Around the Helix: Cell and Gene Therapy Company Updates – September 21, 2022

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. Eli-Cel Approved for CALD, Marking Second Gene Therapy Win for bluebird bio

The FDA has granted accelerated approval to bluebird bio's elivaldogene autotemcel (eli-cel; Lenti-D) for the treatment of early, active cerebral adrenoleukodystrophy (CALD) in patients without an HLA-matched donor.

2. Gene Therapy Trial for Stargardt Disease Reaches Full Enrollment

Nanoscope Therapeutics Inc. announced full enrollment of its Phase 2 clinical trial of MCO-010, an ambient-light activatable Multi-Characteristic Opsin (MCO) optogenetic monotherapy to restore vision in blind patients, for Stargardt disease.

3. ICER Throws Support Behind Clinical, Cost Value of Hemophilia Gene Therapies in Draft Report

The Institute for Clinical and Economic Review (ICER) has released a draft report comparing clinical benefit and cost effectiveness of CSL’s etranacogene dezaparvovec (EtranaDez), an investigational gene therapy intended for the treatment of hemophilia B, and BioMarin’s valoctocogene roxaparvovec (val-rox; BMN-270), a gene therapy for the treatment of hemophilia A, to current standards of care.

4. Real-World Study on Ide-Cel Shines Spotlight on Efficacy in Multiple Myeloma

A single-center study at the Dana-Farber Cancer Institute/Brigham and Women’s Hospital assessed the real-world experience of 20 patients treated with idecabtagene vicleucel (ide-cel; Abecma) with relapsed and refractory multiple myeloma (RRMM) who had exhausted at least 4 lines of prior therapy.

5. First Patient Dosed in Wet AMD Gene Therapy LUNA Trial

The first patient has been dosed in a phase 2 clinical trial (NCT05536973) of Adverum Biotechnologies’ ixoberogene soroparvovec (ixo-vec, ADVM-022), an investigational gene therapy intended for the treatment of wet age-related macular degeneration (wet AMD).

6. Intellia Nets Positive Data Across In Vivo CRISPR Therapies for ATTR Amyloidosis Cardiomyopathy, Angioedema

Intellia has reported positive interim data from 2 ongoing clinical trials examining their in vivo CRISPR/Cas9 gene editing therapies, one for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy and the other for hereditary angioedema (HAE).

7. Hunter Syndrome Gene Therapy Clinical Trial Cleared to Begin in the UK

A clinical trial application for AVROBIO’s AVR-RD-05, an investigational autologous hematopoietic stem cell (HSC) gene therapy intended for the treatment of neuronopathic mucopolysaccharidosis type II (nMPS-II; Hunter syndrome) being developed in a collaboration with University of Manchester (UoM), has been approved by the UK Medicines and Healthcare Products Regulatory Agency (MHRA), Research Ethics Committee (REC), and Health Research Authority (HRA).

8. Cilta-Cel Yields High MRD-Negativity Rates in Multiple Myeloma After Prior Anti-BCMA Treatment

Patients with heavily pretreated multiple myeloma who received prior treatment with a BCMA-targeted therapy experienced high rates of minimal residual disease (MRD) negativity after being treated with ciltacabtagene autoleucel (cilta-cel; Carvykti).

9. Cystinosis Gene Therapy Granted Rare Pediatric Disease Designation by FDA

AVROBIO’s AVR-RD-04, an investigational gene therapy intended for the treatment of cystinosis, has received rare pediatric disease designation from the FDA.

10. Post-transplant AML Cell Therapy Receives $2 Million FDA Grant

Marker Therapeutics has a received a $2 million grant from the FDA for the purpose of advancing the phase 2 ARTEMIS clinical trial (NCT04511130) for MT-401 (zelenoleucel), an investigational allogeneic multi-tumor-associated antigen (MultiTAA) T-cell therapy intended for the treatment of post-transplant acute myeloid leukemia (AML).

11. Artiva to Evaluate CAR-NK Therapy in Solid Tumors

The FDA has cleared Artiva Biotherapeutics’ investigational new drug (IND) application for the chimeric antigen receptor NK (CAR-NK) cell therapy AB-201 for the potential treatment of solid tumors.

12. Rubius Therapeutics Announces Restructuring, Discontinuation of 2 Clinical Trials

The company is discontinuting its phase 1 clinical trials for RTX-240 and RTX-224 and redirecting efforts towards developing its next generation red blood cell-based cell conjugation platform.

13. Head of Irish Government, Micheál Martin, Formally Introduces MeiraGTx's Gene Therapy Manufcaturing Facility

The facility, located in Shannon, Ireland, has been operational since earlier in 2022, is over 150,000 square feet, and contains facilities for viral vector production, plasmid DNA manufacturing, and quality control.

14. IN8bio Announces Manufacturing Partnership With Dunbar CAR T-cell Program

The the University of Louisville Health - Brown Cancer Center GMP Manufacturing Facility will serve as the manufacturing center for INB-400, a DeltEx allogeneic drug resistant immunotherapy platform (DRI) candidate intended to treat glioblastoma, which is expected to be investigated in a phase 2 clinical trial with the help of the partnership.

15. New Biotech Company Carver Biosciences to use Seed Funding for Cas13 Experiments

Carver Biosciences, founded in 2021, will explore bacterially-derived RNA-directed RNase Cas13 for use in the treatment of respiratory infections.

16. Rocket Pharmaceuticals Announces Upcoming Acquisition of Renovacor

A focal point of the acquisition is Renovacor's REN-001, an investigational AAV-based gene therapy for the treatment of BAG3-associated cardiomyopathy.

17. Non-viral Cell Therapy Manufacturing the Focus of New GenScript, Avectas Partnership

Avectas' SOLUPORE delivery platform and GenScript'sGenCRISPR sgRNA and ss/dsDNA HDR templates will be used to improve the non-viral cell therapy manufacturing process.

18. Cellistic Announces Upcoming Acquisition of Celyad Oncology’s Manufacturing Business Unit

The Manufacturing Business Unit consists of an 11,000 square foot cell therapy manufacturing facility, which Cellistic plans to optimize, and related personnel.

19. BioIVT Launches New Leukopak Product Line

The VivoSTART leukopaks are GMP-grade and are capable of use in both research and manufacturing.

20. TCR-Mimic Antibody-based Cell Therapy Focus of New Biocytogen, FineImmune Collaboration

Bioctogen's TCR-mimic antibody platform and FineImmune's cell therapy platform will be utilized in the development of therapies that target intracellular tumor-associated antigens.

21. Coeptis Therapeutics to Acquire Rights to "Multi-Antigen" CAR-T Technology from the University of Pittsburgh

The SNAP-CAR technology is designed to be adaptable to targeting different hematologic and solid tumor types and has demonstrated proof-of-concept in preclinical mouse studies.

22. Carisma Therapeutics Will Merge With Sesen Bio

The combined company will focus on furthering the development of Carisma's chimeric antigen receptor macrophage (CAR-M) therapies, which have shown proof-of-mechanism in clinical trials.