Neurology

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.

With advancements happening at unprecedented rates, these are the cell and gene therapy companies and pipelines we’re keeping a close eye on.

The clinical-stage biotech company recently announced the expansion of their phase 1 trial of CYNK-001 in patients with relapsed/refractory acute myeloid leukemia.

The co-founder, president, and chief executive officer of Solid Biosciences, whose own son has DMD, discussed promising updates from their phase 1/2 IGNITE-DMD trial.

The head of neuroscience at Sangamo Therapeutics discussed advances in understanding disease biology that allow for more specific gene targeting.

The head of neuroscience at Sangamo Therapeutics discussed the potential of ZFPs in central nervous system disorders.

Interim data from a phase 1 study from Intellia Therapeutics and Regeneron Pharmaceuticals is the first to support in vivo CRISPR genome editing in humans.

The head of global research and executive vice president of Atara Biotherapeutics discussed the development of ATA188.

Corlieve Therapeutics’ lead program, AMT-260, previously demonstrated proof-of-concept in preclinical studies of temporal lobe epilepsy.

Newly published data continues to demonstrate Zolgensma's efficacy in presymptomatic and symptomatic SMA Type 1.