Around the Helix: Cell and Gene Therapy Company Updates – June 22, 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. IND Cleared for γδ T-Cell Therapy in Non-Hodgkin Lymphoma

The FDA has cleared the investigational new drug application (IND) for Acepodia’s ACE1831, a γδ T-cell therapy, for the potential treatment of non-Hodgkin lymphoma, which will soon be evaluated in a phase 1, first-in-human clinical trial.

2. Children Treated With Zolgensma for Presymptomatic SMA Achieve Age-Appropriate Motor Milestones

Novartis’ onasemnogene abeparvovec was shown to be both effective and well-tolerated for children expected to develop spinal muscular atrophy type 1 or type 2, according to findings from SPR1NT (NCT03505099), a phase 3 multicenter, single-arm trial.

3. Vutrisiran Approved for hATTR Amyloidosis

The FDA has approved Alnylam’s investigational subcutaneous RNA interface therapy vutrisiran, marketed as Amvuttra, for the treatment of transthyretin-mediated (ATTR) amyloidosis, based on positive 9-month results from the phase 3 HELIOS-A study (NCT03759379).

4. Collaboration to Focus on CAR T Therapy in Solid Tumors

MD Anderson Cancer Center, Invectys, and the Cell Therapy Manufacturing Center, which is a joint venture between MD Anderson and National Resilience, are collaborating to develop a scalable process for human leukocyte antigen-G targeted CAR T cell therapies for the potential treatment of solid tumors.

5. Akadeum Gets Patent for Cell-Sorting Technology

Akadeum Life Sciences has been granted a patent for their proprietary method of buoyancy-based cell sorting from biological fluid samples. Notably, the patent applies to the company’sT and B Cell isolation kits used in cell therapy manufacturing.

6. Partnership to Advance Gene-Editing Cell Therapy in Solid Tumors

Affini-T Therapeutics is combining their T-cell expertise with Metagenomi’s gene-editing technologies to create ex vivo T cell receptor therapies for solid tumors.

7. NorthX Biologics Opens Cell Therapy Manufacturing

The company will begin to manufacture cell therapies at their existing facilities as well as at the Karolinska University Hospital campus in Stockholm, Sweden. NorthX is also partnering with Alder Therapeutics, a cell therapy company.

8. OrganaBio Launches Cell Therapy Manufacturing

The company has launched contract operations at its new cell therapy manufacturing facility.

9. Invetech’s Korus to Improve Cell Elutriation

Korus is a closed system for autologous cell therapies that uses gentle counterflow centrifugation to improve elutriation and cell wash. So far, data indicate improved manufacturing performance compared to standard washing.

10. New Startup Immuneel Launches CAR T Trial

The startup has launched with $15 million and has started dosing patients in the phase 2 IMAGINE trial of ARI-0001 in patients with acute lymphoid leukemia. The trial is India's first CAR T-cell therapy trial.

11. EMA Validates Breyanzi Application

The EMA has validated Bristol Myers Squibb’s type II variation application to extend lisocabtagene maraleucel’s indication to include patients with diffuse large B-cell lymphoma, high grade B-cell lymphoma, primary mediastinal large B-cell lymphoma and follicular lymphoma grade 3B, who are refractory or have relapsed within 12 months of initial therapy and are candidates for hematopoietic stem cell transplant.

12. Rochester and Sana Partner to Develop Neurologic Cell Therapies

The partnership builds on existing collaboration between University of Rochester Medical Center and Sana Biotechnology and will support the development of cell therapies for possible indications including Huntington disease, Pelizaeus-Merzbacher disease, and secondary progressive multiple sclerosis.

13. Verismo and UPenn Enter Research Partnership

Verismo will fund additional research focused on the novel KIR-CAR platform for CAR T-cells to advance treatment options for solid tumors under a Sponsored Research Agreement.

14. Carbon Biosciences Launches With Cystic Fibrosis Gene Therapy Program

Carbon has launched with $38 million of funding, including $6 million from the Cystic Fibrosis Foundation. It’s lead program is CGT-001, a gene therapy for the potential treatment of cystic fibrosis developed with the company’s novel platform.

15. Novartis and Precision Bio Collaborate on Gene Editing

The partnership is targeting hemoglobinopathies such as sickle-cell disease and β-thalassemia. Therapies will be developed with Precision's ARCUS genome editing platform.

16. Kiromic Prioritizes γδ T-Cell Therapy

Kiromic Biopharma has shifted to prioritize its allogeneic, non-engineered off-the-shelfγδ T-cell therapy candidate, Deltacel (KB-GDT) for solid tumors. The company expects to submit an IND in the second half of 2022.

17. Lifespan Cancer Institute Gets Stem Cell Therapy Approval

The institute located at Rhode Island Hospital will now be able to treat patients with cancer with stem cell therapies.

18. Galapagos’ Acquisitions Expand Cell Therapy Operations

Galapagos NV is set to acquire CellPoint and AboundBio, giving the company access to new platforms and cell therapy models.