Gene therapies for neurodegenerative diseases that involve direct injection into portions of the brain have become acceptable enough to regulators that there is now progress towards the clinic on this front. This is despite the invasive nature of the delivery, and despite the high bar for safety that that tends to be applied to treatments that produce permanent changes in a patient. One might look at a recent trial for Huntington’s disease, for example. This willingness on the part of regulators to allow more adventurous approaches might reflect a growing frustration with the failure to generate curative (or even meaningfully preventative) therapies for prevalent neurodegenerative conditions, after decades of significant funding and effort.
Another example at an earlier stage of development is the reprogramming approach taken by YouthBio Therapeutics. The company intends to deploy a viral vector injected into the brain in order to insert reprogramming factors that are only activated in the presence of a small molecule such as doxycycline – so after the initial therapy, partial reprogramming of cells to restore a more youthful function can be induced as needed. This approach will be used to treat Alzheimer’s disease. The company recently held its first formal interaction with the FDA, one step on a long path towards discovering and satisfying regulatory requirements prior to a clinical trial.
YouthBio Therapeutics, a biotechnology company pioneering partial cellular reprogramming to treat diseases of aging, today announced a successful INTERACT meeting with the FDA for its lead Alzheimer’s candidate, YB002. In its formal response, the FDA agreed that existing preclinical data support the bioactivity of YB002 and YouthBio’s proposed first-in-human trial. This feedback represents a major de-risking event for YouthBio, which will now focus on CMC activities and a pilot toxicology study to support a Pre-IND meeting and finalize designs for IND-enabling studies.
This milestone continues YouthBio’s strong record of execution. It builds upon compelling scientific evidence, including a study in which YB002 ameliorated cognitive decline in mice. Additional Alzheimer’s models have demonstrated that partial reprogramming can reverse disease pathologies, counteract epigenetic aging, and rescue memory and learning. YB002 is a first-in-class gene therapy designed to safely and transiently express Yamanaka factors in the brain – a process known as partial reprogramming. This approach aims to reverse epigenetic changes that accumulate with aging while preserving cell identity, thereby restoring youthful gene expression and improving cellular function.
we are working on a gene therapy that involves delivering genes responsible for epigenetic rejuvenation into target tissues. These genes would be inactive by default but can be periodically activated by a small molecule, rolling back the epigenetics of target tissues to a younger level and rejuvenating them. Our long-term vision is to apply this therapy systemically, decreasing patients’ biological age and improving their health. Numerous studies have provided strong evidence for the rejuvenating power of partial reprogramming, showcasing its potential in various contexts, such as improved muscle regeneration, heart regeneration, and intervertebral disc rejuvenation. The results of these studies serve as the foundation for our work at YouthBio.
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