Victoria Johnson

Taysha also provided updates on its gene therapy program for Rett syndrome.

Detailed topline data will be presented at the 2023 International Congress on Parkinson’s Disease and Movement Disorders in August.

With an original PDUFA date in August 2021, the decision is a long time coming.

Updated data are from a total of 36 patients treated in a phase 1/2 study as of June 2023.

Susan Bal, MD, assistant professor of medicine at University of Alabama – Birmingham, discussed new data on BMS-986393.

The new analysis is from a phase 1 trial of Celularity’s legacy MLASC therapy.

Delandistrogene moxaparvovec treatment will reportedly cost $3.2 million.

Data from a phase 1/2 clinical trial of NRTX-1001 were presented at the ISSCR 2023 Annual Meeting.

The company believes that limitations on bridging therapies were a contributing factor to the death.

More work remains to be done to improve accessibility to populations and countries that have the highest unmet need with SCD treatment.

The advocacy groups are funding a 1-year study to be led by Barry Byrne, MD, PhD, at University of Florida.

CGTLive takes a look at challenges that have beset the first gene therapy coming close to market for treating Duchenne muscular dystrophy in 2023.

The phase 1/2 Euplagia-1 trial is currently ongoing in Europe.

New data from 3 patients were published in the New England Journal of Medicine.

The therapy seems to have a similar benefit in the first patient with TDT in the EdiTHAL trial.

Sang Yoon Moon, PhD, postdoctoral Research Associate, Lions Eye Institute, discussed research with CRISPRa in human fibroblast cultures.

Follow-up data up to 3 years showed supraphysiologic levels of blood IDUA and decrease of GAGs.

CRISPR Therapeutics and Vertex Pharmaceuticals completed their rolling submissions in April 2023.

The most recent data from CARTITUDE-4 were presented at the 2023 ASCO Meeting.

Updated data were presented at the ISCT 2023 Meeting.

The study has enrolled 17 patients as of May 2023 and enrollment is expected to complete by the end of 2024.

New overall survival analysis data on Yescarta from ZUMA-7 were presented at the ASCO 2023 meeting.

Safety findings were also improved when compared with the previous CARTITUDE-1 study, suggesting the therapy may be better tolerated in earlier lines of therapy.

The study began enrollment in January 2023.

Follow-up data were presented from the discontinued phase 3 OVAL study.

Maria Pia Morelli, MD, PhD, assistant professor at MD Anderson Cancer Center, discussed the first-in-human study of Sleeping Beauty TCR-T cells.

In more positive news, the company’s phase 2 trial for treating DMD will enroll patients in the second half of 2023.

The promising preclinical data, observed in an accurate model of brain metastases, support initiation of a phase 1 trial.

Lena Winstedt, PhD, Hansa Biopharma, discussed imlifidase, which is being investigated in combination with gene therapies from Sarepta and Genethon.

All evaluable patients had improvements in natriuretic peptides, left ventricular volume, and NYHA class.