Around the Helix: Cell and Gene Therapy Company Updates – November 22, 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. MHRA Approves First CRISPR-Based Therapy Exa-cel for Sickle Cell and Transfusion-Dependent β-Thalassemia

The United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) has approved Vertex Pharmaceuticals and CRISPR Therapeutics’ autologous gene-edited cell therapy exagamglogene autotemcel (exa-cel), which is to be marketed under the name Casgevy, for the treatment of both sickle cell disease (SCD) and transfusion-dependent β-thalassemia in patients aged 12 years and older.

2. Sanfilippo Awareness Day: A Peek Into the CGT Pipeline

November 16 was World Sanfilippo Awareness Day so CGTLive™ took a look at the current investigational pipeline of disease-modifying therapies in development for Sanfilippo syndrome.

3. BMS and 2seventy’s PDUFA for Abecma in Earlier-Line Multiple Myeloma Pushed to Accommodate New Adcomm Meeting

The FDA’s Oncologic Drugs Advisory Committee (ODAC) will review data related to a supplemental biologics license application (sBLA) for idecabtagene vicleucel (ide-cel; Abecma; Bristol Myers Squibb, 2seventybio), a marketed BCMA-directed chimeric antigen receptor (CAR) T-cell therapy, in a newly announced advisory committee (AdComm) meeting. As a result of the AdComm meeting, which has not yet been scheduled for a specific date, the Prescription Drug User Fee Act (PDUFA) action date for the sBLA, which was originally scheduled for December 16, 2023, has been pushed back to an as yet unspecified later date.

4. BrainStorm to Meet With FDA on Confirmatory Trial for NurOwn ALS Therapy

BrainStorm Cell Therapeutics is meeting with the FDA to discuss a possible regulatory path forward for its NurOwn cell therapy for the potential treatment of amyotrophic lateral sclerosis (ALS) on December 6, 2023.

5. Cabaletta Bio’s CAR-T CABA-201 Receives Fourth IND Clearance, With New Trial Set for Myasthenia Gravis

Cabaletta Bio’s CABA-201, an investigational CD19-directed CAR T-cell therapy intended to treat autoimmune diseases, has received clearance from the FDA of its investigational new drug (IND) application for a clinical trial in generalized myasthenia gravis. Earlier in 2023, the CAR-T previously was cleared by the FDA for separate trials in systemic lupus erythematosus and lupus nephritis; active idiopathic inflammatory myopathy (also referred to as myositis); and systemic sclerosis.

6. Alkermes Spins Out New Company Mural Oncology

In order to focus its efforts entirely on neurological indications, Alkermes has spun off a new company, Mural Oncology, which will take on the development of engineered cytokine therapies directed at cancer indications. “Our protein engineering expertise allows us to reimagine the development of proinflammatory cytokine-based therapeutics that could address the key limitations with current cancer immunotherapies,” Caroline Loew, PhD, Mural’s chief executive officer, said in a statement. “I am thrilled to lead this company and am energized by the enormous potential of our lead clinical candidate, our preclinical programs, and our underlying protein engineering capabilities.”

7. Publicly-Traded Gene-Editing Company Graphite Bio Strategically Agrees to Merger With Privately-Owned Lenz Therapeutics

The newly combined and publicly traded company will focus on the development of Lenz’s pipeline of eye drops for the management of presbyopia. Graphite Bio had previously been developing a gene-edited cell therapy for the treatment of SCD but dropped its development following an unexpected serious adverse event that occurred in a phase 1/2 clinical trial.

8. Century Therapeutics and Fujifilm Cellular Dynamics Broaden Their Collaboration to Autoimmune Diseases

The 2 companies, which have been collaborating on the development of induced pluripotent stem cell (iPSC)-derived cell therapy products for cancer indications since 2018, have agreed to expand their collaboration and licensing activities to cover the development of iPSC-derived cell therapies for autoimmune indications such as type 1 diabetes, multiple sclerosis, lupus, and rheumatoid arthritis.

9. CDMO GenScript Probio Agrees to Take On Manufacturing Of Viral Vectors for Curocell’s CAR-T Therapies

Under a new memorandum of understanding, GenScript ProBio, a contract development and manufacturing organization (CDMO), and Curocell have expanded their preexisting collaboration to include the former company taking on manufacturing responsibilities for viral vectors for Cruocell’s investigational CAR-T therapies. The new agreement will help to “establish a stable cooperative relationship spanning the entire process, from the developmental stage to the commercialization of CAR-T therapy,” Gunsoo Kim, the CEO of Curocell, said in a statement.

10. Smart Immune Makes Progress in Series A Financing

Smart Immune, a French biotech developing T-cell progenitor cell therapy SMART-100 for acute leukemia, has crossed the halfway point on its path to garnering $54 million in a Series A round of financing, according to a report from Endpoints News. The round of financing, which will support clinical development of SMART-100 in multiple indications, is expected to reach its end goal early next year.