CGTLive’s Weekly Rewind – March 25, 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. T-Cell Therapy Gets Orphan Drug Designation for AML

The FDA has granted orphan drug designation to TC Biopharm's T-cell therapy TCB-002 (OmnImmune) for the potential treatment of relapsed/refractory acute myeloid leukemia (AML).

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

Researchers Kenneth Johnson, PhD, professor, biochemistry, and member, Institute for Cell and Molecular Biology, and David Taylor, PhD, assistant professor, department of molecular biosciences, The University of Texas at Austin, and colleagues, have identified regions in Cas9 that allow mismatches in DNA binding.

3. Neutralizing Antibody Versus Total Antibody Assays in Gene Therapy Development: Key Considerations for Assessing Immunogenicity

Viral vectors have become the most common method for delivering gene therapy; however, pre-existing humoral immunogenicity can potentially render a gene therapy ineffective. In cases in which clinical trial sponsors choose to exclude patients with pre-existing antibodies to a GT product, the FDA recommends consideration of “contemporaneous development of a companion diagnostic” to detect such antibodies.

4. Lineage Cell Therapeutics to Develop Cell Therapy for Hearing Loss

Lineage Cell Therapeutics is expanding its pipeline to develop a cell therapy for the potential treatment of hearing loss. With the expansion, Lineage is adding a third neuronal cell type to its platform. The company has filed for intellectual property covering the composition and methods of generating this type of cell therapy, including the generation of auditory neuronal progenitors, as well as methods of treatment involving these potential cell therapies.

5. CAR T-Cell Therapy Promising in R/R Chronic Lymphocytic Leukemia

The CD19-directed chimeric antigen receptor (CAR) T-cell therapy ARI-0001 has demonstrated efficacy in relapsed or refractory (R/R) high-risk chronic lymphocytic leukemia (CLL) and Richter transformation, according to a recent analysis of a small study.