CAP-1002 met primary and secondary end points in the HOPE-2 trial.
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Patient-reported outcomes, 6-minute walk test, and NSAA scores all showed improvements over natural history data.
The director of the Powell Gene Therapy Center at the University of Florida discussed gene therapy programs being conducted for Pompe disease.
Review top news and interview highlights from the week ending September 24, 2021.
The recent FDA Advisory Committee meeting follows a turbulent year for gene therapy studies.
Following a voluntary pause, a patient experiencing a liver AE has died and the trial is now on clinical hold.
Samarendra Mohanty, PhD, cofounder and president, Nanoscope Therapeutics, discussed the company’s technological platforms and RESTORE trial data.
NeuExcell and Spark Therapeutics each add to their gene therapy pipelines with the collaborative program.
Eric Crombez, PhD, senior vice president and chief medical officer of Ultragenyx Gene Therapy, discussed indications the company is targeting.
Magali Taiel, MD, chief medical officer, GenSight Biologics, discussed future research the company plans to pursue.
With numerous targets in CNS diseases and a unique partnership with gene therapy experts at UT Southwestern, Taysha is hoping to fill significant unmet needs.
The first US-based patient has been dosed in Lysogene’s clinical trial of LYS-GM101, following 2 other competing trials.
The 2 companies will focus their strategic partnership on the development of gene therapies for rare diseases, as well.
Salvador Rico, MD, PhD, chief medical officer, Encoded Therapeutics, discussed the different initiatives the company is conducting in Dravet syndrome.
A phase 1/2 study evaluating Vivet Therapeutic’s VTX-801 is set to initiate in August 2021.
Parexel cell and gene therapy director Izaskun Elorza, MD, shares key factors for industry to consider as this emerging field becomes more prominent.
Salvador Rico, MD, PhD, chief medical officer, Encoded Therapeutics, discussed the company’s gene therapy pipeline, including their lead program ETX101.
Bluebird Bio’s latest clinical hold follows another report of MDS in a treated patient.
The company announced plans to initiate the STEER study of intrathecal OAV-101 for older patients with SMA type 2.
The designations allow for expedited drug development and review of the investigational gene therapies for CLN2 Batten disease and SOD1 ALS.
The co-founder, president, and chief executive officer of Solid Biosciences, whose own son has DMD, discussed challenges in developing gene therapies for DMD.
The chief executive officer and co-founder of Flexion Therapeutics discussed the company’s pain-focused pipeline, which includes a gene therapy that targets inflammation.
Joseph Sullivan, MD, and Elaine C. Wirrell, MD, discuss new developments for the treatment of Dravet syndrome beyond seizure control including gene editing and therapy.
The co-founder, president, and chief executive officer of Solid Biosciences, whose own son has DMD, discussed some challenges the company is facing.
John Brandsema, MD, discusses gene transfer for the management of SMA and the recent approval of onasemnogene abeparvovec-xioi.
