Regerna Therapeutics, Inc.
Regerna Therapeutics is developing a new class of regenerative medicines built on a first-in-class RNA-binding protein therapeutic platform. Its lead therapeutic, AUF1, orchestrates functional muscle regeneration by stabilizing beneficial mRNAs and degrading inhibitory ones, with a lead program, REG-A40, delivering AUF1 via AAV8 gene therapy for Duchenne Muscular Dystrophy. The company is also targeting sarcopenia and acute muscle injury, with IND submissions planned for both LNP and AAV lead programs.
- Drug Discovery & Therapeutics
- Biotechnology
- Healthcare Technology
Funding
Founders
Founder details are not available yet.
Product
Problem
Muscle loss from aging, injury, and genetic dystrophies is a major driver of morbidity and mortality, yet there are currently no approved pharmacological treatments for conditions like sarcopenia. Muscular dystrophies, including Duchenne Muscular Dystrophy which affects approximately 1 in 5,000 boys, progressively destroy muscle function and lead to premature death.
Solution
Regerna Therapeutics is developing a new class of regenerative medicines built on a first-in-class RNA-binding protein therapeutic platform. The company's lead therapeutic, AUF1, is a master controller of muscle repair that orchestrates the full cascade of functional muscle regeneration by stabilizing and increasing translation of muscle, mitochondrial, and neuromuscular junction regeneration mRNAs while degrading mRNAs that inhibit muscle regeneration. Regerna's lead program, REG-A40, delivers AUF1 via AAV8 gene therapy and has demonstrated the ability to enhance muscle strength and endurance, promote muscle regeneration, restore neuromuscular junctions, and stimulate mitochondrial biogenesis. By increasing utrophin, a paralogue of dystrophin, AUF1 restores muscle stability and function in the absence of dystrophin, offering a potential therapeutic approach for DMD and other muscle-wasting diseases.
Target Audience
Primary customers are patients with muscle-wasting conditions including sarcopenia in the elderly, Duchenne Muscular Dystrophy, and severe muscle injury or atrophy, along with the clinicians and healthcare systems treating these populations.
Features
- AUF1 mRNA-binding protein platform that precisely corrects RNA defects by stabilizing beneficial mRNAs (e.g., PGC1-alpha, utrophin) and degrading inhibitory mRNAs (e.g., TNF-alpha, MMP9, Twist) involved in muscle regeneration
- REG-A40 lead program delivering AUF1 via AAV8 gene therapy for Duchenne Muscular Dystrophy, targeting the disease's root cause by compensating for missing dystrophin with utrophin upregulation
- Demonstrated enhancement of muscle strength, endurance, and functional control with improved mitochondrial number and activity
- Restores neuromuscular junctions and maintains the ability to regenerate muscle after injury
- Broad pipeline spanning sarcopenia, muscular dystrophies, and acute muscle injury, addressing multiple stages of muscle dysfunction
- Clinical timeline includes IND submission for both AUF1 LNP and AAV lead programs with initiation of clinical testing