CGTLive®’s Weekly Rewind – April 12, 2024

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Review top news and interview highlights from the week ending April 12, 2024.

CGTLive®’s Weekly Rewind - April 12, 2024.

CGTLive®’s Weekly Rewind

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. BrainStorm Reaches Accord With FDA on New Phase 3b Trial Design for ALS Cell Therapy NurOwn

The 2-part, multicenter BCT-006-US trial will seek to enroll patients who are in the earlier stages of ALS.

2. Frederick “Eric” Arnold, PhD, on Investigating MARK3 Polyadenylation and Microtubule Dynamics in ALS Models

The postdoctoral scholar at University of California – Irvine discussed further questions he is continuing to investigate.

3. CAR TEAM Cells Show Promise in Multiple Preclinical Models of Pancreatic Cancer

The cells showed activity toward PDAC cells as well as cancer-associated fibroblasts, which may address challenges with the tumor microenvironment.

4. Amar Kelkar, MD, on Assessing Price and Value for Cell and Gene Therapies

The stem cell transplantation physician at the Dana-Farber Cancer Institute discussed how to look at the value therapies provide and how to improve the cost-effectiveness of their use.

5. Aurion Biotech’s ABA-1 Clinical Trial for Corneal Edema Cell Therapy AURN001 Doses First Canadian Patient

AURN001 consists of allogeneic human corneal endothelial cells referred to as “neltependocel” and Y-27632, a small molecule drug.

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Sekar Kethiresan, MD, on Following up VERVE-101 With Next-Generation Editing Therapies
Maria Escolar, MD, the chief medical officer of Forge Biologics
Leigh Ramos-Platt, MD, on Allowing Access and Ensuring Preparation for Gene Therapies
John Murphy, PhD, the chief scientific officer of Arbor Biotechnologies
Erika Fullwood Augustine, MD, MS, the associate chief science officer of the Kennedy Krieger Institute
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Casey Maguire, PhD, associate professor of neurology and associate investigator of neurology, Harvard Medical School
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