Clinical Trial
Open access until Aug. 11, 2025

Disease: Type 1 Diabetes, T1D, (NCT06239636)

Disease info:

Diabetes is a chronic disease that affects the bodies ability to metabolise food. Individuals with diabetes fail to make sifficient insulin, or cannnot utilise insulin made in the body efficiently to break down sugar.

Type 1 diabetes (T1D) is an autoimmune disease that results in destruction of the insulin-producing beta cells of the pancreas. The resulting insulin deficiency leads to rising blood glucose levels that over time can lead to a range of complications involving the heart, eyes, kidneys, nerves, gums and teeth. Approximately 5-10% of diebetes patients have type 1.

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

The exact genetic causes of T1D are currently unknown, as the disease is multifactorial, but several risk factors have been identified, with certain variants of the HLA-DQA1HLA-DQB1, and HLA-DRB1 genes being associated with the disease. 

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:
First-in-human Safety Study of Hypoimmune Pancreatic Islet Transplantation in Adult Subjects With Type 1 Diabetes
Who:

Name: Per-Ola Carlsson, MD, PhD

Phone: +46 18 4710000 ext 4425

Email: per-ola.carlsson@mcb.uu.se

Sponsor:

Per-Ola Carlsson

Partners:

Sana Biotechnology
 

Locations:

Uppsala, Sweden

Uppsala University Hospital, Uppsala, Sweden, 75185

Study start:
Mar. 8, 2024
Enrollment:
2 participants
Gene editing method:
CRISPR-Cas12b
Type of edit:
Gene depletion and gene overexpression
Gene:
Depletion of HLA class I and II overexpression of CD47
Delivery method:
Lentivirus - Ex-vivo
Indicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active recruiting

Description

The current study tests the hypothesis whether genetically modified Langerhans islet cells containing insulin-producing cells from a deceased organ donor can

be transplanted safely and
help to regain insulin production in individuals with type 1 diabetes without need in simultaneous treatment with immunosuppressive medicines.
The study is an open, one-armed study where adult subjects with longstanding type 1 diabetes will receive transplantation of Langerhans islet cells (25 000 000-80 000 000) into forearm muscle. Both subjects receive active treatment. Safety is monitored with frquent follow-up visits over a year, including medical examinations, blood tests and MRI scans. Insluin producing cell function is monitored with blood samples and continuous glucose measurement.

Main objective is to to investigate the safety of an intramuscular transplantation of genetically modified allogeneic human islets (study product UP421) in adult subjects diagnosed with type 1 diabetes.

Secondary objectives are to study changes in beta-cell function, metabolic control and immunological response to pancreatic islets during the first year following treatment.

Last updated: Aug. 8, 2025
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