M34 Inc. – Redefining Human Gene Therapy and Neuroreprogramming

Founded on June 4, 2021, in Chapel Hill, North Carolina, USA

M34 Inc. is a next-generation biotechnology company pioneering transformative therapies for neurodegenerative diseases through in vivo, in situ cellular transdifferentiation and recombinant adeno-associated virus (rAAV) vectors engineered and selected directly in humans.

The company was founded by two of the most influential leaders in the field of human gene therapy: Professor Dr. R. Jude Samulski, PhD—the first scientist to clone the AAV genome for therapeutic purposes—and Ms. Sheila Mikhail, JD, MBA, an internationally recognized serial biotech entrepreneur with a proven track record of building and scaling successful gene therapy ventures.

Built by pioneers and fueled by data, M34 is unlocking the future of precision human cellular reprogramming—advancing gene therapy applications that redefine the landscape of regenerative medicine.

Prof. Samulski and Ms. Mikhail previously co-founded several high-impact biotech ventures, including Asklepios BioPharmaceutical (AskBio), which they built from a small startup into a global leader in AAV gene therapy with over 1,000 employees across four countries. Under their leadership, AskBio pioneered multiple first-in-class treatments and developed a robust portfolio of foundational AAV technologies—including the self-complementary AAV (scAAV) vector platform, now a core component of Zolgensma®, the first FDA-approved gene therapy for spinal muscular atrophy (SMA)—a one-time, potentially curative therapy that has changed the lives of thousands of children worldwide.

AskBio’s expansive pipeline advanced dozens of AAV-based candidates into clinical development and ultimately led to its $4 billion acquisition by Bayer. In parallel, Prof. Samulski and Ms. Mikhail co-founded and successfully exited additional ventures—Bamboo Therapeutics (acquired by Pfizer), Chatham Therapeutics (acquired by Baxter), and Viralgen/TAAV (acquired by Bayer)—each of which played a defining role in shaping today’s gene therapy landscape.

At M34, we’re not repeating the past—we’re leveraging deep experience and proven success to build a future where neurodegenerative diseases are not just treatable but potentially curable through one-time administration. By engineering AAV vectors with unprecedented tissue specificity and translational relevance—developed in humans, for application in humans—we are eliminating the historical risk of preclinical-to-clinical failure rooted in animal models. M34 is leading a new era of neuro-targeted gene therapy, advancing treatments for diseases of the brain and spinal cord with unmatched precision, efficacy, and clinical relevance.