USA — Vertex Pharmaceuticals has inked a non-exclusive licensing deal with CRISPR Therapeutics to expedite the development of its hypo immune cell therapies for the treatment of type 1 diabetes (T1D).

Vertex and CRISPR Therapeutics will use CRISPR’s gene editing technology to develop new hypo immune cell therapies for Type 1 diabetes, aiming to generate fully differentiated, insulin-producing hypo immune islet cells that can evade the host immune system.

Vertex’s preclinical hypo immune stem-cell-derived islet cell assets will benefit from CRISPR’s Cas9 gene editing technology.

The technology involves cutting DNA and allowing natural DNA repair processes to take over.

In exchange for these rights, CRISPR Therapeutics will receive an upfront payment of US$100m, with the potential for additional payments of up to US$230m in research and development milestones, and royalties on future products.

Vertex Pharmaceuticals Cell and Genetic Therapies chief and executive vice-president, Bastiano Sanna, said that the agreement will deepen the company’s relationship with CRISPR Therapeutics, enabling it to generate fully differentiated, insulin-producing hypo immune islet cells for T1D.

Vertex’s clinical-stage diabetes pipeline is led by VTX-880, which utilizes stem-cell-derived islet cells coupled with standard immunosuppression.

The company released proof-of-concept data from one patient in October 2021, demonstrating restored islet cell function.

Recruitment for the second stage of trials is complete, and Vertex plans to release more data later this year.

Vertex acquired ViaCyte last year, which is collaborating with CRISPR to develop gene-edited allogeneic stem cell therapies for the treatment of diabetes called VCTX211.

Vertex and CRISPR have a longstanding collaboration that began in 2015 with a gene-editing deal targeting diseases such as cystic fibrosis.

In 2019, Vertex acquired Semma Therapeutics for US$950 million in cash, marking the company’s entry into the diabetes field.

The acquisition gave Vertex access to pancreatic islet cells that are now used in its two clinical-stage cell therapies for diabetes, VX-880 and VX-264.

While the administration of VX-880 requires immunosuppressive drugs, VX-264 utilizes a device to avoid the need for additional treatment.

By eliminating the need for immunosuppressants, Vertex believes that the use of the device could expand the patient population eligible for cell therapy.

Despite initial excitement over VX-880’s proof-of-concept patient data, the treatment has faced some setbacks during clinical trials.

In mid-2022, the FDA placed a two-month clinical hold on the medication due to concerns regarding the justification for increasing the dosage. However, Vertex reported that these concerns were resolved by July 2022.

The FDA cleared VX-264 therapy for a phase 1 trial earlier this month, and the company aims to launch the study in the first half of the year.

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