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Niagen Bioscience Launches Pharmaceutical Program for Accelerated Aging and Rare Genetic Diseases

Business Wire
Wed, July 8, 2026 at 6:02 PM GMT+5:30
8 min read
- NAGE
-1.43%
NB4168, a patented therapeutic candidate designed to have significantly increased bioavailability and a differentiated safety and pharmacokinetic profile compared to NRC, is the first investigational product to advance the Company’s NAD+ platform into regulated drug development
LOS ANGELES, July 08, 2026–(BUSINESS WIRE)–Niagen Bioscience, Inc. (NASDAQ: NAGE), the global authority on NAD+ science, today announced the formal launch of the first drug candidate of NAD Pharmaceuticals Corp., its wholly owned subsidiary focused on developing therapies for accelerated aging and rare genetic diseases. The program will primarily focus on how NAD+, a coenzyme central to energy metabolism, DNA repair, mitochondrial function and cellular stress responses, modulates in rare diseases for which DNA misrepair and mitochondrial dysfunction are foundational underlying causes. This represents a strategic expansion of Niagen Bioscience’s NAD+ platform from cellular-health innovation into regulated drug development. A derivative of the NAD+ precursor, nicotinamide riboside (NR), NB4168 is the first publicly announced investigational pharmaceutical product candidate within the program, with Ataxia Telangiectasia (A-T) as the initial indication.
“NB4168 is the next step in our strategy to translate Niagen Bioscience’s NAD+ leadership into pharmaceutical development,” said Rob Fried, Chief Executive Officer of Niagen Bioscience. “There are two independent, published clinical studies investigating the impact of NR on A-T, both of which have shown statistically significant results, in addition to several non-clinical studies. We believe this body of work significantly de-risks the development pathway for NB4168, which we specifically developed for therapeutic applications.”
A-T is a rare genetic disease caused by mutations in the ATM gene. The disease typically presents in early childhood and is characterized by progressive loss of motor coordination, impaired immune function, increased susceptibility to infections, pulmonary complications, and a substantially elevated risk of cancer. Children living with A-T often experience worsening neurological disability over time, with many requiring wheelchair assistance as the disease progresses. There are currently no FDA-approved therapies for A-T, and treatment is largely limited to supportive care. A-T impacts roughly 1 in 40,000 people in the U.S. (Riboldi et al., 2023; Tieve et al., 2015) and 1 in 150,000 people in Europe (Bhatt et al., 2015).


