Lysosomal Disorders

The recent FDA Advisory Committee meeting follows a turbulent year for gene therapy studies.

Eric Crombez, PhD, senior vice president and chief medical officer of Ultragenyx Gene Therapy, discussed indications the company is targeting.

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 chief scientific officer and chief medical officer of Ultragenyx Gene Therapy discussed the company’s future research in gene therapies.

Parexel cell and gene therapy director Izaskun Elorza, MD, shares key factors for industry to consider as this emerging field becomes more prominent.

Sam Wadsworth, PhD, chief scientific officer of Ultragenyx Pharmaceuticals, discussed the company’s platform for efficiently producing AAV vectors.

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

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

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

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

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

Maria A. Croyle, PhD, discusses the manufacturing challenges facing the gene therapy space and her inspiration for developing a novel preservation method for viral vector-based therapies.

Typically treated with stem cell transplantation, this lentiviral vector-based gene therapy approach may resolve challenges related to donor availability and post-transplantation complications.

An autologous lentiviral-based gene therapy demonstrated significant benefit, including high overall and event-free survival rates, immune reconstitution, and metabolic correction, for patients with ADA-SCID.

The full regulatory approval of agalsidase beta has blocked the accelerated approval pathway for AVR-RD-01 as a treatment for patients with Fabry disease.

The first patient has been enrolled and treated with RGX-121 in the third cohort of a phase 1/2 study for patients up to 5 years old with severe MPS II.

Orchard Therapeutics released its plans for 2021, which included the launch of OTL-200 (Libmeldy) in Europe and development of an FDA submission strategy in the United States.

Descriptive findings from the phase 1/2 study allude to promise for the gene therapy in the rare pediatric disorder.

New phase 2/3 findings meet investigators' hypotheses on the gene therapy's mixed benefits for heparan sulfate reduction.

The AAV-based gene therapy was well-tolerated and showed enough promising effect to warrant further investigation, researchers reported.

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

The investigators found the primary efficacy endpoints occurred in 20 evaluable patients (87%).

FLT201 is a combination of AAVS3 capsid and a liver-specific promotor to drive the expression of GCasevar85.

New WORLDSymposium findings show a treated patient had 100% reduction in the accumulating fatty substrate associated with the burdensome rare disease after 1 year.