CureDuchenne Ventures Invests in hC Bioscience and its Novel tRNA-Based Therapeutic Approach for Duchenne Muscular Dystrophy

February 28, 2023
Cambridge, MA

hC Bioscience, a pioneer in the development of tRNA-based therapeutics, received an investment from CureDuchenne Ventures, the strategic investment arm of CureDuchenne, a global nonprofit committed to finding and funding a cure for Duchenne muscular dystrophy. This investment will come in as an extension of hC Bioscience’s series A and serves as recognition of the potential of the Company’s tRNA-based approach to finding a cure for rare diseases like DMD.

“Our venture investments have successfully contributed to the development of life-changing therapies and technologies that aim to produce near-complete dystrophin, including exon skipping therapy and gene therapy,” said Debra Miller, founder and chief executive officer of CureDuchenne. “tRNA is another promising therapeutic approach that offers the potential to produce full-length functional dystrophin protein. This investment marks the start of a long-term relationship with hC Bioscience and allows us to further diversify the treatments we hope to help bring to patients.”

DMD is a debilitating genetic disorder that affects approximately 1 in every 3,500 male births worldwide. It is caused by mutations in the dystrophin gene, located on the X chromosome. In individuals with DMD, mutations in the dystrophin gene result in the absence or significant reduction of functional dystrophin protein, leading to progressive muscle degeneration and weakness.

“We’re honored to receive this investment and to be a part of CureDuchenne’s unwavering commitment to finding a cure,” said Leslie Williams, president and CEO of hC Bioscience. “Nonsense mutations, or premature stop codons (PTCs), drive approximately 20% of DMD cases. We have begun research to apply our PTCX (“Patch”) technology to suppress PTC’s in the dystrophin mRNA that is responsible for loss of muscle function in DMD patients. Our ultimate goal is to deliver a tRNA therapeutic that can penetrate muscle and produce clinically meaningful levels of full-length functional protein.”

About hC Bioscience, Inc.
hC Bioscience is dedicated to improving the lives of patients with the development of first-in-class tRNA-based therapeutics to target protein dysfunction. hC Bioscience’s innovative approach to precision protein editing has the potential to treat genetically defined conditions, which account for 10-15% of all human disease. The lead platform in development is directed at restoring protein function in diseases caused by nonsense mutations or premature termination codons (PTCs). A single tRNA therapy has the potential to treat many diseases, regardless of the gene or location of the mutation. The Company has raised $40 million to date and its investors include ARCH Venture Partners, Takeda Ventures, 8VC, Taiho Ventures and Panacea Venture.

About CureDuchenne
Twenty years ago, CureDuchenne was created with one goal: to find and fund a cure for Duchenne muscular dystrophy, the leading genetic killer of young boys. Today, CureDuchenne is recognized as a global leader in research, patient care, and innovation for improving and extending the lives of those with Duchenne. CureDuchenne’s innovative venture philanthropy model has advanced transformative treatments for Duchenne muscular dystrophy, including 17 projects that advanced to human clinical trials and multiple projects to overcome the limitations of exon-skipping and gene therapy. In addition, CureDuchenne contributed early funding to the first FDA-approved Duchenne drug, pioneered the first and only Duchenne physical and occupational therapist certification program and created an innovative biobank and data registry, accelerating research toward a cure. For more information on how to help raise awareness and funds needed for research, please visit