GeneTherapyLive’s Weekly Rewind – November 26, 2021

Review top news and interview highlights from the week ending November 26, 2021.

Developing Gene Therapies for Indications With Unmet Needs

The chief executive and scientific officers of Lysogene discussed Lysogene’s patient-focused therapy development.

CD19+ B-Cell Malignancy Cell Therapy Receives RMAT Designation

In 26 patients with LBCL followed for at least 28 days, the objective response rate (ORR) was 58% with complete responses (CRs) in 38% of patients.

Advantages of Developing Platform-Agnostic Gene Therapies

The chief executive officer of Rocket Pharmaceuticals, Gaurav Shah, MD, discussed the company’s ex-vivo and in-vivo gene therapy platforms.

Hemophilia A Gene Therapy Placed on Clinical Hold

Pfizer and Sangamo voluntarily paused the trial after participants experienced FVIII activity levels of over 150%.

Targeting the Putamen in AADC Deficiency: Paul Wuh-Liang Hwu, MD, PhD

The professor from National Taiwan University Hospital discussed the benefits of delivering gene therapy directly to the putamen in AADC deficiency.