Turn on, tune in: New York biotech scores $15M+ in chemogenetics bet
Typically, once a biological target has been identified, scientists work on developing molecules focused on that target. But in an emerging field called chemogenetics, researchers start with a small molecule and then design a target for it.
Banking on its chemogenetics technology to address CNS disorders, New York-based Redpin Therapeutics has secured a $15.5 million injection, as it shepherds its two preclinical programs — for chronic pain and refractory epilepsy — into the clinic.
Existing gene therapies tend to replace defective genes with healthy ones. But Redpin’s technology uses AAV vectors to deliver engineered ion channels to specific cell populations. And then these engineered receptors, once activated or triggered by their ligand or the small molecule, control the functioning of that particular cell type.
The small molecule chosen by Redpin is Pfizer’s smoking cessation treatment, Chantix. The drug is approved in 80 countries, has suitable pharmacokinetic characteristics, and can also penetrate the blood-brain barrier, co-founder and CEO Elma Hawkins noted in an interview, adding that the company also has other small molecule-receptor pairs in the pipeline.
“Without Chantix’s presence the (engineered) ion channel is completely inert, and then with Chantix there, the ion channel gets activated,” Hawkins said. “And so basically you are turning the ion channel on and off with an external switch. And that switch is Chantix.”
Since the engineered receptors only respond to specific drugs, doses can be low, and the process limits the off-target systemic effects of the drug on other tissues — the once-and-done approach is also tunable and reversible.
These advantages lured London-based 4BIO Capital, which led the Series A injection. The VC shop has a $150 million fund focused on advanced therapies — including cell and gene therapies, RNA-based therapies and the microbiome.
“When we do treat patients with small molecules for neuroscience, psychology, and functional neurology over a long time, they do create quite significant side-effects,” noted managing partner Dmitry Kuzmin in an interview.
But cell and gene therapies have the edge — they are one-time treatments, and can be very precise, he said.
“Given the combination of the local surgical administration, the AAV advantages and the promoter control we can be very, very precise in the brain,” Kuzmin stressed. “We can achieve fantastic delivery to functionally and genetically distinct circuits in the brain and elsewhere in the body.”
Kuzmin is set to join Redpin’s board. The company also counts Eli Lilly veteran David Bleakman as its chief scientific officer.
4BIO, along with Arkin Bio Ventures, led the Series A round. Other investors such as the corporate venture arm of Takeda, New York Ventures and Alexandria Venture Investments, also participated in the round.
Versant, MPM Capital-backed Coda Therapeutics, which also has a chemogenetics platform, raised $15 million last November bringing its total Series A haul to $34 million — as it further develops its lead programs: chronic neuropathic pain and focal epilepsy.
Social image: Elma Hawkins via Youtube