Dermetel's for-profit arm, Darling Therapeutics, is taking an innovative approach to select the 6 patients for the P1/P2 TMAU AAV gene therapy clinical trial, the 4 patients for the P1 TMAU enzyme replacement therapy clinical trial, and the 6 patients for the P1/P2 IVA AAV gene therapy clinical trial. The goal is to have 20% children (3-18) and 80% adults (18+). We want selection to be in compliance with RCT statistical standards so regulators feel as comfortable as possible when they review the numbers.
We're creating a full HIPAA and GDPR compliant patient database of patients who have confirmed diagnoses on medical letterhead and using a fair, unbiased algorithm to select patients to participate fully at random. Dermetel is collecting names, DOB, confirmed diagnosis, medical insurance information, and more, so that Darling Therapeutics can negotiate a fair price to get as many insurance carriers and government health systems on board as possible when the time comes. Many gene therapies fail to gain traction due to absurdly high prices way out of range for government health systems, and Darling Therapeutics is taking a fair pricing approach rooted in health equality principles. We need to know which insurances and governments to target. Even if patients are not selected for the clinical trials, Dermetel and Darling Therapeutics are both committed to ensure every patient worldwide has a 'fast track' pathway to receive immediate treatment the second the orphan designated treatments are approved by regulators. We want this to be a smooth procedure for all stakeholders.
Both gene therapy clinical trials are taking place at UCLA Medical Center in Los Angeles, CA and enzyme replacement therapy clinical trials are taking place Mayo Clinic in Rochester, MN.