Neurology

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.

LogicBio's Mariana Nacht, PhD, shares details of the company's novel capsid discovery platform and gene editing technology.

The news comes after CRISPR’s favorable data readouts in its sickle cell and beta thalassemia studies.

Cynata Therapeutics' Ross Macdonald, PhD, discusses the company's pipeline and clinical-phase trials.

Bayer provided an update on trials currently underway for the treatment of PD by its subsidiaries.

Locanabio CEO James Burns, PhD, discusses the firm's targeted approach to treating genetic disorders of the central nervous system and the eye.

Asklepios BioPharmaceuticals noted that it plans to initiate dosing for the LION-101 phase 1/2 clinical trial in the first half of 2022.

Ross Macdonald, PhD, managing director and CEO of Cynata Therapeutics, discusses the biotech company's approach to overcoming reproducibility challenges with mesenchymal stem cells.

An independent board has advised that a phase 1/2 study assessing AMT-130 for adults with early manifest Huntington disease should continue as planned.

Maria A. Croyle, PhD, discusses the development of a novel film matrix that can safely and effectively store and transport AAV-based gene therapies.

SRP-9001 manufactured with commercial materials demonstrated robust transduction efficiency and high gene expression levels for DMD.