This fall the leaders of the eleven biotechs dubbed “2023’s most ambitious” by biotech, drug, pharma and health news network Endpoints met together in downtown Boston’s State Room for an awards gala and celebratory sit-down dinner. 

“We are on the cusp of a revolution in genetic medicine that is as exciting as anything that has happened in the history of biotech,” said Drew Armstrong, Executive Editor for Endpoints News, at the pre-event fireside chat. “What the people in this room do has the power to change not just the course of one life, but how all of us see the world.”  

Included among the Endpoints 11 are BioProcure and Prendio clients Aera Therapeutics, Chroma Medicine, Maze Therapeutics, Orbital Therapeutics, ReNAgade Therapeutics, and Seismic Therapeutic. Read on to learn more about the exciting science behind these six promising startups. 

Aera Therapeutics emerged from stealth in early 2023 with $193 million in funding, thanks to the enormous potential latent in founder Feng Zhang’s innovative method for delivering genes into cells in areas of the human body that have been historically more difficult to reach with existing forms of gene therapy. Under the scientific leadership of Zhang (Broad Institute, Harvard) and Jason Shepherd (University of Utah), Aera is on a mission to harness enabling delivery technologies and precision payloads to develop transformative genetic medicines. Read more about the work being done to revolutionize the treatment of human disease by honing in on underlying causes with never-before-seen specificity by visiting www.aeratx.com  

What if the next big thing in gene editing technology doesn’t actually alter the DNA sequence? That’s the question many are asking thanks to Chroma’s innovative approach to switching genes on or off. Epigenetic editing can change gene expression without cutting the DNA itself. Thanks to groundbreaking work by scientists like Jennifer Doudna, Jonathan Weissman, Luke Gilbert, David Liu, Keith Joung, Luigi Naldini and Angelo Lombardo, there is new hope for patients living with diseases like cystic fibrosis and hepatitis B.  

Learn more about how Chroma is reimagining genome regulation by visiting www.chromamedicine.com  

Competition is heating up among the few organizations making new treatment discoveries for chronic kidney disease, and startup Maze Therapeutics is emerging as a key player. Fueled by funding from Sanofi, Third Rock Ventures, ARCH Venture Partners, and a long list of other venture, capital, and investment firms, Maze is advancing a small molecule Apolipoprotein L1 (APOL1) inhibitor program that offers a potentially disease-modifying treatment option for people with APOL1-mediated chronic kidney disease. With trials ahead for later this year, this new therapy is poised to revolutionize treatment for those suffering from APOL1-driven chronic kidney disease.

Learn more at www.mazetx.com  

mRNA medicines have been all over the news since the breakthrough Covid-19 vaccines hit the market, but some scientists in the field are focusing in on a different type of RNA—circular RNA, as opposed to linear strands—and the potential these more stable molecules present for treating disease. Launched one year ago by ARCH Venture Partners Kristina Burow and Carol Suh, Giuseppe Ciaramella, the president of Beam Therapeutics, and John Maragnore, who led RNAi pioneer Alnylam for nearly 20 years, Orbital Therapeutics is looking forward to the future of RNA-based treatment approaches by building the most expansive toolboxes in RNA medicines. Ciaramella said that Orbital is testing circular RNA “in every program that we’re doing,” but that the company is “somewhat agnostic” to which form of RNA is used in its drug candidates. “We’re just letting the science dictate which one works,” he is quoted as saying in this article by Endpoints. 

Read more about Orbital and the potential applications of circular RNA at www.orbitaltx.com  

Welcome to the era of RNA medicine, a field that experts agree holds almost limitless potential for rewriting the underlying basis of disease. According to the company website, “ReNAgade aims to overcome the limitations of RNA medicines by applying our comprehensive deliver, code, edit, insert technologies to precisely correct disease on an unprecedented scale.” Founded by Ansbert Gadicke and headed up by CEO Amit Munshi and CSO Peter Smith, ReNAgade Therapeutics is driven by many challenges, perhaps the biggest being figuring out a way to deliver next-gen RNA drugs to areas outside the liver. Success in this area would generate a veritable revolution for entire fields of medicine.  

Read more at www.renagadetx.com  

What do you get when an expert on protein structures teams up with a leading computational biologist? When Harvard colleagues Tim Springer and Debbie Marks did just that, the result was the IMPACT platform, a remarkable feat of machine learning that has already resulted in the generation of two programs to create novel therapies for immune disorders–in just eighteen months. With early results like those, we can expect to see much more from Seismic in the future.  

Visit www.seismictx.com to learn more about how machine learning is shaping the landscape of novel biologics.  

Read about all 11 of the biotech startups identified by Endpoints as “making the biggest bets with the most exciting science in 2023” in this article.