CGTLive’s Weekly Rewind – April 1, 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. B-VEC Gene Therapy Heals Recessive Dystrophic Epidermolysis Bullosa Wounds

Beremagene geperpavec (B-VEC, Krystal Biotech) promotes durable wound healing in patients with recessive dystrophic epidermolysis bullosa (RDEB), according to preliminary data from the phase 3 GEM-3 trial (NCT04491604).

2. Renal Cell Carcinoma CAR T-Cell Therapy Fast Tracked

The FDA has granted fast track designation to Allogene Therapeutics’ chimeric antigen receptor (CAR) T-cell therapy ALLO-316 for the potential treatment of advanced or metastatic clear cell renal cell carcinoma (RCC).

3. Improving Speed and Fidelity of CRISPR/Cas9: Kenneth Johnson, PhD, and David Taylor, PhD

Kenneth Johnson, PhD, and David Taylor, PhD, professors and researchers from The University of Texas at Austin discussed new findings of Cas9 structure and mechanisms,

4. CAR T-Cell Expansion Indicative of Response in B-cell Lymphoma

Data from a prospective in vivo assessment suggest that good chimeric antigen receptor (CAR) T-cell expansion on day 10 (C10) after infusion could be an early biomarker to help predict response and survival in patients with B-cell lymphoma.

5. Liso-cel Shows Survival Benefit in R/R Large B-Cell Lymphoma

Lisocabtagene maraleucel (liso-cel, Breyanzi), a chimeric antigen receptor (CAR) T-cell therapy, demonstrated benefit to event-free survival (EFS) for patients with relapsed/refractory large B-cell lymphoma (R/R LBCL).