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The program’s cancellation follows a previous announcement that its phase 3 trial had missed its primary end point.

Ractigen is also evaluating saRNA therapies for ALS and SMA.

The cofounder and chief science officer of Longeveron discussed updated data from the phase 2a CLEAR MIND study.

Pending positive outcomes, Actinium plans to later evaluate Iomab-ACT as a conditioning agent for SCD gene therapies.

The European Commission granted conditional marketing authorization under the name Beqvez to the European Union.

The chief scientific officer of Tome Biosciences discussed the company’s technologies in the context of integrative gene therapy and cell therapy.

There were no serious adverse events related to the cell therapy and no evidence of ARIA.

Steve Kanner, PhD, the chief scientific officer of Caribou Biosciences, discussed results from preclinical research evaluating the gene editing approach.

Omar Nadeem, MD, clinical director, Myeloma Cellular Therapies, Dana-Farber Cancer Institute, discussed GPRC5D-targeted CAR-T, BMS-986393.

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

The principal investigator at Seattle Children’s Research Institute discussed her lab’s work on using ultrasound mediated gene delivery to target hemophilia A.

The clearance is based on the agency’s acceptance of clinical trial protocols and a chemistry, manufacturing, and controls clinical development plan for the program.

Cell and gene therapies can transform the paradigm of care for patients with chronic, complex conditions, but these therapies come at an up-front cost of several million dollars per treatment, complicating the pipeline of access to them.

IASO also received IND clearance for a trial in myasthenia gravis in April 2024.

The Don and Marilyn Anderson Professor of Oncology at University of Wisconsin – Madison discussed the state of cell and gene therapy development.

It was noted that the patient, who is 1 of 6 treated in the study so far, may be showing clinical activity in response to their first dose with the CER-T.

Most participants maintained only over a 5% FVIII level after at least 15 months of follow-up.

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

The principal investigator at Seattle Children’s Research Institute discussed her lab’s preclinical research with UMGD for the treatment of hemophilia.

Both bluebird bio and Vertex Pharmaceuticals have received unfavorable opinions from the Office of the Inspector General on the matter.

The cofounder and chief scientific officer of Mammoth Biosciences discussed the company’s mouse model research on treating hypertriglyceridemia.

After 3 tries, Mesoblast finally has a PDUFA date for its MSC therapy.

Steve Kanner, PhD, the chief scientific officer of Caribou Biosciences, discussed the company’s platform for genome editing.

The cofounder and chief scientific officer at Encoded Therapeutics shared preclinical research with the company’s AAV and miRNA platforms.

Of 724 patients treated at the center, the cumulative incidence of secondary hematologic malignancy at 3 years posttreatment was 6.5%.

In honor of World Brain Day, observed annually on July 22 by patient and clinician communities, CGTLive is taking a closer look at this ongoing study.

The chief medical officer of Cartesian Therapeutics discussed data presented at ASGCT 2024 from a phase 2a study.

Paul Y. Song, MD, the chairman and chief executive officer of NKGen, discussed the potential of SNK01, the company’s autologous natural killer cell therapy, in treating PD.