector insertions and gene mutations. The FDA advises considering alternative therapies and is evaluating further regulatory action.

Neurogene and Taysha Gene Therapies are developing gene therapies for Rett syndrome, with clinical trials in Phase 1/2. Neurogene's NGN-401 and Taysha's TSHA-102 aim to regulate MECP2 expression, crucial for treating the disorder. Despite a severe adverse event in Neurogene's high-dose trial, both companies remain optimistic about their therapies' potential to treat Rett syndrome at its source.

A girl with Rett syndrome died after receiving Neurogene’s high-dose gene therapy, prompting the company to halt tests of that dose but continue with a lower dose. The FDA allowed the trial to proceed with the lower dose after reviewing safety data. Neurogene plans to remove the high-dose from the trial protocol and does not plan to enroll further participants in that group. The therapy aims to deliver the MECP2 gene via AAV9 vector to address Rett syndrome’s symptoms.

Cerevance’s phase 3 ARISE trial for Parkinson disease has begun dosing with solengepras. Sage Therapeutics discontinued SAGE-718 development after phase 2 DIMENSION study failed to meet end points in Huntington Disease. Neurogene paused high-dose NGN-401 use in Rett syndrome trial due to a serious adverse event.

This week's highlights include FDA actions, orphan drug designation, interim data from a CAR-T trial, trends in cell and gene therapy, and biomarker data indicating potential for treating autoimmune diseases.