Many Researchers and Companies Will Aim to Produce Small Molecule Reprogramming Therapies

The typical path for any program in biomedical research and development is to first demonstrate interesting results in animal studies using forms of genetic engineering or gene therapy, and then find small molecules that adjust the same mechanism. Small molecules are never as good as genetic manipulations, the size of the effect is always smaller, usually much smaller, and there are inevitably side-effects. Small molecule development is much easier to conduct, however, more familiar to investors and regulators and program managers, a well-trodden path. Thus while the future of medicine is gene therapy, in search of large effect sizes and no side-effects, the present industry remains near entirely focused on small molecules. Given the popularity of reprogramming as an approach to treat aging, an increasing number of research groups and companies are working to find small molecules that induce reprogramming to some degree, an alternative to gene therapies that induce expression of the Yamanaka factors. Based on the discoveries to date, it seems plausible that they will succeed. Two weeks ago, longevity biotech startup Clock.bio emerged from stealth with $4 million in funding, and setting itself an ambitious goal to be in a Phase 3 trial for a healthspan-extending intervention by the end of the decade. With the clock ticking, the company is already working to map rejuvenation biology across the entire human genome over the next 12 months. Having completed an initial scr...
Source: Fight Aging! - Category: Research Authors: Tags: Daily News Source Type: blogs