Neurology

Investigators observed sustained, dose-dependent benefits for young patients at 24 months.

Vamshi Rao, MD, attending physician of Neurology at Ann and Robert H. Lurie Children’s Hospital of Chicago, discusses how disease modifying therapies have improved spinal muscular atrophy (SMA) prognosis.

The associate professor at Hokkaido University and investigator of STEMTRA also discussed other efforts in stroke, Alzheimer disease, and Parkinson disease.

The Pfizer candidate PF-06939926 has also received fast track, orphan drug, and rare pediatric disease designations from the FDA.

Minimal fat infiltration was observed on MR images from the SRP-9001 arm compared to participants from the natural history cohort.

Results of the phase 2 study support continued dose escalation of SRP-5051 and further clinical development.

These data follow a recent announcement from Sio Gene Therapies that the first patient in the high-dose cohort has been dosed.

Solid Biosciences noted that its response to the agency requests for further information about manufacturing as well as updated safety and efficacy data on all dosed patients led to the lift.

Nearly all patients in this cohort achieved a clinically meaningful >3-point increase during the study period, demonstrating a consistent response to the gene therapy.

The agency said the gene therapy should remain on the market while it assesses the situation and does not impact their evaluation of data from the human clinical trials.

Interim data from the first 8 pediatric patients showed that the AAV-CLN6 gene therapy demonstrated a positive impact on motor and language function compared to a natural history dataset, as well as in comparison to in-study sibling pairs.

Preclinical trials and success stories suggest that much is riding on vector-based therapies for the treatment of rare neurological conditions.

AveXis—a Novartis company—announced that it will work with payers to implement 5-year outcomes-based agreements and novel pay-over-time options. The company also said it will offer a patient program to support affordability and access.

This is the first gene therapy approved for a devastating condition that leads to permanent ventilation or death for many patients by age 2.

Zolgensma, an adeno-associated virus vector-based, one-time gene therapy administered via intravenous infusion, is the first and only FDA-approved gene therapy for SMA.

New early-stage data suggest that vector‐mediated gene silencing of striatal CaV1.3 expression may hold promise for preventing the induction of levodopa-induced dyskinesias in Parkinson disease.

In a pair of phase 2 trials and a phase 1 study, patients with SMA types 1 and 2 treated with the gene therapy displayed a number of motor milestone achievements and a prolonged event-free survival rate.

Across 3 cohorts, the therapy showed improvements in on-time without troublesome dyskinesia, ranging from 2.1 hours to 3.5 hours.

The FDA has granted a Breakthrough Therapy designation to bluebird bio, Inc’s Lenti-D, a gene therapy for patients with cerebral adrenoleukodystrophy, an X-linked genetic disorder caused by a defect in the gene ABCD1.

Supported by positive data from an ongoing Phase 2/3 study, bluebird bio’s Lenti-D has been granted Breakthrough Therapy designation by the US FDA for the treatment of patients with cerebral adrenoleukodystrophy.

Pfizer announced that it has begun a Phase 1b clinical trial for PF-06939926, its mini-dystrophin gene therapy candidate, intended for boys with Duchenne muscular dystrophy.

Parent Project Muscular Dystrophy announced that the first patient with Duchenne muscular dystrophy has been dosed with microdystrophin gene therapy.