News

PepGen’s ongoing multiple ascending dose trial is expected to read out initial data in mid-2024.

The president and head of R&D at Taysha Gene Therapies discussed his expectation that younger patients my see even better results with the company’s gene therapy than the adult patients treated so far.

Kiromic announced that the first patient had been dosed with the gamma delta cell therapy in December 2023.

The Medical Director of Pediatric Hematology/Oncology at Sarah Cannon Research Institute discussed unmet needs that remain after gene therapy approval for SCD.

TAC01-CLDN18.2 integrates TAC, Triumvira’s proprietary chimeric receptor that is intended to activate and direct T-cells against tumor cells.

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

Tami John, MD, a clinical associate professor at Stanford Medicine, also discussed ongoing trends in sickle cell disease research.

Dose level 3 showed the most efficacy and has been selected for the phase 2 portion of the trial.

The chief hematology/oncology fellow at University of Chicago discussed further research his center is working on with cell therapy in B-cell acute lymphoblastic leukemia.

Hemogenyx originally submitted the IND to the FDA in May 2023 with the intention of gaining clearance for a phase 1 clinical trial in acute myeloid leukemia.

The cofounder and CEO of Neurona Therapeutics discussed the company’s goals to evaluate the cell therapy in more types of epilepsy and potentially even in Alzheimer disease.

Updated data from the AAVIATE trial were presented at the Hawaiian Eye and Retina meeting.

The cofounder, executive vice president, and chief medical officer, CLL Society, discussed unmet needs that remain for people with CLL and how the society is working to advance research in the field.

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

The clinical professor in the Department of Human Genetics at University of Texas Rio Grande Valley discussed research on racial differences in the Factor VIII gene that can impact treatment efficacy for hemophilia A.

Three of CARsgen’s CAR therapies are currently on clinical hold due to CMC questions.

The therapy’s original PDUFA date was March 30, 2024, but the TDT approval followed only a month after exa-cel's sickle cell approval.

Further research is needed to determine if cell therapy may be beneficial to certain subgroups of patients.

Among 3 patients in the study’s first cohort, a 3.6 to 6.6-fold increase in CSF PGRN over baseline was observed at 30 days posttreatment.

The cofounder and CEO of Neurona Therapeutics discussed results from a phase 1/2 clinical trial presented at AES’s annual meeting.

The trial is recruiting 5 more participants for the highest dose cohort following a positive DSMB review.

Tami John, MD, a clinical associate professor at Stanford Medicine, discussed a study on samples from patients who had received either HSCT or gene therapy for sickle cell disease.

The associate professor at Peter MacCallum Cancer Centre discussed highlights from the 2023 ASH meeting.

The president and head of R&D at Taysha Gene Therapies discussed positive early results from the first 2 patients treated with gene therapy TSHA-102.

Kadimasem’s lead clinical candidate, AstroRx, was recently cleared for trials in the US for people with ALS.

The first pediatric patient was treated in the United States, but the company has now also received clearance to treat pediatric patients in the UK.

Review top news and interview highlights from the week ending January 12, 2024.

Atara plans to submit a BLA for tab-cel, approved in Europe, in the second quarter of 2024.

Among 11 patients with PD who were treated in the study, there no serious adverse events (AEs) attributed to AB-1005.

The cofounder and CEO of Neurona Therapeutics discussed NRTX-1001, the company’s allogeneic regenerative neural cell therapy.