CGTLive’s Weekly Rewind – March 11, 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. Working to Find a Cure for Charcot-Marie-Tooth: Keith Fargo, PhD

The chief scientific officer of the CMT Research Foundation discussed the foundation’s mission. He discussed CMT and its disease burden on patients as well as the unmet needs in the disease.

2. Mesenchymal Stromal Cell Therapy Yields Improvements in Colitis

Remestemcel-L, Mesoblast’s mesenchymal stromal cell therapy, demonstrated rapid mucosal healing and disease remission in patients with refractory ulcerative (NCT04543994) or Crohn colitis (NCT04548583), according to results from a phase 1b/2a study published in the Journal of Crohn’s and Colitis.

3. Advantages of Invariant Natural Killer T Cell Therapies

Jennifer Buell, PhD, president and chief executive officer, MiNK Therapeutics, discussed the company’s iNKT cell platform. She discussed the advantages of iNKT therapies over more traditional cell therapies.

4. Intellia’s CRISPR-Engineered Cell Therapy Receives Orphan Drug Designation for Acute Myeloid Leukemia

The FDA has granted orphan drug designation to Intellia Therapeutics’ novel T-cell receptor (TCR) T-cell therapy NTLA-5001 for the potential treatment of acute myeloid leukemia (AML).

5. Global Awareness, Diagnosis a Priority for Rare Diseases: Michael Parini, JD

The chief executive officer and director of Freeline Therapeutics discussed the importance of Rare Disease Day and diagnosis of rare diseases outside the US and Europe, and how collaboration is key to developing therapies in this space.