UNSW 100 Innovations Booklet | Page 67

100 + INNOVATIONS

Advanced Therapeutics for Type-1 Diabetes Cure

A single-dose cure for people newly diagnosed with type-1 diabetes
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Challenge
Type-1 diabetes affects ~ 140,000 Australians and millions worldwide. This autoimmune disease destroys insulinsecreting beta cells, causing lifelong insulin dependence and high blood sugar. Chronic T1D leads to severe complications, including blindness, kidney failure and amputations, costing Australia’ s healthcare system ~ AUD3.4B annually, representing a significant unmet burden.
Solution
The technology, BABS-beta- SMART-AAV-TNFAIP3, represents a cutting-edge advanced therapeutic with potential to cure type-1 diabetes. This is the first gene therapy to be wholly discovered and developed at UNSW, and within the Australian research ecosystem. It is comprised of a lead candidate gene to control the immune response, combined with cuttingedge advanced manufacturing of viral vectors through collaboration with Asia Pacific’ s only viral vector manufacturing facility, based at Westmead Hospital Precinct and clinical partners, the Royal Adelaide Hospital.
Target customers / end-users
• patients with severe type-1 diabetes
• potential benefit for all diabetes sufferers.
Progress
• AU $ 7.1m research translation funding
• established MOUs with leading institutes, including Royal Adelaide Hospital, Breakthrough T1D and Children’ s Medical Research Institute
• successful pilot and prototype testing.
Research Group
Validated( real-world setting)
Multidisciplinary team
TRL 5
Healthy Living
For patients with type-1- diabetes( T1D), immune damage to their bodies leads to high blood sugar levels and a reduced quality of life. This collaborative team will develop a gene therapy to protect insulin-producing cells, thereby stopping immune destruction and restoring normal insulin production.
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