Company Profile / Biotechnology / RNA Therapeutics
NextRNA Therapeutics
The Boston biotech that set out to drug the genome's non-coding majority - one RNA-protein handshake at a time.
The Boston biotech that set out to drug the genome's non-coding majority - one RNA-protein handshake at a time.
For most of the modern history of medicine, drug hunters have chased proteins. Proteins are the machines of the cell, and the roughly two percent of the human genome that codes for them has been the target of nearly every pill and injection ever approved. NextRNA Therapeutics was built on a different premise: that the other ninety-eight percent - the sprawling, non-coding portion of the genome once dismissed as filler - is a rich and largely untouched source of medicines.
The company, founded in 2020 and headquartered at 40 Guest Street in Boston, focused on a specific slice of that dark genome: long non-coding RNAs, or lncRNAs. These molecules do not make proteins. Instead they regulate gene expression, acting as switches and scaffolds that help decide which genes turn on and off. When those switches misfire, disease can follow - in cancer, in immune disorders, and in the nervous system.
NextRNA's core idea was deceptively simple and technically hard. lncRNAs do their work by binding to proteins. If you could design a small molecule - the same kind of compact, orally-available chemistry behind most conventional drugs - to wedge into that interaction and break it, you could switch a disease-driving RNA program off. The field had long labeled such RNA-protein interactions "undruggable." NextRNA built a company to prove otherwise.
The science traced back to the Dana-Farber Cancer Institute laboratory of Carl Novina, whose research on lncRNA biology formed the intellectual foundation. From that base the company built what it called a target and drug discovery engine: a systematic way to identify disease-relevant lncRNAs, map the proteins they grab onto, and screen for small molecules that pry them apart.
NextRNA is committed to leading the next revolution of RNA-directed therapeutics.
- The company's stated mission
Rather than betting on a single molecule, NextRNA built a repeatable process. The goal was to make lncRNA drug discovery systematic - not lucky.
Identify long non-coding RNAs that drive a specific disease.
➔Pinpoint the RNA-binding proteins the lncRNA depends on.
➔Screen for small molecules that disrupt the RNA-protein interaction.
➔Optimize selective compounds toward oncology and immunology programs.
A proprietary system to systematically identify disease-relevant lncRNAs, their interacting proteins, and selective small molecules to drug those interactions.
An early-stage small-molecule program targeting lncRNA-protein interactions in cancer - one of two programs advanced under the Bayer collaboration.
A discovery-stage effort aimed at dysregulated lncRNA-protein interactions implicated in immune-mediated disease.
Most RNA-focused biotechs - Alnylam, Ionis and their peers - build oligonucleotide drugs that must be delivered by injection. NextRNA instead pursued classic small molecules that could, in principle, be taken as pills. Within the smaller cluster of companies chasing RNA with small molecules - Arrakis, Skyhawk, Expansion, Gotham - NextRNA's distinguishing bet was its focus on long non-coding RNAs specifically, and on disrupting the interactions between those RNAs and the proteins that read them, rather than binding the RNA structure alone. Its business model paired that platform with a pipeline: build proprietary programs, then fund them through strategic pharma partnerships carrying upfront payments, research funding, milestones and royalties.
A year after inking a two-program oncology pact with Bayer worth up to $547 million, NextRNA began winding down - the science outpaced by the market.
- On the 2025 wind-down
Co-founder, President and CEO. Verhelle led NextRNA's scientific and corporate strategy from launch. Before founding the company she was Head of Academic Innovation at Takeda's Center for External Innovation, and earlier a Principal at Third Rock Ventures, where she helped launch Fulcrum Therapeutics and Cedilla Therapeutics. She brought more than two decades of R&D and company-building experience - and led one of the few women-run biotechs in the space.
Scientific founder. NextRNA's platform was built on Novina's long non-coding RNA research at the Dana-Farber Cancer Institute, which established the biological rationale for identifying disease-relevant lncRNAs and drugging their interactions with RNA-binding proteins.
Established in Massachusetts around lncRNA science from Carl Novina's lab at Dana-Farber Cancer Institute.
Launches publicly with $9.3M seed and a $46.8M Series A led by Cobro Ventures and Lightchain Capital; co-founder Dominique Verhelle is named CEO.
Advances its discovery engine and small-molecule programs across oncology and immunology.
Enters a strategic oncology collaboration and license agreement with Bayer to develop lncRNA-targeting small molecules.
Facing a weak biotech market and a delayed Bayer milestone, NextRNA announces a wind-down; Bayer continues the program independently.
Explore related interviews and coverage featuring NextRNA and CEO Dominique Verhelle.
It was a Boston biotech developing small-molecule drugs that target long non-coding RNAs (lncRNAs) by disrupting their interactions with RNA-binding proteins, with programs in oncology and immunology.
Co-founder Dominique Verhelle, PhD, MBA, served as President and CEO. The company's science was founded on the work of Carl Novina at Dana-Farber Cancer Institute.
Roughly $56 million in combined seed ($9.3M) and Series A ($46.8M, 2022) financing, led by Cobro Ventures and Lightchain Capital, plus a Bayer collaboration worth up to $547 million in potential payments.
In August 2024, NextRNA signed a strategic collaboration and license agreement with Bayer worth up to $547 million to develop lncRNA-targeting small molecules across two oncology programs, including upfront, milestones, and royalties.
In August 2025 the company announced it would wind down operations, citing weak biotech market conditions and a delayed Bayer milestone that would have extended its cash runway. Bayer said it would continue the underlying program.
Profile compiled from public sources including Business Wire, Bayer, Fierce Biotech, GBH, Crunchbase and PMLiVE. Funding and deal figures are as reported; the Bayer figure reflects total potential value including milestones. Details are approximate where public data is limited.