Around the Helix: Cell and Gene Therapy Company Updates – August 23, 2023

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. No AdComm Meeting for Sickle Cell Therapy Lovo-Cel

Bluebird bio has announced that the FDA will not be holding an advisory committee meeting prior to the Prescription Drug User Fee Act goal date of December 20, 2023, for the company’s gene-edited cell therapy lovotibeglogene autotemcel (lovo-cel) for the treatment of patients with sickle cell disease aged 12 or older who have a history of vaso-occlusive events.

2. ImmPACT Bio’s Systemic Lupus Erythematosus CAR-T Cleared for US Trial

ImmPACT Bio’s IMPT-514, an investigational bispecific CD19/CD20-directed chimeric antigen receptor T-cell (CAR-T) therapy, has received clearance of an investigational new drug (IND) application from the FDA for a clinical trial in patients with active, refractory systemic lupus erythematosus (SLE).

3. Ornithine Transcarbamylase Deficiency Gene Therapy Gets FDA Orphan Drug Designation

Bloomsbury Genetic Therapies’ BGT-OTCD, an investigational adeno-associated virus (AAV) vector-based gene therapy intended to treat ornithine transcarbamylase deficiency, has received an orphan drug designation from the FDA. BGT-OTCD, which is delivered via a single intravenous injection and utilizes the AAV-LK03 vector, with high specificity for the cells of the liver, is being developed in a collaboration between Bloomsbury and University College London.

4. Wugen’s NK Cell Therapy Trial Doses First Patient With AML

The first patient has been dosed in a first-in-human phase 1 clinical trial (NCT05470140) evaluating Wugen’s WU-NK-101, an investigational allogeneic natural killer cell therapy, for the treatment of acute myeloid leukemia (AML).

5. BlueRock Therapeutics Lets Go of Approximately 50 Employees

The subsidiary of Bayer will be reducing its workforce by about 12%, according to Endpoints News, which received a relevant statement from a company spokesperson. The layoffs are related to BlueRock narrowing its focus and redirecting its efforts mainly into its cell therapy programs for primary photoreceptor disease, Parkinson disease, and heart failure.

6. CARsgen and Moderna Throw Weight Behind CAR-T and Cancer Vaccine Combination Therapy

Under a new collaboration agreement, the 2 companies may carry out preclinical research and a potential clinical trial assessing a combination treatment consisting of CARsgen’s CT041, an investigational autologous Claudin18.2-directed CAR-T, and Moderna’s investigational Claudin18.2-encoding mRNA cancer vaccine. "CT041 is the most advanced solid tumor CAR-T in development (pivotal phase 2) and continues to show promise in treating gastric and pancreatic cancers... Attacking tumors with CAR T-cell therapy in combination with a cancer vaccine could potentially provide greater clinical benefit to patients," Zonghai Li, MD, PhD, the founder, chairman of the board, CEO, and chief scientific officer of CARsgen Therapeutics, said in statement.

7. Single-Cell Biology Research Solutions Company PhenomX to Become Wholly Owned Subsidiary of Bruker

PhenomX and the Bruker Corporation have entered an agreement for the latter to acquire PhenomX, which has been assessed to have a total equity value of approximately $108 million, in a deal expected to close before the end of the year. “The unique single-cell analysis platforms of PhenomeX are enabling researchers to more rapidly and precisely unlock new insights in functional cell biology research leading to important discoveries across the large and rapidly growing markets of antibody therapeutics, cell line development, cell therapy, and gene therapy,” Mark R. Munch, PhD, the president of the Bruker NANO Group, said in a statement.

8. Cabaletta Bio Expands Preexisting Agreements to Include CABA-201

Cabaletta Bio has licensed Oxford Biomedica’s LentiVector platform for use in its CD19-direced CAR-T program CABA-201, an extension of a preexisting license and supply agreement, and expanded its manufacturing agreement with WuXi Advanced Therapies to cover CABA-201's clinical trial supply. CABA-201 has received clearance of 2 IND applications for trials in SLE and myositis, respectively.