Around the Helix: Cell and Gene Therapy Company Updates – June 1, 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. BioMarin’s Val-Rox for Hemophilia A Delayed Again

The FDA has requested additional durability and safety data for the Biologics License Application (BLA) of BioMarin’s hemophilia A gene therapy program, valoctocogene roxaparvovec (val-rox; Roctavian), delaying filing to September 2022 instead of June.

2. FDA Approves Tisa-Cel Expanded Indication in R/R Follicular Lymphoma

The FDA has granted accelerated approval to tisagenlecleucel (Kymriah) for the treatment of adult patients with relapsed or refractory follicular lymphoma following 2 or more lines of systemic therapy based on data from the phase 2 ELARA clinical trial.

3. Iovance’s Lifileucel Shows Promising Data in Advanced Melanoma

Iovance Biotherapeutics’ tumor infiltrating lymphocyte therapy (TIL) lifileucel (LN-144) has shown promising clinical results in patients with advanced melanoma, according to updated data from cohort 4 of the C-144-01 clinical study (NCT02360579).

4. Liso-Cel Yields High Response Rates and Positive PROs in LBCL

Participants with large B-cell lymphoma (LBCL)in the phase 2 TRANSCEND-PILOT study (NCT03483103) treated with lisocabtagene maraleucel (Breyanzi; Bristol Myers Squibb) had significant improvements in fatigue and lymphoma symptoms.

5. FDA Lifts Clinical Hold on Legend’s CAR T Therapy for T-Cell Lymphoma

The FDA lifted its hold of Legend Biotech’s phase 1 clinical trial of LB1901, a CD4+ autologous chimeric antigen receptor (CAR) T-cell therapy for the treatment of relapsed or refractory T-cell lymphoma (TCL).

6. First Patient With Advanced Liver Cancer Treated with ARTEMIS T-Cells

Eureka Therapeutics and City of Hope have treated the first patient with GPC3-positive advanced hepatocellular carcinoma with the ARTEMIS T-cell therapy ECT204 as part of a phase 1/2 study (NCT04864054).

7. Athenex Partners With GenScript ProBio

The partnership with the Contract Development and Manufacturing Organization founded in 2020 builds off of the existing relationship between Athenex and GenScript to support the clinical development of cell therapies.

8. BlueRock Completes Enrollment of Cell Therapy Trial in Parkinson Disease

BlueRock Therapeutics, a subsidiary of Bayer AG, has completed enrollment in their phase 1 trial (NCT04802733) of BRT-DA01, a pluripotent stem cell-derived dopaminergic neuron therapy.

9. IND Cleared for TScan’s TCR T Therapy for Hematologic Malignancies

TScan Therapeutics’ TSC-101 will be evaluated in patients with hematologic malignancies who are undergoing allogeneic hematopoietic cell transplantation in a phase 1 trial that includes an arm treated with TSC-100, another T-cell receptor engineered T cell therapy whose IND was previously cleared.

10. Evotec Expands Cell Therapy Manufacturing Capabilities

Evotec has acquired Rigenerand, a cell technology company in the field of Current Good Manufacturing Practice manufacturing of cell therapies. In so doing, they have expanded their expertise and capability in cell manufacturing with their EVOcells platform.

11. Triumvira to Digitalize Cell Manufacturing With L7 Agreement

TriumviraImmunologics will implement the L7|ESP platform to digitalize biologic operations including research, development, and manufacturing of their allogeneic T-cell therapies.

12. Bone Therapeutics Secures Funding for Allogeneic Bone Cell Therapy

Bone has signed an agreement with ABO Securities for a maximum EUR 5 million convertible bonds to advance the clinical development of its allogeneic mesenchymal stem cell therapy, ALLOB, for the treatment of difficult tibial fractures.