CGTLive’s Weekly Rewind – March 4, 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. Advancing Treatments for Rare Diseases Requires a Team Approach

Clinical and industry leaders share their perspectives on the importance of collaboration in developing treatments for rare diseases for Monday, February 28, 2022, which was Rare Disease Day.

2. Developing Therapies for Rare Diseases: Kinnari Patel, PharmD

The president and chief operating officer of Rocket Pharmaceuticals discussed the importance of Rare Disease Day. She collaboration between companies, academia, and patient advocacy groups and how it could be improved moving forward.

3. Around the Helix: Cell and Gene Therapy Company Updates – March 2, 2022

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

4. CRISPR Patents Decision Favors Harvard and MIT’s Broad Institute

In the latest line of CRISPR patent interferences, the US Patent and Trademark Office (USPTO) Patent Trial Appeal Board (PTAB) has ruled that the Broad Institute of Harvard University and the Massachusetts Institute of Technology were the first to invent CRISPR/Cas9 editing in eukaryotic cells.

5. New Findings May Help Bolster CRISPR/Cas9 Accuracy

Researchers at The University of Texas Austin have identified regions in Cas9 that allow mismatches in DNA binding which interferes with on-target activation of the enzyme in CRISPR/Cas9 editing, according to a recent paper published in Nature. Their research may inform the design of future next-generation CRISPR/Cas9 variants.