Lysosomal Disorders

Michael Singer, MD, PhD, cofounder and chief scientific officer of Cartesian Therapeutics, discussed the company’s lead candidate, Descartes-08.

Kinnari Patel, PharmD, president and chief operating officer of Rocket Pharmaceuticals, discussed Rocket Pharma’s programs.

Viral vectors have become the most common method for delivering gene therapy; however, pre-existing humoral immunogenicity can potentially render a gene therapy ineffective.

Review top news and interview highlights from the week ending March 18, 2022.

The draft guidance from the Center for Biologics Evaluation and Research outlines safety considerations throughout the clinical development program timeline.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The associate professor from UC San Diego discussed the ongoing phase ½ trial of CTNS-RD

The company is planning to initiate a phase 1 trial in the second half of 2022.

Review top news and interview highlights from the week ending March 11, 2022.

The director of research and development at AVROBIO discussed the molecular follow-up of cell therapies.

The senior vice president of clinical development at Passage Bio discussed biomarker activity seen in the IMAGINE-1 trial.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The chief executive officer and director of Freeline Therapeutics discussed the importance of Rare Disease Day.

Both patients dosed experienced a stable increase in cerebrospinal fluid HexA activity.

Review top news and interview highlights from the week ending March 4, 2022.

David Taylor, PhD, an assistant professor in the department of molecular biosciences at UT Austin, discussed the Cas9 research conducted between 2 labs at UT Austin.

The senior vice president and head of clinical development at Mustang Bio discussed the importance of Rare Disease Day.

The decision is the latest action in a line of patent interferences over the revolutionary gene-editing technology.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The cofounder and chief scientific officer of Cartesian Therapeutics discussed the importance of Rare Disease Day.

The therapy was well-tolerated in 15 patients and no clinically relevant liver findings were observed.

The professor from National Taiwan University Hospital discussed the importance of Rare Disease Day for raising awareness.

Clinical and industry leaders share their perspectives on the importance of collaboration in developing treatments for rare diseases.

The president and chief operating officer of Rocket Pharmaceuticals discussed the importance of Rare Disease Day.

The director of the Powell Gene Therapy Center at the University of Florida discussed the importance of Rare Disease Day.