CGTLive’s Weekly Rewind – January 20, 2023


Review top news and interview highlights from the week ending January 20, 2023.

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. Late-Onset Pompe Disease Gene Therapy Cleared to Continue Trial

The FDA has lifted a clinical hold placed in the summer of 2022 due to a mild but medically significant case of peripheral sensory neuropathy.

2. Diane Simeone, MD, on Addressing Unmet Needs in Colorectal Cancer, NSCLC, and Pancreatic Cancer

Simeone discussed the design of BASECAMP-1, an observational study for patients previously treated for solid tumors who are at a high risk of relapse.

3. Editas Sells off iNK Preclinical Cell Therapy Program, Gene-Editing Technologies to Shoreline

The company is reprioritizing to focus on its clinical stage programs, which ran into a number of setbacks in 2022.

4. Akshay Sharma, MBBS, on Investigating CRISPR/Cas9 Edited Cells for Sickle Cell Disease

The assistant member of the bone marrow transplant department at St. Jude Children’s Research Hospital discussed advantages of OTQ923 in treating SCD.

5. EtranaDez “Transformative” for Patients With Hemophilia B

Steven Pipe, MD, CS Mott Children’s Hospital, discussed the latest follow-up data from the HOPE-B study of the approved Hemgenix therapy.

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