News

Patients treated at Dose 2 in the dose-escalation phase of Part 1 of the study showed a persistent 68% reduction in CK levels over 2 years.

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

OAV101 IT is a version of Zolgensma that is delivered directly to the spine.

The chief scientific officer at Avidity Biosciences discussed the function and mechanism of the investigational antisense treatment, which is in development for DMD amenable to exon 44 skipping.

Acute liver injury is known to be a possible adverse event associated with AAV vector-based gene therapies such as Elevidys.

The findings were presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference

CGTLive® took a closer look at the single-center, open-label study, which aims to assess the safety and efficacy of the CAR-T therapy.

Adverse events related to the gene therapy itself were mild or moderate, with most occurring in the first 90 days posttreatment.

The ASTRA and accompanying POLARIS studies are actively enrolling patients.

John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia, offered insights into the obstacles the clinical community is facing around integrating gene therapies into clinical practice.

The study included 48 patients, 36 of whom received laromestrocel and 12 of whom received a placebo.

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

David-Alexandre Gros, MD, the chief executive officer of Eledon Pharmaceuticals, discussed the company’s collaboration with EGenesis.

In Episode 2 of ImmunoLogic, Bruce Levine, PhD, discussed the current the risk-benefit-ratio for CAR-T therapy.

The pediatric neurologist at Children’s Hospital of Philadelphia offered insights into the obstacles the clinical community is facing around integrating gene therapies into clinical practice.

In observance of Bleeding Disorders Awareness Month, held annually in March, catch up on some of the latest news and expert insights in gene therapy for hemophilia, which is among the most common of these disorders.

David Barrett, JD, the chief executive officer of ASGCT, broke down the highlights of the organization’s final report for 2024.

Review top news and interview highlights from the week ending March 7, 2025.

CALEC completely restored the cornea in 50% of participants at their 3-month visit and that rate of complete success increased to 79% and 77% at their 12- and 18-month visits, respectively.

Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive™ team.

Neurotech expects that the therapy will become available in the United States in June 2025.

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

According to Ocugen, the EMA has granted OCU410 and OCU410ST Advanced Therapy Medicinal Product classification.

Reena Sharma, MD, an adult metabolic consultant at Salford Royal Hospital, discussed areas of interest for future study for the Gaucher disease gene therapy product.

The chief executive officer of Eledon Pharmaceuticals discussed the company’s collaboration with EGenesis.

The agency has set the PDUFA action date for the BLA as August 31, 2025.

In observance of World Hearing Day, held annually on March 3, CGTLive® took a look back at the past year's news and expert insights in gene therapy for hearing loss indications.

Following up on Rare Disease Day, observed on February 28, Mary Schroth, MD, FAAP, FCCP, chief medical officer at Cure SMA, shared insights on the advances and promise for treating spinal muscular atrophy.