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The CAR T-cell therapy was also recently approved for treating follicular lymphoma, chronic lymphocytic leukemia, and small lymphocytic lymphoma.

No serious adverse events or significant retinal atrophy occurred.

The CEO of Mission Bio discussed the company’s Tapestri platform for single cell sequencing.

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

An ASGCT poster focused on special ethical considerations of gene therapy human research and how to address them.

The company also announced that it will be reporting new data at the European Hematology Association 2024 Hybrid Congress.

The head of research at Mass General Brigham’s Cell and Gene Therapy Institute discussed research from the center presented at ASGCT 2024.

The company announced that the fifth patient was treated in its phase 1 clinical trial.

In honor of Healthy Vision Month, CGTLive® took a closer look at the clinical trial design for this novel treatment.

Transient responses in 2 of 3 participants may be due to limited persistence of CARv3-TEAM-E T-cells in the weeks after infusion.

The associate professor of otolaryngology from Harvard Medical School also shared his excitement for the positive data on AAV1-hOTOF.

Review top news and interview highlights from the week ending May 24, 2024.

The data comes from the phase 1/2 RESET-Myositis clinical trial (NCT06154252) and the phase 1/2 RESET-SLE clinical trial (NCT06121297).

Fifty percent of patients had stable disease or better, with 2 complete responses and 2 partial responses.

The cofounder and chief technology officer of Mammoth Biosciences discussed the importance of diverse approaches to gene editing to address a variety of indications.

Evan Weber, PhD, an assistant professor of pediatrics at Children's Hospital of Philadelphia, discussed his work on the role of the FOXO1 gene in T-cell persistence and exhaustion.

The cofounder and chief executive officer of Verve Therapeutics discussed the company’s pipeline.

B-VEC is approved in a gel formulation under the name Vyjuvek for skin wounds in dystrophic epidermolysis bullosa.

The chief medical officer of Forge Biologics discussed how expanded newborn screening practices may help the company’s gene therapy for Krabbe disease, FBX-101, reach more patients.

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

Ocugen’s gene agnostic modifier gene therapies include OCU400 and OCU410.

The chief scientific officer of Arbor Biotechnologies discussed the company’s preclinical candidate ABO-101.

Tab-Cel is approved under the name Ebvallo in Europe, Switzerland, and United Kingdom.

Excision is evaluating a higher dose in a second cohort as well as exploring alternative, potentially redosable, delivery methods.

The associate chief science officer of the Kennedy Krieger Institute discussed an aspect of clinical trial design highly relevant to gene therapy development for rare diseases.

Most participants had improved or stable cognitive function in a proof-of-concept study.

The chief medical officer of Forge Biologics discussed updated data from the REKLAIM clinical trial evaluating FBX-101.

Abhishek Gupta, BS, the senior vice president of genetic medicines at Syneos Health, discussed common setbacks in gene therapy trials and how to overcome them.