Retro Biosciences to Begin Clinical Trial for Brain-Aging Reversal Pill RTR242

San Francisco, CA – Retro Biosciences, a longevity biotechnology company backed by OpenAI CEO Sam Altman, is set to commence human clinical trials for its experimental drug, RTR242. The pill is designed to target and reverse the aging process in the brain by restoring cellular recycling, known as autophagy, and eliminating damaged proteins or "gunk" that accumulate over time. The company aims to add 10 healthy years to the average human lifespan through such interventions.

Retro Biosciences, founded in 2021, has garnered significant attention, notably from Sam Altman, who initially invested $180 million in the startup. More recently, the company announced it is raising a $1 billion Series A funding round, with Altman participating, to accelerate its research and development efforts. CEO Joe Betts-LaCroix stated that the company plans to dose its first trial patient with RTR242 by the end of 2025, with Australia selected as a clinical trial site due to its expedited regulatory process for Phase 1 safety trials.

RTR242 focuses on reviving autophagy, a crucial cellular process that often falters with age. "There are old, misfolded, mutated, broken, undigestible proteins inside cells that build up over time," Betts-LaCroix explained, adding that the normal cellular recycling system gets disrupted. The pill aims to clean up this damage, particularly in brain cells, and is specifically being developed to help reverse Alzheimer's disease. This approach differs from current Alzheimer's drugs that primarily slow cognitive decline by targeting amyloid plaques.

Beyond RTR242, Retro Biosciences is also developing other therapies, including a treatment for blood diseases (RTR890) and a stem cell-derived therapy for central nervous system disorders (RTR888). The company has also partnered with OpenAI to develop a custom AI model, GPT-4b micro, which has shown promise in enhancing stem cell reprogramming markers. This integration of AI and biotechnology underscores Retro Biosciences' ambitious strategy to tackle aging-related diseases.