Utilizing CRISPR Technology to Develop Cell Therapies for Diabetes, Blood Diseases


Both Vertex and Arbor are adding to their list of gene and cell therapy partnerships with the announcement.

Vertex Pharmaceuticals and Arbor Biotechnologies have announced a partnership to develop novel ex vivo engineered cell therapies.1

As part of the partnership, Vertex will use Arbor's proprietary CRISPR gene-editing technology to research and develop cell therapies to produce fully differentiated, insulin-producing hypoimmune islet cells for the treatment of type 1 diabetes as well as next-generation cell therapy approaches for the treatment of sickle cell disease (SCD) and beta thalassemia.

"This new collaboration further expands our toolkit in cell and genetic therapies and, specifically, our work to discover and develop cell therapies for the treatment of multiple serious diseases," said Bastiano Sanna, PhD, executive vice president and chief, cell and genetic therapies, Vertex, in a statement.1 "We are excited to bring Arbor's technology together with Vertex's ongoing programs and capabilities in diabetes, hemoglobinopathies and other diseases to create improved cell replacement therapies for broad populations of patients."

Arbor has been using their CRISPR gene-editing technology to target different genetic diseases. In addition to the company’s partnership with Vertex, they also recently announced a licensing agreement for their gene-editing technology with Lonza in December 2020.2

READ MORE: New Collaboration to Evaluate Lower-Dose Gene Therapy Conditioning for Sickle Cell Disease

"Arbor and Vertex share a common goal to improve the lives of people with life-threatening diseases through the discovery and development of innovative therapies, and Vertex has proven to be an ideal partner in that effort," added Devyn Smith, PhD, chief executive officer, Arbor, to the statement.1 "This agreement demonstrates the versatility and strength of Arbor's platform and reinforces our strategic vision of expanding our engineered cell therapy capabilities through partnerships with leading organizations in our industry, while we focus our internal portfolio efforts on genetic medicines."

While Vertex’s main focus is currently on treating cystic fibrosis, for which they have multiple medicines approved and in development, they have research programs in pain, alpha-1 antitrypsin deficiency, and APOL1-mediated kidney diseases. They have also recently been expanding their pipeline of cell and genetic therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy, and type 1 diabetes.

Vertex is also collaborating with CRISPR Therapeutics to take the lead on worldwide development, manufacturing, and commercializing of CTX001, an agreement originally reached in 2015 but amended in April 2021.3 CTX001 is a CRISPR/Cas9-based gene editing therapy in development as a potentially curative therapy for SCD and transfusion-dependent beta-thalassemia (TDT).

The FDA has granted CTX001 regenerative medicine advanced therapy (RMAT), fast track, orphan drug, and rare pediatric disease designations for both TDT and SCD, based on early findings from 7 patients with at least 3 months of follow-up that showed increases in fetal hemoglobin production and total Hb overall. These data were presented at the 2021 Transplantation & Cellular Therapy (TCT) Meetings Digital Experience.

“Our increased investment in our partnership with CRISPR is based on the compelling clinical profile of CTX001, which shows its potential to be a durable cure for patients with SCD and TDT, and the rapid progress that we and our partners at CRISPR have made toward registration and commercialization," Jeffrey Leiden, MD, PhD, executive chairman of Vertex, said in a statement at that time.3 "We see a significant commercial opportunity for CTX001, and we believe we will be able to further enhance that opportunity by fully leveraging the breadth of Vertex’s capabilities – including our established and proven R&D and commercialization expertise in serious diseases – to bring CTX001 to more patients around the world, more quickly.”

1. Vertex and Arbor Biotechnologies establish new partnership to develop novel ex vivo engineered cell therapies. News release. Vertex Pharmaceuticals. August 24, 2021. https://www.benzinga.com/pressreleases/21/08/g22630920/vertex-and-arbor-biotechnologies-establish-new-partnership-to-develop-novel-ex-vivo-engineered-cel?utm_campaign=partner_feed&utm_source=yahooFinance&utm_medium=partner_feed&utm_content=site
2. Arbor Biotechnologies and Lonza enter into gene editing technology licensing deal. News release. Arbor Biotechnologies. December 23, 2020. Accessed August 26, 2021. https://www.globenewswire.com/en/news-release/2020/12/23/2149973/0/en/Arbor-Biotechnologies-and-Lonza-Enter-into-Gene-Editing-Technology-Licensing-Deal.html
3. Vertex Pharmaceuticals and CRISPR Therapeutics amend collaboration for development, manufacturing and commercialization of CTX001™ in sickle cell disease and beta thalassemia. News release. Vertex Pharmaceuticals. April 20, 2021. https://investors.vrtx.com/news-releases/news-release-details/vertex-pharmaceuticals-and-crispr-therapeutics-amend 
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