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David Porter, MD, director of Cell Therapy and Transplant, Penn Medicine discussed how experience in oncology enabled the logical expansion.

Another recent program in the investigational landscape was Helixmith’s deprioritized Engensis diabetic neuropathy gene therapy.

The first-in-human, open-label, single-arm trial is expected to enroll 9 participants over the course of a year.

The chief of cardiology at Weill Cornell Medical College shared his outlook on the future of gene therapy research in the field.

Emilie Aschenbrenner, PharmD, BCOP, a hematology coordinator for pharmacy at Froedtert and the Medical College of Wisconsin, discussed the advantages of each of the 2 modalities in the third and second line settings.

Review top news and interview highlights from the week ending April 26, 2024.

The decision was supported by the phase 3 BENEGENE-2 trial, which suggested that the Pfizer gene therapy was superior to standard of care FIX prophylaxis.

The cells may have potential to modulate disease in other cases of respiratory disease and critical illness.

The medical director of the Center for Inherited Blood Disorders discussed the entrance of BioMarin’s val-rox into his center’s practice.

AlloNK is being administered in the trial alone or alongside 1 of 2 antiCD20 monoclonal antibodies: rituximab or obinutuzumab.

The professor from Cedars Sinai discussed data he is anticipating from further investigations into lifileucel.

The company expects to open the first clinical site in the DOSED study in the second quarter of 2024.

The President Elect of ASGCT and a distinguished professor of microbiology at Keck School of Medicine of USC also discussed recent milestones in gene therapy.

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

A case study previously described successful immune modulation with AgenT-797 in a patient with high-risk gastric cancer leading to a partial response.

The lead scientist at Percheron Therapeutics discussed a phase 2 blinded study of ATL1102 being conducted in the Europe and Australia.

The EC’s decision comes several weeks after the FDA approved cilta-cel for a similar expanded indication.

The Assistant Professor of Medicine at Weill Cornell Medical College discussed the changing cell transplant landscape.

The principal investigator at the Gene Therapy Program of Dana-Farber/Boston Children’s Cancer and Blood Disorder Center discussed innovative uses of gene editing technology.

The FDA is allowing a small, single-arm study to support a BLA submission of ST-290, if Sangamo can find help to continue developing the therapy.

The FDA is requesting more CMC information, specifically with regard to validation requirements for specific manufacturing and release testing methods.

The results come from the randomized, double-masked, placebo-controlled phase 3 REFLECT clinical trial (NCT03293524).

Acepodia is also evaluating a CD20-targeted γδ2T-cell therapy in patients with non-Hodgkin lymphoma.

The attending physician and assistant member of bone marrow transplantation and cellular therapy at St Jude Children’s Research Hospital also discussed hurdles to accessibility for SCD gene therapy.

At the 90 day timepoint, the patient showed improvements in swallowing compared to their own natural history data, as measured by videofluoroscopic swallowing studies.

The associate professor of dermatology at Stanford University discussed how the May 2023 approval of B-VEC may shift the treatment field for RDEB.

Alexis Kuhn, PharmD, BCOP, a pediatric oncology pharmacist at Mayo Clinic, discussed the incorporation of the recently FDA-approved gene therapies for SCD and TDT into the work of pharmacists.

The medical director of the UCLA Bone Marrow Transplantation Stem Cell Processing Center discussed results from a phase 1b trial for allo-HCT alternative Orca-T that he presented at Tandem’s 2024 conference.

Review top news and interview highlights from the week ending April 19, 2024.

The FDA has requested that black box warnings related to secondary cancer risks be added to all 6 CAR-T therapies currently on the market.