Retro Biosciences is developing a drug designed to help reverse Alzheimer’s by restarting autophagy, the body’s cellular recycling system. This natural process, which is also triggered by fasting, tends to deteriorate with age.

"There are old, misfolded, mutated, broken, undigestible proteins inside cells that build up over time," said Retro CEO Joe Betts-LaCroix. "The normal cellular recycling system gets messed up."

The experimental drug, RTR242, aims to clear this cellular buildup—sometimes described as “gunk”—especially in brain cells. If successful, the treatment could offer a new way to tackle Alzheimer’s and Parkinson’s by reactivating stalled autophagy instead of targeting amyloid plaques, as traditional therapies do.

Retro’s Clinical Strategy: A First Step in Australia

To move quickly, Retro has selected Australia as the launchpad for its Phase 1 safety trial. The company has chosen a clinical trial site, signed with lab vendors, and expects to enroll its first participant toward the end of the year.

The trial’s early results will be essential. They could help the company raise more capital to support larger studies. Retro has openly shared its plan to raise $1 billion in Series A funding.

This would place the company closer to major players like Altos Labs, a Jeff Bezos-backed biotech that has secured over $3 billion from top-tier tech investors, including Yuri Milner, Joe Lonsdale, and Robert Nelson.

Beyond the Brain: Expanding into Blood and CNS Therapies

RTR242 is just one of three major programs in development. Another Retro initiative, RTR890, involves generating new blood stem cells using a patient’s own cells. A third program, RTR888, is focused on central nervous system disorders, using stem cell-derived therapies.

Betts-LaCroix said that all of the company’s efforts are designed to "reset some aspect of our biology back to essentially a younger age." He prefers therapies that aim to reverse aging, rather than those that only slow it. "The 'slow it down' thing just feels kind of like a weak sauce," he said.

Longevity Philosophy: Between Pharma and Moonshots

Within the longevity space, strategies range from conventional to radical. Some companies follow a cautious path, developing one disease-targeting drug at a time to attract pharmaceutical interest. Retro is aiming for something in between.

Physicist Peter Fedichev, founder of rival longevity firm Gero, commented on Retro’s work: "I think they pretend to be careful. With all my respect, I hope that they are more radical on the inside."

Betts-LaCroix believes the company’s goal—to add 10 healthy years to human life—is ambitious. "Curing cancer would add about three years to life expectancy, and curing heart disease about four," he said. "Adding 10 years of healthy lifespan to the adult population will be an even greater impact—one of the greatest achievements in the history of healthcare."

AI-Driven Protein Design and the Future of Longevity Tech

Retro has also collaborated with OpenAI to create a tool called GPT-4b micro, a protein-designing AI model. According to the company, this model increased the expression of stem cell reprogramming markers by 50-fold, compared to results from natural reprogramming factors.

Cellular reprogramming is currently a leading trend in longevity science. Researchers are trying to find ways to make old cells behave like young ones and build new treatments from that foundation.

Still, despite these advancements, Retro is proceeding cautiously with its clinical trial work. Betts-LaCroix has stated that the company is still in “hardcore preclinical mode.”

PHOTO: FORBES/JUSTIN WINOKUR

This article was created with AI assistance