Clinical Trial

Disease: Type 1 Diabetes, T1D (NCT05210530)

Disease info:

Type 1 diabetes (T1D) is an autoimmune disease that results in destruction of the insulin-producing beta cells of the pancreas. The resuling insulin deficiency leads to rising blood glucose levels that over time can lead to a range of complications that include the heart, eyes, kidneys, nerves, and gums and teeth.

T1D may debut at any age but it usually begins in childhood or early adulthood, and is fatal if untreated. The standard current treatment is regular blood glucose monitoring and subcutaneous administration of modified human insulin expressed in heterologous hosts, e.g., E. coli.

Frequency:
Approximately 1 out of every 300 individuals will develop type 1 diabetes by the age of 18 in the United States and Europe. The rate is much lower in Asia and South America with only 1 in 1 million new cases per year.
Official title:
An Open-Label, First-In-Human Study Evaluating the Safety and Tolerability of VCTX210A Combination Product in Subjects With Type 1 Diabetes Mellitus (T1D)
Who:

Investigators: Manasi Jaiman, Sandeep Soni

Partners:
Locations:

Canada, Alberta

Canada, British Columbia

Canada, Ontario

Study start:
Jan. 30, 2022
Enrollment:
10
Gene editing method:
CRISPR-Cas9
Type of edit:
Disruption and insertion
Gene:
Delivery method:
- Ex-vivo
Indicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active recruiting

Description

VCTX210A combination product (unit) comprises 2 components: (1) allogeneic pancreatic endoderm cells (PEC210A) genetically modified using Clustered Regularly Interspaced Short Palindromic Repeats/CRISPR-associated protein 9 (CRISPR/Cas9) to promote immune evasiveness and survival, and (2) a durable, removable, perforated device designed to deliver and retain the PEC210A cells.

Last updated: Apr. 8, 2022
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