Around the Helix: Cell and Gene Therapy Company Updates – February 1, 2023

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

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.

1. Senti Bio Prioritizes Logic Gated Cell Therapies

Senti Bio will be prioritizing the development of its logic gated cell therapy candidates which are based on the company’s Gene Circuit technology, in particular SENTI-202 and SENTI-401, according to a corporate update.

2. Cilta-cel CARTITUDE-4 Trial to be Unblinded After Meeting Primary End Point

The phase 3 CARTITUDE-4 clinical trial (NCT04181827), which is evaluating Janssen’s and Legend Biotech's ciltacabtagene autoleucel (cilta-cel; CARVYKTI) against standards of care in the treatment of relapsed/refractory multiple myeloma, will be unblinded according to an independent data monitoring committee recommendation following the study meeting its primary end point in a pre-specified interim analysis.

3. Remestemcel-L Cell Therapy Makes Comeback With Resubmitted BLA for Pediatric GvHD

Mesoblast has resubmitted a biologics license application (BLA) for remestemcel-L, its mesenchymal stromal cell therapy for treating steroid-refractory acute graft versus host disease (SR-aGVHD) in children, almost 4 years after it first initiated a rolling BLA for the therapy.

4. Giant Axonal Neuropathy Gene Therapy Needs Double-blind Trial Before BLA Submission

The FDA has recommended Taysha Gene Therapies to dose additional patients with TSHA-120, an investigational adeno-associated virus (AAV) vector-based gene therapy intended to treat giant axonal neuropathy, in order to support a BLA submission.

5. Sensorion Collaborating With EVEON on an Injection System to Deliver Gene Therapies Into the Inner Ear

Sensorion's investigational gene therapy for otoferlin-related hearing loss, OTOF-GT, could potentially be among the therapies able to be delivered with such an injection system.

6. Spark Therapeutics, Neurochase to Collaborate on Improving AAV Vectors for CNS Delivery

Spark Therapeutics expects that Neurochase's technology will improve targeting of its AAV gene therapy candidates for delivery to neural structures via Convection Enhanced Delivery.

7. Kite, Arcellx Finalize Agreement to Jointly Advance and Commercialize R/R Multiple Myeloma CAR-T in the US

CART-ddBCMA is currently under evaluation in a pivotal phase 2 clinical trial.

8. iTOL-100 Platform Technology is the Focus of New Master Services Agreement Between iTolerance, Diabetes Research Institute

The preclinical platform technology is intended to enable the transplant of insulin producing stem cells without the need for life-long immunosuppression.

9. uniQure Acquires Global Development and Commercialization Rights to SOD1 ALS Gene Therapy from Apic Bio

APB-102, which is intended to treat superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS), recently received clearance of its IND application by the FDA.

10. Aptamer Group, BaseCure Therapeutics Announce Collaboration for Optimer-targeted Gene Therapies

The research will attempt to use optimer binders to target specific cell types for siRNA -based therapeutics.

11. Kytopen, Mirus Bio Announce Collaboration on Non-viral Approach to Genetic Engineering of Cells

The collaboration will seek to identify improved methods of delivering complex genetic material in the production of cell therapies.

12. Newly Launched Company Vector BioMed to Provide Preclinical, Clincal, and Commercial Lentiviral Vector Manufacturing Services

Viking Global Investors and Casdin Capital led the new CDMO's first round of funding.

13. BioCardia, CellProthera Extend Collaboration on Acute Myocardial Infarction Cell Therapy

CellProthera's ProtheraCytes is currently being evaluated in a phase 1/2b clinical trial with the use of BioCardia's Helix transendocardial biotherapeutic delivery system.

14. California Institute for Regenerative Medicine Grants $8 Million to Immusoft for MPS I Cell Therapy's Clinical Development

ISP-001, which has previously received orphan drug and rare pediatric disease designations from the FDA, will be the first ever engineered B-cell therapy to be evaluated in a clinical trial.

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George Tachas, PhD
Alexandra Gomez-Arteaga, MD
Pietro Genovese, PhD, the principal investigator at the Gene Therapy Program of Dana-Farber/Boston Children’s Cancer and Blood Disorder Center
Akshay Sharma, MBBS, a bone marrow transplant physician at St. Jude Children’s Research Hospital
Caspian Oliai, MD, MS, the medical director of the UCLA Bone Marrow Transplantation Stem Cell Processing Center
Frederick “Eric” Arnold, PhD
Genovefa (Zenia) Papanicolaou, MD, an infectious diseases specialist at Memorial Sloan Kettering Cancer Center
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