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At this year’s BIO International Convention in Boston, Riboway Therapeutics emerged as the winner of the 2025 BIO Start-Up Stadium seed-stage category. For founder and CEO Dr. Qinghong (Avery) Yan, the honor is more than just an industry milestone. It is a testament to a lifelong journey defined by resilience, determination, and a refusal to yield to limitations—personal or scientific.
From a remote village to the biotech stage
Avery’s story began far from Boston, in a small, mountainous village in China where traditional values favored sons over daughters. With two girls in the family, prejudice and hardship were constant companions, Avery explains. From an early age, she and her sister shouldered responsibilities far beyond their years—working on the farm, chopping firewood, and overseeing every aspect of difficult farm life—all to help the household survive and to earn their way through school, where resources were scarce and teachers undertrained.
But hardship forged resilience, and through her studies, Avery found her own unique sense of empowerment.
“In high school biology, I remember learning that gender is determined by chromosomes, not by mothers. I ran home to tell my father: it wasn’t my mom’s fault for not having sons!” she recalls. Knowledge, for Avery, became the great equalizer.
This drive carried her across continents to the U.S. and culminated in her founding Riboway Therapeutics. Her graduate training at Stony Brook University developed her expertise in neuroscience and gene expression regulation that shapes the diverse electrophysiological properties of excitable cells. Her postdoctoral work at Columbia University revealed thousands of new potentially druggable RNA splicing events. She led translational programs at Amgen, started working on building in intro (human organoids) and in silico (AI) models to bypass animal testing back in 2015, and contributing to multiple Investigational New Drug (IND) applications and breakthrough medicines. She then moved into venture incubation, bridging science and business, which developed her entrepreneurial skills.
Each step was part of a deliberate journey focused on turning discovery into impact, which culminated in the creation of Riboway Therapeutics.
A mission born from science
Riboway Therapeutics was founded on the insight that RNA biology holds untapped layers of therapeutic control.
More than 80% of human proteins, particularly transcription factors driving genetic and neurodegenerative diseases, are considered “undruggable” by traditional modalities. Existing RNA therapeutics offered promise but remained constrained, mostly limited to silencing genes.
Riboway’s AI-enabled Smart ASO (Antisense oligonucleotides) platform changes that equation. By decoding hidden RNA regulatory events, it enables precise modulation of protein expression—not only downregulation, but also upregulation, activation, and inhibition. This breakthrough opens the door to drugging targets once considered impossible to reach.
“We see an RNA molecule born from the nucleus like a baby: innocent and undefined, with no idea what it will become until RNA-binding proteins come to guide and shape it through processes such as splicing, eventually turning it into a mature form ready to produce proteins,” says Avery. “Using AI, we gain deep knowledge of how RNA is regulated throughout this life journey by integrating information about RNA sequence features, the behavior of binding proteins that create the regulatory microenvironment, and the isoforms into which RNA matures.”
Dr. Sebastien Weyn, Riboway’s co-Founder and RNA computation lead, adds: “We found countless opportunities to modulate RNA regulation in ways that change the fate of the RNA molecule itself, enabling precise therapeutic control over the target.” Sebastien completed his PhD in the same lab as Avery at Columbia University, where the two co-authored several papers, including the landmark discovery of thousands of new RNA splicing events.
Riboway calls the ASOs they design “Smart ASOs,” emphasizing their flexibility. Unlike traditional antisense oligonucleotides that rely on RNAse H-mediated degradation, or siRNAs that trigger RISC-mediated silencing, Smart ASOs work by leveraging the cell’s own endogenous RNA regulatory pathways.
“There are validated successes in this class of ASOs, such as Spinraza, the current market leader in oligonucleotide drugs. But RNA biology is complex, and it often takes years of research to uncover a mechanism that allows a drug like Spinraza to have an impact. What makes us unique is the ability to rapidly apply and extend this class of ASOs to virtually any target, in any direction—activation, inhibition, or up/down-regulation—through our AI-powered discovery of novel RNA regulation mechanisms,” explains Avery.
The platform has already demonstrated its breadth, validated across all five targets pursued to date. “We are likely the first to achieve every possible mode of modulating a target, including, most notably, the activation of protein function,” Avery adds.
Pioneering impact: Addressing unmet needs
Riboway’s lead program targets spinocerebellar ataxia type 2 (SCA2), a rare neurodegenerative disorder with no approved disease-modifying therapies. With the program on track for clinical proof-of-concept within three years, Riboway is working to address a critical unmet need for patients facing progressive motor impairment and debilitating symptoms.
“We have demonstrated potency, safety and dosing advantages of our ASO compared to competitor compounds. And we have worked with clinical KOLs and physicians who see the patients every day to develop a biomarker strategy that would work for SCA2.” says Lijun Wu, co-founder and advisor of Riboway. Riboway believes that the Smart ASO approach, combined with a carefully optimized clinical trial design, will give their program a significant advantage in advancing toward meaningful patient outcomes.
Riboway’s second program is a groundbreaking, first-in-class antisense oligonucleotide capable of directly activating a high-value transcription factor, a target previously considered undruggable. This achievement opens entirely new possibilities for therapeutic intervention in diseases where traditional approaches fall short.
Together, these programs showcase Riboway’s platform potential to tackle a wide range of indications, spanning neurodegenerative, autoimmune, and metabolic disorders. The company is strategically prioritizing indications, starting with rare, genetically defined diseases like SCA2—valued at an estimated $1–2 billion in U.S. and EU markets—to rapidly establish clinical proof-of-concept and de-risk development. Success in these initial programs will enable expansion of the Riboway therapies to more complex diseases with larger markets, including ALS ($10B+) and Alzheimer’s disease ($30B+), where the need for innovative, disease-modifying therapies is urgent.
By addressing conditions that currently have limited or no treatment options, Riboway aims not only to capture significant market opportunity but also to make a meaningful difference in the lives of patients with high unmet medical needs.
Building with purpose
The BIO Start-Up Stadium award is energizing for the Riboway team and helps reaffirm their focus on patients. “Our goal is to make every therapeutic target druggable,” says Avery. With a seasoned team that has advanced over 20 INDs and contributed to five approved drugs, Riboway is pursuing a strategy that balances scientific boldness with pragmatic milestones: advancing a pipeline of rare disease programs for rapid validation while expanding toward larger indications like ALS and Alzheimer’s.
Beyond the lab, Avery has also co-founded and leads BioSpark Group, a nonprofit dedicated to fostering biotech dialogue and innovation. It reflects her belief that science thrives in community and that resilience grows when challenges are faced together.
The road ahead for Riboway is ambitious, but Avery is no stranger to overcoming barriers. Just as she transformed her own trajectory—rising from a remote village to lead a biotech shaping the future of medicine—she now leads Riboway in transforming what’s possible in drug development. Because just as Avery changed her own fate through resilience and hard work, Riboway is focused on changing the fate of our genes: making every therapeutic target druggable.
