Around the Helix: Cell and Gene Therapy Company Updates – January 24, 2024


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

CGTLive Around the Helix

The cell and gene therapy sectors are growing exponentially, with new players emerging daily and much progress being made both in and out of the lab. CGTLive™’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more.

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1. FDA Requests Black Box Warning Be Added for All Currently Approved CAR-T Therapies

The FDA has requested that black box warnings related to secondary cancer risks be added to all 6 of the chimeric antigen receptor T-cell (CAR-T) therapy products currently approved by the agency for use in the United States.

2. Hemogenyx Sends the FDA a Complete Response Related to the IND Hold on AML CAR-T HEMO-CAR-T

Hemogenyx Pharmaceuticals has submitted a complete response to the FDA regarding the clinical hold that was placed on the company’s investigational new drug (IND) application for HEMO-CAR-T, an autologous CAR-T therapy developed with the use of Hemogenyx’s proprietary humanized monoclonal antibody against an acute myeloid leukemia target.

3. Experts Weigh In on Exa-cel's FDA Approval for Transfusion-Dependent Thalassemia

On January 16, 2024, Vertex Pharmaceuticals’ and CRISPR Therapeutics’ exagamglogene autotemcel (exa-cel; marketed as Casgevy), an autologous CRISPR-based gene-edited cell therapy, was approved by the FDA for the treatment of patients 12 years and older with transfusion-dependent beta thalassemia (TDT). In light of this major decision by the FDA, CGTLive™ decided to reach out to several experts to get their thoughts on how exa-cel could impact the landscape of care for TDT.

4. Autolus Therapeutics’ Obe-cel Will Be Reviewed by the FDA for the Treatment of R/R B-ALL

Autolus Therapeutics’ biologics license application (BLA) for obecabatagene autoleucel (obe-cel) in relapsed/refractory (r/r) adult B-Cell acute lymphoblastic leukemia (ALL) has been accepted for filing by the FDA, with a Prescription Drug User Fee Act date set for November 16, 2024.

5. BlueRock Licenses FUJIFILM Cellular Dynamics’ and Opsis Therapeutics’ Investigational iPSC Therapy

OpCT-001 is an induced pluripotent stem cell (iPSC) derived cell therapy currently in development for the treatment of primary photoreceptor diseases. BlueRock, which plans to file an IND for the therapy this year,optioned to exclusively license it under an existing research and development collaboration agreement established between the 3 companies in 2021.

6. Orchard Therapeutics Officially Becomes Part of Kyowa Kirin

Kyowa Kirin has stated that its previously-announced agreement to acquire Orchard Therapeutics has been completed, with Orchard now being a wholly-owned subsidiary of the Japanese pharmaceutical company. Orchard Therapeutics’ atidarsageneautotemcel (arsa-cel, Libmeldy) is a cell therapy for the treatment of metachromatic leukodystrophy that is approved in the European Union, UK, Iceland, Liechtenstein, and Norway, but remains an investigational therapy in the United States. Recently, the FDA accepted a biologics license application for arsa-cel with priority review.

7. GenEdit and Genentech Forge Alliance to Tackle Autoimmune Disease With Nucleic Acid-Based Approaches

GenEdit and Genentech have entered a collaboration in which the 2 companies will utilize GenEdit’s NanoGalaxy platform in discovery and development activities focused on hydrophilic nanoparticles for use in the delivery of Genentech’s nucleic acid-based medicines. "Genentech is a leader in advancing breakthrough science to advance the development of novel therapeutic modalities," Kunwoo Lee, PhD, the CEO of GenEdit, said in a statement. "This collaboration underscores the promise of GenEdit’s NanoGalaxy platform to deliver nucleic acid-based medicines via our hydrophilic nanoparticle technology.”

8. Blue Cross Blue Shield Will Provide Coverage for Sickle Cell Disease Gene Therapies

Blue Cross Blue Shield has become the first health insurance company in the United States to agree to provide coverage for the 2 sickle cell disease gene therapies recently approved by the FDA, Vertex Pharmaceuticals' and CRISPR Therapeutics’ exagamglogene autotemcel (exa-cel, marketed as Casgevy) and bluebird bio’s lovotibeglogene autotemcel (lovo-cel, marketed as Lyfgenia). The cost of both one-time treatments are in the range of millions of dollars.

9. Solvias to Handle Release Testing for Exa-cel in Sickle Cell Disease

Solvias, a chemistry, manufacturing, and control (CMC) analytics company, has inked an agreement with Vertex Pharmaceuticals to handle release testing activities for exa-cel, which is the first CRISPR-based gene therapy to have received FDA approval in the US. "Solvias is honored to be playing a critical role in delivering this breakthrough therapy to patients," Archie Cullen, BS, MBA, the CEO of Solvias, said in a statement.

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