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 companies 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.

The full 52-week analysis of the phase 2 LUNA trial are expected in the first half of 2025.

Passage Bio received the feedback on a proposal to amend the upliFT-D protocol in the context of a Type C meeting process with the FDA.

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

In observance of Sarcoma Awareness Month, held annually in July, we took a look back at the past year's news in cell/gene therapy for this cancer.

The associate professor of medicine at University of Colorado discussed data presented at ASCO 2024.

The gene therapy has continued to demonstrate efficacy in updated data from the phase 2 PRISM trial.

The chief scientific officer of Caribou Biosciences discussed results from preclinical research evaluating the gene editing approach.

Longeveron will present updated data from the phase 2a CLEAR MIND study at AAIC 2024 at the end of July.

The chief medical officer at Creyon Bio overviewed the development of an allele-selective TNPO2 protein for a single patient with a de novo pathology.

BRG01 is engineered to target the EBV antigen, which is frequently expressed on nasopharyngeal carcinoma tumor cells.