CGTLive’s Weekly Rewind – September 9, 2022

Article

Review top news and interview highlights from the week ending September 9, 2022.

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. CAR T/CARVac Therapy Yields Responses in Refractory Testicular Cancer

BNT211 showed encouraging responses in testicular cancer but less encouraging responses in ovarian cancer.

2. Sequential T-Cell Redirection Therapies Yield Deep, Durable Responses in R/R Multiple Myeloma

Patients receiving CAR T-cell therapy or bispecific antibody therapies as first salvage therapy had an ORR of 84%.

3. Mark Forman, MD, PhD, on Genetic Testing and Further Research in Frontotemporal Dementia

The chief medical officer of Passage Bio discussed research needs in FTD.

4. Polycythemia Vera mRNA Therapy Receives FDA Fast Track Designation

SLN124 was well-tolerated in a previous clinical trial involving healthy volunteers.

5. Ornithine Transcarbamylase Gene Editing Program Nabs Another FDA Designation

iECURE is planning to submit an IND for GTP-506 in mid-2023.

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Linda Marbán, PhD, the CEO of Capricor Therapeutics
Matthew Wicklund, MD, a professor of neurology at the University of Texas Health Science Center San Antonio
Matthew Wicklund, MD, a professor of neurology at the University of Texas Health Science Center San Antonio
Renier Brentjens, MD, PhD, the chair of the department of medicine and the deputy director at Roswell Park Comprehensive Cancer Center
Renier Brentjens, MD, PhD, the chair of the department of medicine and the deputy director at Roswell Park Comprehensive Cancer Center
Renier Brentjens, MD, PhD, the chair of the department of medicine and the deputy director at Roswell Park Comprehensive Cancer Center
Renier Brentjens, MD, PhD, the chair of the department of medicine and the deputy director at Roswell Park Comprehensive Cancer Center
Robert Califf, MD, MACC, a cardiologist and former FDA commissioner
Natalie Goedeker, CPNP, on Handling Neuromuscular Gene Therapy at Real-World Sites
Alfred L. Garfall, MD, MS, associate professor of medicine (hematology-oncology) and director, Autologous Hematopoietic Cell Transplantation, Cell Therapy and Transplant Program, Hospital of the University of Pennsylvania; and section chief, Multiple Myeloma, Division of Hematology/Oncology, Department of Medicine, Perelman School of Medicine, University of Pennsylvania,
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