HC Bioscience, Inc.

The reauthorization of FDA’s Rare Pediatric Disease Priority Review voucher (PPRV) program has been making headlines as a critical path forward to ensuring continued innovation on behalf of rare pediatric disease patients.     Since launching in 2012, the program has awarded 53 vouchers for medicines to treat 39 rare pediatric diseases, benefiting more than 200,000 patients.    Recently, our CEO, Leslie J Williams, authored an article in the Boston Business Journal on the urgent need for congress to renew the program that addresses obstacles to progressing rare disease research.     Read Leslie’s timely perspective on the decision, which has now been delayed until December 20: https://bit.ly/3TOZu9W    #RareDisease

We’re thrilled to highlight Dr. Jose Lora, our Chief Scientific Officer! At HC, Dr. Lora leverages his extensive experience within translational research to lead the development of our cutting-edge tRNA-based therapies. Jose’s experience across multiple therapeutic areas and modalities— including autoimmunity, oncology, and rare disease as well as gene therapies, small molecules and proteins— provides him insight to advance our pipeline across multiple therapeutic areas including hemophilia, DMD, and cancer.   Jose is a distinguished scientist and biotech leader with an extensive background in translational research and therapeutic development. Prior to HC, he held roles as Chief Scientific Officer at Intergalactic Therapeutics and enGene, as well as senior roles at major companies such as Synlogic, Constellation Pharmaceuticals, GSK, Roche and Millennium Pharmaceuticals. We’re grateful to have him guiding our research and development efforts!

Each of our team members has taken a different, and sometimes winding path, into science, but we’re so glad that they’ve all made their way to HC!   When we welcomed Alex for his Make-A-Wish Day, HC Bio scientists participated in a round-table discussion with Alex and his family where they shared their backgrounds, what sparked their interest in science, and what they find most interesting about their current work. Alex and his brother shared their interest in planets and programming robots, and asked questions.

We recently welcomed the kids of HC Bio to our labs to help them learn about what their parents do every day. These junior scientists participated in a range of experiments that painted a picture of our innovative work on tRNA-based therapeutics. It was a fun-filled day, as we shared our passion for science with our children – perhaps they will enter this exciting field one day. The kids of HC are definitely ready for back-to-school season after this educational event!

We’re thrilled to announce that Simon Tsang, Ph.D., has joined our team as Chief Business Officer! Dr. Tsang brings a wealth of experience to his new role, including over two decades of industry experience and a proven track record in building innovation-focused companies and pipelines. Most recently, he served as an Operating Partner at Pivotal bioVenture Partners where he acted as Chief Business Officer to multiple biotech companies.     We’re excited to welcome Dr. Tsang and look forward to continuing to build a strong, skilled, and dedicated team.

We’re still thinking about our amazing day with Alex, alongside the Make-A-Wish® Massachusetts and Rhode Island Foundation! Sara Evke, Ph.D., Scientist II – In Vivo Pharmacology Discovery, guided Alex through his experiments during his day as an HC Bio scientist. She reflected on the experience and what this day meant to her as a DMD researcher. On this #WorldDuchenneAwarenessDay, we’re proud to advance research and develop pioneering treatments to improve the lives of those affected by this disease. We’re honored to have hosted Alex and to continue striving towards transformative therapies for patients like him.  

This week our CEO, Leslie J Williams, will be participating in the RNA Leaders USA Congress in San Diego, CA.    On Thursday, Leslie will present on tRNAs, molecular interpreters that translate the genetic code into proteins, and how they can be engineered to treat genetically defined diseases. Later that day, she will also join a panel on the future of RNA therapeutics alongside biotech leaders Trevor Hallam, Rachel Groppo, Brendan Frey, and moderator Michael Hodges, where they’ll discuss promising approaches that are being fast-tracked to the clinic.    We look forward to sharing our insights and engaging with other stakeholders on the latest #RNA developments! Learn more: https://bit.ly/4cswWcw    #RNALeadersUSA #biotech #RNAtherapeutics #RNALeaders 

Meet Chris Katanski, our Director of Platform Discovery. He leads tRNA Biology here at HC. Previously, Chris served as the Chief Executive Officer at 4SR Biosciences where he focused on advancing tRNA therapeutics to treat missense mutations. Prior to that role, Chris worked at the University of Chicago as a postdoctoral researcher with Professor Tao Pan, where he developed new technologies to study tRNA.    Chris holds a doctoral degree in biochemistry, biophysics, and molecular biology from the University of Chicago as well as a bachelor’s degree in biochemistry from the University of Michigan.    According to Chris, he is “competing for the title of most excited person for tRNA therapies.”    We love having you on the team, Chris! Visit our website to learn more about the HC Bio team: https://bit.ly/3VQzxqw

Explore our groundbreaking PTCX (‘Patch”) platform. Our novel technology tackles a broad spectrum of genetic diseases caused by nonsense mutations or premature stop codons (PTCs) that lead to the production of truncated and nonfunctional proteins.   PTCX tRNAs recognize and overwrite PTCs by incorporating the cognate amino acid, allowing the cell to endogenously produce full-length, functional proteins. Crucially, our minimally engineered RNAs which target PTCs have minimal effects on read-through of natural termination codons (NTCs), allowing proper translation termination   Discover more about our innovative work in genetic medicine by visiting our platform page here: https://bit.ly/3y6gbGc

Last week, we had the honor of fulfilling a wish for our amazing guest scientist of the day, Alex! Through the Make-A-Wish® Massachusetts and Rhode Island foundation, Alex was able to visit our headquarters in Boston and learn more about our drug development efforts for Duchenne muscular dystrophy, which specifically targets the nonsense mutation that personally impacts him.   Alex enjoyed a tour of the lab and participated in a variety of experiments, such as visualizing plasmids in agarose gel and examining muscle cells through a microscope. Following this, Alex presented his findings and was recognized as a “Rising Science Star!” We are so appreciative that Alex’s family was able to make the trip to Boston for this special day. It was truly an unforgettable experience. Thank you to Make-A-Wish America, Sean Holleran, Make-A-Wish North Texas and Make-A-Wish® Southern Florida as well as our dedicated team whose passion and commitment make moments like this possible.   In the coming weeks, we’ll be sharing more highlights from this amazing day! #MakeaWish #DMD