Cell therapy weekly: Priority Review for bioengineered vascular replacement
This week: Clinical trials for a T-cell receptor-engineered T (TCR T) cell therapy for human papillomavirus (HPV)-associated solid tumors will commence following Investigational New Drug approval, the US Food and Drug Administration (FDA; MD, USA) has granted Priority Review for a bioengineered vascular replacement and approval of the Biologics License Application (BLA) for the gene therapy KRESLADI has been delayed.
The news highlights:
- Clinical trials to commence for HPV-specific TCR T-cell therapy
- Priority Review for bioengineered vascular replacement
- Biologics License Application delayed for immunodeficiency gene therapy
Clinical trials to commence for HPV-specific TCR T-cell therapy
SCG Cell Therapy (Singapore) has announced it has received Investigational New Drug approval from the FDA, granting the company permission to commence multi-center Phase I/II clinical trials investigating SCG142, a T-cell receptor-engineered T (TCR T) cell therapy.
SCG142 is designed to specifically target human papillomavirus-associated solid tumors using a viral-specific TCR.
Ke Zhang, Chief Scientific Officer of SCG Cell Therapy explained: “SCG142 is a novel and differentiated HPV-specific TCR T cell therapy. By armoring the TCR T cells with the chimeric switch receptor, it overcomes the hostile tumor microenvironment and converts inhibitory effects into a co-stimulatory signal. This process is essential for effective immunotherapy treatment of solid tumors. With this unique next-generation design, SCG142 represents a groundbreaking innovation that translates from our in-house discovery platforms into clinics.”
Priority Review for bioengineered vascular replacement
The FDA has granted Priority Review to the BLA for Humacyte’s (NC, USA) off-the-shelf bioengineered vascular replacement called the Human Acellular Vessel (HAV). The BLA is seeking approval for the use of the HAV in emergency arterial repair following trauma when synthetic grafts are unsuitable and utilizing autologous is unfeasible.
CEO of Humacyte, Laura Niklason, stated: “We are very pleased that the FDA has accepted our BLA and has recognized the potential importance of the HAV technology by granting us Priority Review. The BLA acceptance brings us a major step closer to our goal of providing an innovative regenerative medicine product for patients suffering traumatic vascular injury. Many patients with severe injuries are underserved by the current standard of care, and we are proud of the results that have been seen in our clinical trials and real-world humanitarian efforts.”
Biologics License Application delayed for immunodeficiency gene therapy
In response to Rocket Pharmaceuticals’ (NJ, USA) BLA for the gene therapy KRESLADI, the FDA has issued a Complete Response Letter asking for additional chemistry, manufacturing and controls (CMC) information. CMC covers various procedures to establish the quality and consistency of a drug product during manufacturing. The FDA will use this additional CMC information to complete its review.
KRESLADI is designed to treat Severe Leukocyte Adhesion Deficiency-I, a rare pediatric immunodeficiency caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18. The investigational gene therapy comprises patient-derived hematopoietic stem cells that have been genetically modified to deliver a functional copy of ITGB2.
Gaurav Shah, CEO of Rocket Pharmaceuticals, stated: “It is reassuring to have the FDA as a close collaborator who understands the high unmet medical need, clear clinical benefit and importance of timely patient access. [The Center for Biologics Evaluation and Research] leadership’s direct involvement and commitment to working expeditiously to deliver this therapy to patients gives us great hope on behalf of the primary immunodeficiency community.”
