Targeting with Transfer RNA: Startup gets $24 million in funding

Targeting with Transfer RNA

The Cambridge, Massachusetts-based startup, hC Bioscience, will be able to advance its work in transfer RNA-based (tRNA-based) therapies, thanks to $24 million in series A funding from investors like Takeda Ventures, ARCH Venture partners and 8VC. The company wants to advance two tRNA-based platforms for use in treating rare diseases and cancer. 

Known for flexibility and versatility, tRNA-based drugs could become pharma’s next big thing. The technology can be used interchangeably across many disorders, with the potential to treat many diseases regardless of the gene or location of the mutation, according to hC Bio. The goal is to tamp down on the errors in the genetic code so the full-length protein can be produced.

The tRNA acts as a translator between nucleotides and amino acids when building proteins in the body. It is responsible for turning codons -- sets of three nucleotides that compose DNA or RNA -- and building proteins.  

The company was founded by Leslie J. Williams, president and CEO, and Professor Christopher Ahern. Ahern, who had worked at the University of Iowa, has extensive experience researching RNAs. He was also part of a team that identified engineered transfer RNA (tRNA) for human premature termination codon (PTC) variants. If PTC variants cut proteins short, they can cause diseases such as Duchenne muscular dystrophy, cystic fibrosis, hemophilia and some types of cancers.  

Now, Williams and Ahern are developing two proprietary platforms for hC Bioscience to treat, and possibly cure, those diseases. Both platforms are based on transfer RNA, a molecule that helps translate genetic codes into proteins.  

The first is a platform, PTCX or “Patch,” which analyzes proteins and identify “nonsense mutations,” premature termination codons that cut off proteins before they are fully developed. Premature termination codons cause about 10 to 15 percent of all human diseases. Patch uses tRNA to suppress errors in genetic codes, allowing cells to develop healthy full-length proteins. 

The second platform is SWTX, or Switch, designed to target diseases caused by missense proteins by identifying and marking any mutations in proteins that may cause diseases. By leaving a biologic marker on these malformed proteins, the body can recognize the errors and destroy the misshapen protein.  

According to Williams, “We are creating medicines to restore protein function to its intended state without editing genes. A single tRNA therapy has the potential to treat many diseases, regardless of the gene or location of the mutation.” 

While announcing the funding, hC Bioscience also appointed a new chief technology officer: David Altreuter, Ph.D., who has more than 40 years of experience in patent creation and applications. From his time at companies such as Lyndra Therapeutics, Selecta Biosciences, Quiet Therapeutics, Alnylam and Acusphere, he has expertise in developing and designing novel drugs and leading those discoveries through clinical development.  

A newly assembled Scientific Advisory Board for the company includes Ahern and his previous research associate John Lueck, Ph.D., assistant professor for RNA biology at the University of Rochester. Other notable board members include Nobel laureate Craig Mello, Ph.D., from UMass Medical Center, who is now the chairman of the scientific advisory board, as well as Page Bouchard, former chief scientific officer and head of research at Novartis Gene Therapies John DiPersio, MD, Ph.D., Chief of Oncology, Washington University; John Bell, Ph.D., Scientific Director of the National Centre of Excellence for the development of Biotherapeutics for Cancer Therapy, University of Ottawa; Oladapo Yeku, MD, Ph.D., Assistant Professor of Medicine, Oncology, Harvard University; and Jeff Bockman, Ph.D., Head of Oncology Practice, Cello Health; along with John Lueck, Ph.D., Assistant Professor, Center for RNA Biology, University of Rochester and Christopher Ahern, Ph.D., Department of Molecular Physiology and Biophysics, University of Iowa.  

“We look forward to the team translating its technology into class-leading therapies for patients,” explained Steven Gillis, ARCH Venture member and chairman of the board of directors for hC Bioscience.  

Dr. Mello noted, “hC Bio’s tRNA-based technology brings to bear an entirely new class of RNA therapeutics to address the ever-growing list of genetically-defined and yet unmet medical needs. To meet these needs, hC Bio will partner with pharma while selecting a few of the most urgent targets to tackle internally.”

hC Bio’s Board of Directors includes Steven Gillis, ARCH Ventures, Sacha Mann, Takeda Ventures, David Moskowitz, 8VC and Leslie Williams, CEO. Dr. Gillis, Chairman of the Board, said, “hC Bio is building the company on strong, foundational IP. We look forward to the team translating its technology into class-leading therapies for patients.”

ARCH Venture Partners, which invests in advanced technology companies, is one of the world’s leading early-stage technology venture firms. It is a recognized leader in commercializing technologies developed at academic institutions, corporate research groups and national laboratories. ARCH invests primarily in companies it co-founds with leading scientists and entrepreneurs, bringing innovations in life sciences and physical sciences to market. For more information, visit www.archventure.com.

Takeda Ventures, Inc., the corporate venture capital group of Takeda, was founded in 2001 with the vision of generating disruptive technologies and therapeutic solutions for patients through venture-based partnerships. Its mission is to create strategic growth opportunities for Takeda by building, managing and investing in innovation-based companies. TVI partners with academic innovators, entrepreneurs and venture investors to create and nurture a portfolio of companies in an array of therapeutic areas, including oncology, gastroenterology, neuroscience and rare diseases. 

8VC, a technology investment firm headquartered in Austin, TX, has offices in San Francisco and New York City.  It partners with entrepreneurs to build technology platforms that create long-term economic and societal value.  8VC was built by a team of entrepreneurs, engineers, investors and philosophers who value operating experience and action, as well as new ideas and creative theories.  The partners have a proven track-record as founders of successful companies including Palantir, Addepar, Affinity and OpenGov, among others.  8VC was founded in 2011 and manages several billion dollars in committed capital to invest in smart enterprise platforms across industries including financial services, healthcare, logistics and Bio-IT.