Cell therapy weekly: US$62 million for Garuda’s off-the-shelf blood stem cell technology platform
This week: Garuda Therapeutics receives US$62 million for off-the-shelf blood stem cell technology platform, RMAT designation granted to AAV-based gene therapy for fatal cardiac disease and IRB approval for cell therapy to treat Type 1 Diabetes.
The news highlights:
Garuda Therapeutics receives US$62 million for off-the-shelf blood stem cell technology platform
Garuda Therapeutics (MA, USA) has secured US$62 million in Series B financing, which will support the continuing development of the company’s technology platform generating off-the-shelf, self-renewing blood stem cell therapies for hematological diseases. In addition to this, Garuda aims to provide potential treatments for patients with oncologic disorders by employing this funding to further the advancement of its proprietary HSC-derived off-the-shelf, durable immune cell program. This round of financing brings the total raised by the company to US$134 million.
Dhvanit Shah, Co-Founder and CEO of Garuda Therapeutics stated: “Garuda is built upon the foundation of what we consider to be the most de-risked and highly validated blood stem cell-based cell therapy approach, and has the potential to overcome challenges to the current standard of care, including lack of consistency, scalability, durability, affordability, and availability of suitable healthy donors. We are grateful for the continued strong financial support from our elite institutional investors that will help enable us to bring our off-the-shelf durable blood stem cell and immune cell programs to patients in need.”
RMAT designation granted to AAV-based gene therapy for fatal cardiac disease
Rocket Pharmaceuticals (NJ, USA) has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the US FDA for the company’s investigational AAV-based gene therapy, RP-A501, for the treatment of Danon Disease. There are no disease-altering therapies available for this fatal cardiac disease and the only treatment option is cardiac transplantation. The RMAT designation is designed to expedite the development and review process of therapies and was granted to RP-A501 following encouraging preliminary safety and efficacy data from a Phase I clinical trial. RP-A501 is also the first cardiac gene therapy to receive RMAT designation from the FDA.
Gaurav Shah, CEO of Rocket Pharma stated: “Today’s exciting RMAT designation demonstrates recognition from the FDA of the early meaningful benefit of RP-A501 in Danon Disease and its potential to deliver lifesaving treatment for patients. We look forward to the FDA’s added guidance and support on the most efficient development and approval pathway for RP-A501, including on our anticipated Phase 2 pivotal trial with the opportunity for accelerated approval.”
IRB approval for cell therapy to treat Type 1 Diabetes
Creative Medical Technology Holdings, Inc. (AZ, USA), a biotechnology company aiming to improve patient lives with best-in-class regenerative therapeutics, has been granted Institutional Review Board (IRB) approval by the US FDA to continue its clinical trial for the treatment of Type 1 Diabetes with the company’s cell therapy, CELZ-201. The trial will investigate CELZ-201 as a treatment for patients with recent onset Type 1 Diabetes.
Timothy Warbington, CEO of Creative Medical Technology Holdings, Inc. stated: “The purpose of IRB review and approval is to assure that appropriate steps are taken to protect the rights and welfare of patients participating as subjects in the research and is an important milestone in proceeding with a clinical trial. We are pleased with achieving IRB approval expeditiously and look forward to moving forward with our Phase 1/2 clinical trial.”
Principal Investigator, Camillo Ricordi also commented: “I am excited to proceed with the CELZ-201 perinatal cell product in this study, as I believe that if successful it could result in a promising treatment for many patients with Type 1 Diabetes.”