Around the Helix: Cell and Gene Therapy Company Updates – August 10, 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. Allogeneic Cell Therapy Trial to Initiate in Ovarian Cancer

The FDA has cleared Avenge Bio’s investigational new drug application (IND) for AVB-001, an allogeneic cell therapy, for the potential treatment of metastatic peritoneal cancers.

2. Phase 2/3 Mesothelioma Cell Therapy Trial Completes Final Patient Active Follow-up

The last patient has completed the follow-up period for Amphera’s phase 2/3 clinical trial (DENIM; NCT03610360) of MesoPher, an autologous cell therapy being evaluated for the treatment of mesothelioma.

3. iPSC-Derived Allogeneic Cell Therapies to Be Focus of New Venture From ElevateBio, Boston Children’s

ElevateBio is partnering with George Daley, MD, PhD, and Boston Children’s Hospital with the intention of using a novel platform for developing cell therapies derived from induced pluripotent stem cells (iPSCs).

4. UniQure Pauses High Dose of AMT-130 Huntington Disease Gene Therapy for Severe Adverse Events

Following the report of promising 12-month data for the lower-dose cohort in the Phase 1/2 study of gene therapy AMT-130 for the treatment of Huntington disease, UniQure has announced that it has since paused enrollment in its higher-dose cohort due to the occurrence of suspected unexpected severe adverse reactions (SUSARs).

5. Sarepta, Broad Institute Strike Licensing Agreement for New Class of AAV Vectors for Gene Therapy

A licensing agreement has been executed between Sarepta Therapeutics and the Broad Institute of MIT and Harvard for Sarepta’s MyoAAV, a new group of adeno-associated viruses (AAV) for use in the treatment of Duchenne muscular dystrophy (DMD) and 4 other neuromuscular and cardiac indications.

6. IND Cleared for Non-Hodgkin Lymphoma T-Cell Therapy

The FDA has cleared Marker Therapeutics’ IND for MT-601, an investigative T-cell therapy for the treatment of relapsed/refractory non-Hodgkin lymphoma (NHL).

7. Allogeneic Cell Therapy for Multiple Hematologic Malignancies Being Assessed in Phase 3 Clinical Trial

Orca Bio has initiated its phase 3 clinical trial for Orca-T, an allogeneic cell therapy being evaluated for the treatment of acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL), and high-risk myelodysplastic syndromes (MDS).

8. RoslinCT and Lykan Bioscience to Form New CDMO Focused on Cell Therapy

The newly combined group will focus its efforts on providing cGMP manufacturing and process development expertise for both autologous and allogeneic cell therapies

9. Global Stem Cells Group Opens Facilities in Indonesia

The new facilities are being launched in a partnership with Dr. Yanti Aesthetic Clinics and will specialize in stem cell therapy and regenerative medicine.

10. NETs-targeted Adoptive Cell Therapies to be Focus of New Collaboration Between Xenetic Biosciences and VolitionRx

The collaboration will attempt to develop cell therapies for the treatment of solid cancers by combining Volition's Nu.Q NETs Test and Xenetic's DNase-Armored CAR-T platform.

11. Charles River Laboratories Receives EMA Approval to Manufacture Allogeneic Cell Therapy at Memphis Facility

The approval marks the first time a North American CDMO has been approved by the European Medicines Agency (EMA) to commercially produce an allogeneic cell therapy.

12. Merck's MilliporeSigma Launches Full Viral Vector Offering

The VirusExpress 293 AAV Production Platform will provide AAV, Lentiviral, CDMO, CTO, and process development to pharmaceutical companies.

13. Bristol Myers Squibb and GentiBio to Collaborate on Treg Therapies to Treat IBD

The companies will aim to use disease-specific engineered regulatory T cells (Tregs) for restoration of immune tolerance and tissue repair in patients with Inflammatory Bowl Diseases (IBD).