Lysosomal Disorders

Partners in the Bespoke Gene Therapy Consortium include the NIH, FDA, private companies such as Pfizer and Biogen, and non-profits.

André Choulika, PhD, chief executive officer and cofounder, Cellectis, discussed the company’s expansion into gene therapies.

The phase 1 study of NTLA-2001 previously showed positive interim data, including serum TTR reductions, which were reported in June 2021.

Review top news and interview highlights from the week ending October 29, 2021.

The FDA cleared the IND application for HMI-203 in October 2021.

The professor from National Taiwan University Hospital discussed the mechanism of gene therapy in AADC deficiency.

Dabocemagene autoficel has previously received orphan drug, rare pediatric disease, fast track, and regenerative medicine advanced therapy designations.

Homology is also developing a gene therapy, HMI-102, being evaluated in the phase 1/2 pheNIX trial.

The phase 1/2 SUNRISE trial is now enrolling patients as young as 6 months old with methylmalonic acidemia after positive review under a DSMB.

Review top news and interview highlights from the week ending October 22, 2021.

Rocket Pharmaceuticals announced positive data on RP-L201 in leukocyte adhesion deficiency-I, as well as RP-A501 for Danon disease.

AVROBIO shared interim safety data from ongoing phase 1 and 2 trials.

The director of the Powell Gene Therapy Center at the University of Florida discussed improving access to gene therapies for patients with rare diseases worldwide.

Review top news and interview highlights from the week ending October 15, 2021.

New human gene editing therapies, drug discoveries, targets, and CRISPR technology holds the potential to usher in a new age in medicine.

The chair of the International Collaborative Gaucher Group Registry discussed challenges in Gaucher research.

Review top news and interview highlights from the week ending October 8, 2021.

The director of the Powell Gene Therapy Center at the University of Florida discussed adverse events in gene therapies.

Paul Wotton, PhD, chief executive officer, Obsidian Therapeutics, discussed the company’s pipeline and technologies.

The chair of the International Collaborative Gaucher Group Registry discussed the place of gene therapies in the Gaucher treatment landscape.

The director of the Powell Gene Therapy Center at the University of Florida discussed the recent meeting of the FDA Cellular, Tissue, and Gene Therapies Advisory Committee.

Review top news and interview highlights from the week ending October 1, 2021.

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.

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.