Noah Stansfield

The FDA also stated that it has concluded its investigation of the death an 8-year-old boy who was treated with Elevidys in Brazil.

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

In episode 5 of ImmunoLogic, Michael T. Lotze, MD, discusses the evolution and future of tumor-infiltrating lymphocyte therapies.

According to Rocket, the RMAT designation was granted based on results from a phase 1 clinical trial (NCT05885412).

The patient, an 8-year-old boy, was treated with Elevidys in Brazil.

Sven Moller-Tank, PhD, a senior director at Regeneron, also discussed his thoughts on trends in the gene therapy field in general.

Sven Moller-Tank, PhD, a senior director at Regeneron discussed the company’s receptor-driven approach to AAV targeting, leveraging antibody engineering to enhance tissue specificity and reduce off-target effects.

Review top news and interview highlights from the week ending July 25, 2025.

Ocugen initiates pivotal confirmatory trial for OCU410ST, its second modifier gene therapy candidate, in patients with Stargardt disease.

The therapy was approved based on findings of the pivotal phase 1b/2 FELIX clinical trial.

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

The McCaw Endowed Chair of Muscular Dystrophy at University of Washington, discussed the ways ongoing research into next-generation DMD therapies aims to improve then.

The agency also put several of the company’s clinical trials on hold.

The company stated that it has informed the FDA about its decision, which will go into effect at close of business on July 22, 2025.

The open-label trial, which launched on May 31, 2018, will treat up to 24 patients who have been diagnosed with ART-SCID.

The McCaw Endowed Chair of Muscular Dystrophy at University of Washington, discussed benefits and eligibility for newer DMD treatment options for older patients.

Review top news and interview highlights from the week ending July 18, 2025.

Atsena Therapeutics will advance ATSN-201 into a pivotal phase 1/2/3 trial following FDA agreement on study design and endpoints.

The CGTLive® team highlights 5 therapeutics that are nearing major decisions by the FDA.

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

Nathan Yozwiak, PhD, the head of research at the Gene and Cell Therapy Institute at Mass General Brigham, discussed the Institute’s efforts to bring about clinical translation of preclinical work.

According to the company, the BLA is the first for a gene-agnostic gene therapy for retinal disease to have been submitted to the FDA.

The head of research at Mass General Brigham’s Gene and Cell Therapy Institute discussed the Institute’s efforts to bring about clinical translation of preclinical work.

According to Ultragenyx, the CRL relates to a need for additional CMC information and improvements and observations from inspections of manufacturing facilities.

Derek Jackson, BS, MA, and Kilian Guse, PhD, of Pacira Biosciences, discussed the company’s symposium at ASGCT 2025.

Derek Jackson, BS, MA, the vice president of cell & gene therapy product development at Pacira BioSciences, discussed findings related to the immunogenicity of the therapy’s high-capacity adenovirus vector.

Review top news and interview highlights from the week ending July 11, 2025.

In the CRL, the FDA stated that the statutory requirement for “substantial evidence of effectiveness” was not met by the BLA and that more clinical data will be needed.

SGT-501 is intended to address CPVT by providing a full-length, codon-optimized copy of the cardiac calsequestrin gene to the muscle cells of the heart.

The McCaw Endowed Chair of Muscular Dystrophy at University of Washington, discussed how comprehensive care for DMD patients involves early diagnosis, steroid treatment, consideration of mutation-specific therapies, and more.