Cell therapy weekly: Gamida Cell closes US$25 million financing for stem cell therapy launch and NK product pipeline
This week: FDA approves Investigational New Drug application for mRNA therapy to treat cystic fibrosis, Gamida Cell closes US$25 million financing for stem cell therapy launch and NK product pipeline and Akron Bio appoints new CEO.
The news highlights:
FDA approves Investigational New Drug application for mRNA therapy to treat cystic fibrosis
Vertex Pharmaceuticals (MA, USA) reported that the FDA has cleared its Investigational New Drug application for its mRNA therapy designed to treat the underlying cause of cystic fibrosis (CF). The therapy, VX-522, was developed for people with CF who are not eligible for treatment with an existing cystic fibrosis transmembrane conductance regulator modulator. Once delivered to the target lung cells, the mRNA is designed to produce functional copies of the cystic fibrosis transmembrane conductance regulator protein. VX-522 is the result of an exclusive research collaboration established with Moderna in 2016. In the coming weeks, Vertex intends to initiate a single ascending dose clinical trial for VX-522 in people with CF.
Reshma Kewalramani, CEO and President of Vertex, stated: “It has been our longstanding goal to bring highly effective therapies to all people with CF. Clearance of the [Investigational New Drug application] represents a pivotal turning point in reaching the remaining ~5,000 people with CF who are still waiting for a medicine to treat the underlying cause of their disease. The partnership with Moderna that began more than five years ago has been instrumental in achieving this milestone, and we look forward to continuing our work together.”
Gamida Cell closes US25 million financing for stem cell therapy launch and NK product pipeline
Gamida Cell (MA, USA) has closed a senior secured convertible term loan of US$25 million managed by Highbridge Capital Management, LLC. The funds obtained will facilitate the launch of omidubicel, an advanced nicotinamide-enabled stem cell therapy candidate for patients with blood cancers. Gamida Cell will also employ the financing to continue the development of its NK product pipeline, including GDA-201, an innate NK cell immunotherapy candidate for the potential treatment of hematologic and solid tumors in combination with standard-of-care antibody therapies.
CEO of Gamida Cell, Abbey Jenkins, stated: “We are pleased to secure additional capital from an existing investor as we continue to prepare for the launch of omidubicel, which is pending FDA review. Omidubicel has the potential to address the unmet need for patients with hematologic malignancies in need of an allogeneic hematopoietic stem cell transplant. As we anticipate our shift from clinical to commercial stage, we are now in a stronger financial position to prepare for launch while continuing development of our promising NK pipeline, including our clinical-stage asset GDA-201.”
Akron Bio appoints new CEO
Akron Bio (FL, USA), a portfolio company of Arcline Investment Management (TN, USA) and a leading materials manufacturer and services provider for the cell and gene therapy industry, has appointed Christopher Murphy as CEO. Murphy has over 30 years of experience in advanced therapies and biological drug development and manufacturing. In his most recent role, Murphy was Vice President and General Manager of Viral Vector Services at Thermo Fisher Scientific (MA, USA).
Arcline commented: “Chris brings an exceptional track record of building and growing businesses that address critical demands in the rapidly developing cell and gene therapy industry. We are thrilled to welcome Chris as Akron’s CEO and believe his passion and expertise will be invaluable in strengthening the company’s position as a leading supplier to cell and gene therapy companies.”