CGTLive’s Weekly Rewind – June 23, 2023

Article

Review top news and interview highlights from the week ending June 23, 2023.

Welcome to CGTLive™’s Weekly Rewind! We’ve compiled 5 highlights from this week’s coverage of advances in gene and cell therapies, including FDA actions, notable research, and interviews with experts across the field.

1. FDA Approves Sarepta's Landmark DMD Gene Therapy Elevidys

Delandistrogene moxaparvovec treatment will reportedly cost $3.2 million.

2. Nikhil Munshi, MD, on Final Data on Cilta-Cel in Multiple Myeloma From CARTITUDE-1

The associate professor from Dana Farber Cancer Center and Harvard Medical School discussed continued efficacy and safety data presented at the 2023 EHA Meeting.

3. 2023 World Sickle Cell Day: The Home Stretch for Gene Therapy in SCD?

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

4. Maria Pia Morelli, MD, PhD, on Promising Efficacy of Sleeping Beauty TCR-T Therapy in Solid Tumors

The assistant professor at MD Anderson Cancer Center discussed new data she presented at the 2023 ASCO Meeting.

5. Patient Death Prompts Hold on Arcellx’s CART-ddBCMA for R/R Multiple Myeloma

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

Recent Videos
Chun-Yu Chen, PhD, a research scientist at Seattle Children’s Research Institute
William Chou, MD, on Targeting Progranulin With Gene Therapy for Frontotemporal Dementia
Alexandra Collin de l’Hortet, PhD, the head of therapeutics at Epic Bio
Joshua M. Hare, MD, on Working to Address Unmet Needs in Alzheimer Disease With Lomecel-B Cell Therapy
William Chou, MD, on Expanding Frontotemporal Dementia Gene Therapy to Both GRN and C9orf72 Mutations
Michael Severino on In Vivo Gene Editing With RNA Gene Writers
Chris Wright, MD, PhD, on Annelloviruses, a Potential Alternative to AAV for Gene Therapy
Joshua M. Hare, MD, on Promising Data on Lomecel-B for Alzheimer Disease
Carol Miao, PhD, a principal investigator at Seattle Children’s Research Institute
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