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Five patients in the cohort achieved this milestone and Sernova noted a sixth patient in the cohort is still awaiting assessment of islet graft function.

Taysha also provided updates on its gene therapy program for Rett syndrome.

The FDA’s decision to approve this first gene therapy in hemophilia A was based on data from the phase 3 GENEr8-1 clinical trial.

Benitec Biopharma is planning to initiate a phase 1b/2a clinical trial to evaluate BB-301.

Detailed topline data will be presented at the 2023 International Congress on Parkinson’s Disease and Movement Disorders in August.

With an original PDUFA date in August 2021, the decision is a long time coming.

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

The vice president of business development and alliances at Prescient Therapeutics discussed advantages of the OmniCAR, SpyTag, and SpyCatcher technologies.

Updated data are from a total of 36 patients treated in a phase 1/2 study as of June 2023.

The director of research and development at Teknova discussed the company’s new screening kit and its potential to speed up AAV gene therapy development.

In addition to the BLA acceptance, Pfizer noted that a European MAA for the gene therapy has been accepted by the EMA.

Susan Bal, MD, assistant professor of medicine at University of Alabama – Birmingham, discussed new data on BMS-986393.

Among 4 patients who were treated at the highest dose level evaluated using a manufacturing process referred to as Process A, all 4 patients achieved responses.

The scientific cofounder and chief executive officer of Alloplex Biotherapeutics discussed progress of the phase 1 trial and possible future evaluations of the therapy.

Jason Westin, MD, FASCP, the director of the Lymphoma Clinical Research Program at University of Texas MD Anderson Cancer Center, discussed factors that limit patient access to treatment and a potential solution.

The director of the Medical Oncology Service at the National Cancer Institute discussed the START-001 trial.

The new analysis is from a phase 1 trial of Celularity’s legacy MLASC therapy.

The new data from the TRANSCEND FL and TRANSCEND NHL 001 studies are the latest in a series of positive updates regarding liso-cel that have been announced by Bristol Myers Squibb in recent months.

The director of the Lymphoma Clinical Research Program at University of Texas MD Anderson Cancer Center discussed axi-cel's safety profile and an important factor for improving access to CAR-T therapies.

Review top news and interview highlights from the week ending June 23, 2023.

A phase 1/1b clinical trial is planned to evaluate the safety and efficacy of the therapy.

The associate professor from Dana Farber Cancer Center and Harvard Medical School discussed continued efficacy and safety data presented at the 2023 EHA Meeting.

Jason Westin, MD, FASCP, the director of the Lymphoma Clinical Research Program at University of Texas MD Anderson Cancer Center, discussed the implications of data he presented at ASCO’s 2023 conference.

Delandistrogene moxaparvovec treatment will reportedly cost $3.2 million.

The company stated that this year it plans to initiate a single dosage study that will serve as a subgroup of a larger phase 3 clinical trial.

The assistant professor at MD Anderson Cancer Center discussed new data she presented at the 2023 ASCO Meeting.

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

uniQure deemed the safety profile for AMT-130 to be manageable.

The assistant professor in the Division of Lymphoma at City of Hope discussed the potential implications of real-world data she presented at ASCO’s 2023 conference.

Data from a phase 1/2 clinical trial of NRTX-1001 were presented at the ISSCR 2023 Annual Meeting.