Unlocking muscle biology with innovative medicines

Restoring Functional and Endurant Muscle for Healthy Aging, Longevity and Muscle Injury and Wasting Diseases.

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First-in-class mRNA Binding Protein Therapeutics.

First-in-class mRNA Binding Protein Therapeutics.

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Restoring muscle, rebuilding lives

ABOUT REGERNA

At Regerna Therapeutics, we are pioneering cutting-edge therapies to restore and regenerate functional muscle for patients suffering loss of muscle from injury, aging and dystrophies.

We are developing a new class of regenerative therapeutics that offer the potential to improve the lives of patients with muscle disorders.

Our mission is to bring life-changing therapies to patients living with conditions where muscle loss severely impacts quality of life.

SCIENCE

Regerna is pioneering a new class of regenerative medicines

RNA modulation is central to many diseases, where disruptions in RNA stability, splicing, or translation can lead to impaired gene expression and cellular dysfunction. RNA-binding proteins, which regulate these processes by recognizing specific RNA sequences, are dysregulated in many diseases. 

At Regerna, we are building a first-in-class RNA-binding proteins therapeutic platform to precisely correct these RNA defects.  Our lead therapeutic, AUF1, is a master controller of muscle repair and regeneration. 


Process Of Muscle Functional Recovery

Diagram illustrating muscle cell development stages from satellite cells to functional muscle, including satellite cell activation, proliferation, myoblast formation, fusion into myotubes, myofiber formation, and muscle repair and recovery, with a detailed section on the gene expression process involving UTR regions and AUF1 proteins.

↑ AUF1 stabilizes and increases translation of muscle, mitochondrial, NMJ regeneration mRNAs
(e.g., PGC1-alpha, utrophin, and other key promoters)

↓ AUF1 degrades mRNAs that inhibit muscle regeneration
(e.g., TNF-alpha, MMP9, Twist, and other key inhibitors)

Our lead mRNA Binding Protein, AUF1, is a Master Regulator of Muscle Repair and Regeneration

SCIENCE

AUF1 orchestrates and controls the full cascade of functional muscle regeneration - activating mitochondria, restoring neuromuscular junctions, and driving strength at the molecular level.

Impact of AUF1 Supplementation in Damaged or Dysfunctional Muscle

  • Increases muscle strength and endurance

  • Restores functional control

  • Reduces impact of injury and risk of further injury

  • Utrophin is a homolog that compensates for lack of dystrophin in DMD mice

  • Number and activity of mitochondria improved by AUF1 Boosts energy, activity and recovery of muscle

  • Maintains and builds ability to regenerate

SCIENCE

Broad range of potential therapeutic indications

Societal problem: Loss or absence of functional muscle

A massive unmet need, leading healthcare economic burden and key driver of morbidity and mortality in aging and metabolic disorders.

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Severe muscle loss, aging, injury, or atrophy

  • Sarcopenia is an age-related condition characterized by the progressive loss of skeletal muscle mass, strength, and function. This decline is driven by factors including reduced muscle protein synthesis, mitochondrial dysfunction, chronic inflammation, and decreased physical activity. As muscle fibers deteriorate, elderly individuals often experience reduced mobility, diminished endurance, and a heightened risk of injury and falls leading to hip fractures and, all too often, premature death. 

    Currently, there are no approved pharmacological treatments for sarcopenia. 

    Regerna’s programs are focused on restoring muscle mass and function by targeting key molecular pathways that are impaired with aging and are involved in muscle regeneration, mitochondrial biogenesis, and protein homeostasis. Though these unique compounds, we hope to offer a therapeutic approach to counteract age-related muscle degeneration.

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  • Muscular dystrophies are a group of genetic disorders characterized by progressive muscle weakness and degeneration of the skeletal muscles. Over time, muscle fibers are replaced by fat and connective tissue, leading to decreased muscle function, reduced mobility, and premature death. Duchenne Muscular Dystrophy (DMD), a severe form of muscular dystrophy caused by the absence of dystrophin, affects approximately1 in 5,000 boys.

    Our lead program focused on DMD, REG-A40, comprised of the mRNA binding protein AUF1 delivered via AAV8 gene therapy, has demonstrated the ability to enhance muscle strength and endurance, promote muscle regeneration, and stimulate mitochondrial biogenesis. These effects are achieved because AUF1 increases utrophin, a paralogue of dystrophin, thereby restoring muscle stability and function.

Muscular Dystrophies

TIMELINE

Regerna timeline for IND submission for AUF1 LNP and AAV lead programs and initiation of clinical testing

Regerna Timeline

Leadership Team

  • Kenneth Moch headshot

    Kenneth Moch

    CEO

    30+ years experience building companies from startup to commercialization, raising over $1 billion in dilutive and non-dilutive capital and leading two market opening IPOs

    Former President & CEO of five life science companies: Cognition Therapeutics, Chimerix, Biomedical Enterprises, Alteon, Biocyte Corporation

    Co-founder and VP of The Liposome Company, first LNP company

    Faculty Affiliate, Division of Medical Ethics, NYU School of Medicine

    LinkedIn
  • A middle-aged man with glasses, short gray hair, wearing a dark suit and a blue shirt, looking directly at the camera.

    Robert Schneider, PhD

    PRESIDENT

    Co-founder of six biotechs including ImClone, Canji, PTC Therapeutics

    30+ years in translational R&D

    Former Associate Dean of NYU for therapeutics development, technology commercialization

    Albert B. Sabin Professor of Microbiology and Molecular Pathogenesis at NYU Langone Health

    Co-inventor on all Regerna IP

    Pioneering research in mRNA binding proteins in regenerative medicine

    LinkedIn
  • Close-up portrait of a woman with dark hair and a slight smile, wearing a black top, against a white background with a purple circular border.

    Dounia Abbadi, PhD

    CSO

    Pioneer of mRNA-binding protein therapeutics, for which received 2025 BioInnovation Institute & Science Prize for Innovation

    Highly experienced investigator in muscle biology

    12 years+ in muscle regenerative medicine and muscle aging process

    Co-inventor on all Regerna IP

    PhD in Biology, Health and Biotechnology from the Toulouse University

    LinkedIn
  • A woman with long dark hair, wearing a blue top and gold hoop earrings, smiling against a white background with a purple circle border.

    Manisha Narasimhan, PhD

    SENIOR ADVISOR

    Chief Corporate Development and Digital Officer, Bausch + Lomb

    Former CFO & co-founder, Vesper Healthcare. Former SVP, Strategic initiatives & Investor relations, Allergan. Former Head of IR, Regeneron

    Former biotech equity research analyst Canaccord Genuity, Piper Jaffray

    LinkedIn

Advisory Board

  • A portrait of a middle-aged man with short gray hair and a beard, smiling, wearing a navy suit and light blue shirt, against a plain white background.

    Ed Saltzman

    BIOTECH CORPORATE STRATEGY

    Founder, CEO, Defined Health

    CEO, Cello Health

    25+ years consulting pharma, biotech, investors

    Advisor to numerous top biotech, pharma and venture capital companies

    LINKEDIN
  • Portrait of an older woman with short gray hair, wearing a black blazer and patterned shirt, smiling slightly.

    Laura Coruzzi, PhD, JD

    INTELLECTUAL PROPERTY & LEGAL STRATEGIES

    Executive VP of IP, RegenxBio

    Partner, Biopharma IP strategies, litigation, Jones Day

    Partner, Biopharma IP strategies, litigation, Pennie & Edmonds

    LINKEDIN
  • A middle-aged man with gray hair and a beard, smiling, wearing a light blue collared shirt, against a white background with a blue circular border.

    Jude Samulski, PhD

    AAV DELIVERY TECHNOLOGIES AND GENE THERAPY

    Co-founder & CSO, AskBio (acquired by Bayer) and Bamboo Therapeutics (acquired by Pfizer)

    Director, UNC Gene Therapy Center

    Former president, American Society of Gene and Cell Therapy

  • Portrait of a man with short gray hair, glasses, smiling, wearing a dark suit jacket and a light-colored collared shirt, against a white background with a blue circular border.

    Imed Gallouzi, PhD

    MUSCLE REGENERATION AND MNRA BINDING PROTEINS

    Director, COO KAUST Smart Health Initiative, Saudi Arabia, King Abdullah University

    Professor of Biochemistry, McGill University

    linkedin
  • Close-up of a middle-aged man with glasses and gray hair, wearing a dark shirt, against a plain white background.

    Bradley Olwin, PhD

    MUSCLE STEM CELLS AND MUSCLE REGENERATION

    Professor, Univ of Colorado

    Expert on muscle stem cells and muscle regeneration, muscle wasting diseases

    Member, University of Colorado BioFrontiers Institute for Regenerative Medicine

    linkedin
  • Close-up portrait of a woman smiling, with short dark hair, gold earrings, and a patterned top.

    Jahannaz Dastgir, DO

    DYSTROPHINOPATHY CLINICAL STRATEGIES

    Clinical Development Lead, DMD Program, RegenxBio

    Director, Clinical Research, Applied Therapeutics

    Assistant Professor of Pediatric Neurology, Columbia University

    LinkedIn
  • Sarah Dolman Headshot

    Sarah Dolman, PhD

    R&D pipeline commercial prioritization

    Partner, Recon Strategy

    Strategy & Business Operations,  Verily Life Sciences

    Consultant, Boston Consulting Group

    Director, Process Research & Process Chemistry, Merck

    Linkedin

News and Key Publications

  • Dounia Abbadi, PhD, Regerna CSO, Wins Finalist in 2025 BioInnovation Institute & Science Prize for Innovation

    July 2025

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  • Interview with Dounia Abbadi. Dounia has spent her career investigating a part of the body we use every single day: our muscles.

    April 2025

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  • mRNA binding proteins join the longevity pipeline: Rebuilding muscle through the power of mRNA binding protein therapeutics

    April 2025

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  • AUF1 gene transfer increases exercise performance and improves skeletal muscle deficit in adult mice

    July 2021

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  • Muscle development and regeneration controlled by AUF1-mediated stage-specific degradation of fate-determining checkpoint mRNAs

    May 2019

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  • Targeted mRNA Decay by RNA Binding Protein AUF1 Regulates Adult Muscle Stem Cell Fate, Promoting Skeletal Muscle Integrity

    July 2016

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  • mRNA decay factor AUF1 maintains normal aging, telomere maintenance, and suppression of senescence by activation of telomerase transcription

    July 2012

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Contact Regerna

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