Around the Helix: Cell and Gene Therapy Company Updates – July 24, 2024

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.

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1. FDA Accepts Mesoblast’s BLA for Remestemcel-L for Steroid-Refractory GvHD

The FDA has accepted Mesoblast’s biologics license application (BLA) for remestemcel-L mesenchymal stromal cell therapy, to be marketed as Ryoncil, for the potential treatment of steroid refractory acute graft versus host disease (GvHD). The FDA has assigned a Prescription Drug User Fee Act goal date of January 7, 2025, for remestemcel-L.

2. Biotechs Seek Federal Coverage of Fertility Preservation Before Gene Therapy

The Office of the Inspector General (OIG) of the Department of Health and Human Services has provided an unfavorable opinion to bluebird bio’s attempt to secure Medicaid coverage for its fertility support program for patients receiving its gene therapies. The OIG explained its unfavorable opinion, stating that it did not have the necessary data to determine the program’s risk of fraud and abuse and therefore grant it immunity to the federal antikickback statute.

3. FDA Recognizes Lomecel-B Cell Therapy for Alzheimer Disease Ahead of New Data

The FDA has granted both Fast Track and Regenerative Medicine Advanced Therapeutic Designations to Longeveron's Lomecel-B investigational allogeneic medicinal signaling cell therapy for the potential treatment of patients with Alzheimer disease.

4. Stanford Study Finds Low Risk of Secondary Blood Cancers After CAR-T

A study from Stanford Medicine has found that the risk of secondary malignancies after chimeric antigen receptor (CAR) T-cell therapy is low in patients treated for hematological malignancies. The review is timely and adds valuable information to the field, as the FDA recently added a requirement for boxed warnings for T-cell malignancies after treatment with CAR T-cell therapies to all such approved therapies.

5. 4D Molecular Therapeutics’ Stock Falls After Announcement of Updated Data

The value of 4D Molecular Therapeutics' shares dipped by 32% after the company announced new data from the phase 1/2 PRISM clinical trial (NCT05197270) evaluating 4D-150, an investigational dual-mechanism gene therapy, for the treatment of wet age-related macular degeneration. Although the company presented the new efficacy and safety data as continuing the positive trend seen in earlier updates, analysts attributed the dip to investors reacting to a single case of grade 2 inflammation reported in the lower dose cohort.

6. Agilent Brings BIOVECTRA Into the Fold

Agilent Technologies has agreed to acquire the Canadian contract development and manufacturing organization BIOVECTRA in a deal worth $925 million. The acquisition will expand BIOVECTRA's ability to provide customers with products relevant to gene editing, such as gRNA. "BIOVECTRA's manufacturing capabilities further expand Agilent's end-to-end biopharma offerings into new growth vectors, including workflows that seamlessly integrate analytical instrumentation, consumables, and a wide range of lab services," Padraig McDonnell, BSc, the president and CEO of Agilent, said in a statement.

7. GC Cell and Lukas Join Forces for Cell Therapy Development

GC Cell, a subsidiary of Korean pharmaceutical group Green Cross Corporation, and Lukas Biomedical, which is based in Taiwan, have entered a new strategic cooperation agreement to share knowledge related to and collaborate on clinical trials for memory T-cell technology for oncology indications in Asia.

8. AbbVie Secures Time-Limited Reimbursement Recommendation from CDA for Epkinly

Canada's Drug Agency (CDA) has given a Time-Limited Reimbursement (TLR) recommendation to AbbVie for epcoritamab-bysp, its T-cell engaging bispecific antibody marketed as Epkinly, in relation to unmet need for adult patients with relapsed or refractory diffuse large B-cell lymphoma who have received at least 2 previous lines of systemic therapy and already received or cannot receive a CAR-T therapy. Notably, AbbVie is the first company to have received a TLR recommendation from the CDA. "EPKINLY is the first medication to be accepted through this process for its clinically meaningful phase 2 clinical data," Rami Fayed, BSc, the vice president and general manager of AbbVie Canada, said in a statement. "This early access pathway is a positive step forward that recognizes that time is of the essence for Canadian patients, especially those living with serious illnesses."