Noah Stansfield

According to the company, this is the first time therapeutic RNA editing has been clinically demonstrated in humans.

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

Among 7 patients’ eyes that were treated with the study’s high dose, the response rate was 57% (4/7 eyes).

Seven of 67 patients treated with eli-cel across multiple clinical trials have developed hematologic malignancies, according to findings published in The New England Journal of Medicine.

Benitec reported that the first patient showed a 35% reduction, constituting clinically meaningful improvement, in total score on the Sydney Swallow Questionnaire.

The REVEAL adolescent and adult trial and the separate phase 1/2 REVEAL Pediatric Study are both evaluating the gene therapy TSHA-102 in Rett syndrome populations.

Daniela van Eickels, MD, PhD, MPH, the vice president and head of medical affairs for Bristol Myers Squibb’s Cell Therapy Organization, also discussed the company’s planned presentation at ACR Convergence 2024.

Nanoscope intends to go forward with the BLA submission in the first quarter of next year.

Daniela van Eickels, MD, PhD, MPH, the vice president and head of medical affairs for Bristol Myers Squibb’s Cell Therapy Organization, discussed the Autoimmunity Cell Therapy Network.

Kiromic reported that the fourth patient to have been treated in the trial showed a 5.3% reduction in tumor size with regard to baseline.

The ORR in the enhanced lymphodepletion arm was 100% in patients who had not previously received an antiBCMA therapy.

Paul Melmeyer, MPP, the executive vice president of public policy & advocacy at MDA, discussed the growing interest in gene therapy for the annual meeting.

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

Arun Upadhyay, PhD, the chief scientific officer and head of research, development, and Medical at Ocugen, also discussed future plans for the gene therapy, which is now in a phase 3 study.

The vice president and head of medical affairs for Bristol Myers Squibb’s Cell Therapy Organization also discussed the company’s planned presentation at ACR Convergence 2024.

Ultragenyx noted that the fourth cohort would use a “moderately increased” dose and an optimized immunodulation regimen.

The vice president of Public Policy & Advocacy of the Muscular Dystrophy Association discussed the growing interest in gene therapy for attendees of the annual meeting.

Arun Upadhyay, PhD, the chief scientific officer and head of research, development, and Medical at Ocugen, discussed OCU400, the company’s gene-agnostic gene therapy for IRDs.

The vice president and head of medical affairs for Bristol Myers Squibb’s Cell Therapy Organization discussed the Autoimmunity Cell Therapy Network.

NEXICART-2 has moved on to its expansion cohort after completing dosing of patients in its initial cohort.

Estrella noted that the patient had been diagnosed with follicular lymphoma grade 3A with high-risk 3B symptoms.

Daniel Hart, PhD, the senior director and head of technology development at Epic Bio, discussed potential applications of the new technology.

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

CSF analyses from 7 patients treated in the study showed reductions of more than 80% in NAA levels from baseline.

Anixa noted that it had previously obtained clearance of a single-patient investigational new drug application to redose a patient in the trial.

The data, from the CARTITUDE-4 clinical trial, compared outcomes to patients treated with standard of care options.

The chief scientific officer and head of research, development, and Medical at Ocugen, also discussed future plans for the gene therapy, which is now in a phase 3 study.

The company pointed out that today, September 30, is LGMD Awareness Day.

John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia, discussed several still-investigational drugs and therapies for DMD.

Enrollment difficulties were cited as the reason for discontinuing recruitment activities for KarMMa-9.