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First Patient Receives CRISPR Gene-Edited Cell Replacement Therapy as a Novel Treatment for Type 1 Diabetes

Anna Osborne
8 February 2022
Biopharmaceutical company, CRISPR Theraputics, and clinical-stage regenerative medicine company, ViaCyte, have announced that the first patient in their Phase I clinical trial for the treatment of Type 1 Diabetes (T1D) has been dosed with CRISPR therapeutic: VCTX210.

CRISPR Theraputics has an established portfolio for their innovative gene-editing platform: CRISPR/Cas9, in pipeline treatments for a plethora of serious diseases. Focused on translating this technology into breakthrough therapeutics targeting underling genetic defects, their co-development and co-commercialisation collaboration with ViaCyte strives to provide the first gene-edited cell replacement therapy to treat T1D.

The investigational product VCTX210 was developed through compounding CRISPR technology with ViaCyte’s proprietary iPSC expertise, generating gene-edited pancreatic islet cells designed to evade immune system recognition. Successfully avoiding immune system attack means the allogenic cells can then allow the patient to produce their own insulin.

“The combination of ViaCyte’s leading stem cell capabilities and CRISPR Therapeutics’ pre-eminent gene-editing platform has the potential to meaningfully impact the lives of patients living with T1D,” commented Samarth Kulkarni, CEO at CRISPR Therapeutics.

This strategic collaboration accelerates a potential functional cure to T1D patients that doesn’t require the current need for immunosuppression. CRISPR/Cas9 targets the genome of iPSC derived islet cells to precisely edits the genomic DNA, producing an immune-evasive beta-cell replacement product.

The requirement for concurrent immunosuppressant treatment means that present donor islet transplantation is scarcely performed and isn’t a durable treatment for T1D. Limitations also arise from the lack of donor islet cell material.

VCTX210 holds many advantages: it can be mass-manufactured to provide a potentially unlimited supply of quality-controlled islet cells, which are safer and more reliable, and it also eliminates the need for immunosuppression, reducing probable patient side effects.

The hope for VCTX201 to facilitate glucose control in patients with type 1 diabetes, reducing the risk of hypoglycaemia and diabetes-related complications, presents it as a promising option to broaden future T1D implantation treatment, pending clinical results.

This initial Phase I clinical trial will assess VCTX210’s immune evasion, tolerability and safety in patients with T1D.

“Being first into the clinic with a gene-edited, immune-evasive cell therapy to treat patients with type 1 diabetes is breaking new ground as it sets a path to potentially broadening the treatable population by eliminating the need for immunosuppression with implanted cell therapies,” noted Michael Yang, President and CEO at ViaCyte.

Source: CRISPR Theraputics press releases – 2nd February 2022, 16th November 2021 and ViaCyte pipe-line for insulin-requiring diabetes