AXONIS Therapeutics, Inc. is a Boston-based neuro-focused biotechnology company developing medicines targeting KCC2, the mediator of inhibition within the brain. The company is self-described to have a proprietary KCC2 discovery engine based on several years of know-how.

Axonis Therapeutics

AXONIS Therapeutics, Inc.

Axonis Therapeutics | 2,815 followers on LinkedIn. Axonis Therapeutics is advancing breakthrough discoveries to develop first-in-class therapies for neurological disorders | Axonis Therapeutics is a Boston-based, neuro-focused biotechnology company developing first- and best-in-class medicines targeting KCC2, the critical mediator of inhibitory neurotransmission within the brain, by translating breakthrough discoveries spun out from Boston Children’s Hospital, Harvard and Laval University.

The company has built a proprietary KCC2 discovery engine, based on several years of world-leading know-how, to become the leaders in this potentially blockbuster drug space. Axonis’ lead development candidate, AXN-027 is a first-in-class oral small molecule designed to potentiate the function of KCC2, a major CNS chloride transporter essential for inhibitory neurotransmission, for the treatment of epilepsy and pain.

Bob Yant

Linda Jones

Chief Medical Officer | Clinician-scientist with clinical practice, research and management experience in academic and industrial settings

Donald M.

Senior Director, Finance & Operations at Axonis Therapeutics

Mike Wiecek

Thrilled to share that the New York Stock Exchange celebrated our Series A financing on the trading floor after yesterday’s opening bell. Thanks, NYSE for the recognition!

Axonis is very excited to share the news that we have raised a $115 million Series A financing to support the development of novel neuromedicines targeting KCC2. The financing was co-led by Cormorant Asset Management, LP and venBio , with significant investments from Sofinnova Investments , MRL Ventures Fund LLC , Perceptive Advisors , Solasta Ventures and Lumira Ventures . Our lead program, AXN-027, is being advanced for the treatment of epilepsy and pain. https://lnkd.in/eXWMmc7v

Looking forward to another great World Orphan Drug Congress USA in 2025! Excited to share the details about our panel discussion on 'Indication selection considerations for orphan drugs with broad therapeutic potential' closer to the event, a topic highly relevant for Axonis Therapeutics first-in-class oral KCC2 potentiators. The WODC is always an excellent cross-functional meeting that attracts a broad range of stakeholders passionate about rare diseases and orphan drug development

Shane Hegarty

It was a great honor to be able to pitch Axonis Therapeutics at this week's inaugural #Boston #Biotech #Investor #Day . Thanks to BioLabs , LabCentral , and The Engine Accelerator for the opportunity and for hosting! A fantastic event the with a great community of founders, investors and sponsors brought together, and it was a pleasure to learn about so many incredible biotech start-ups, as well as from the excellent keynote and panel sessions. Well done to Adam Jenkins , Rachele Ryan , Spencer Plourde , Lynn Walder , Kaitlyn Kozlowski and the rest of the team for organizing, and hope it becomes an annual gathering! photo credit: Rebecca Allan, PhD - one of our very helpful and supportive investors!

Thanks Adam Stasio and Nick Minio for hosting me on the SciMed #Biotech #Podcast , and I thoroughly enjoyed our conversation. Appreciate the opportunity to share the journey of Axonis Therapeutics and myself in recent years, and our vision/plans of transforming #neurology with our first- and best-in-class oral #KCC2 drug to treat #brain disinhibition and #drug resistance in a range of neurological disorders

As an Irish scientist, pushing #innovation in #space did not seem possible when I started my research career. But through the Boeing and ISSNL ‘Technology in Space’ prize Axonis Therapeutics were able to make exciting #biotechnology advances, and I will always be grateful to MassChallenge for the opportunity. Truly appreciate the invitation from International Space Station National Laboratory to share our story today at the inspirational ISSRDC conference in Boston. Onwards and upwards! One exceptional piece of advice from Davide Marotta, Ph.D. for pursuing #microgravity research was ‘think big outside the box, and then fit it inside a tiny box’, which resonates with our own experience of operationalizing our project on orbit. Excited about next steps

Honored to be invited to speak at this ' #Startups in #Space ' fireside chat next week at 'International Space Station Research & Development Conference' in Boston, with a great panel from MassChallenge and other alumni companies who were awarded the unique #TechnologyinSpace prize. Not my usual conference setting, but an exciting opportunity to meet NASA - National Aeronautics and Space Administration astronauts and other trailblazers who push the boundaries of #science in space by leveraging the unique conditions onboard the #ISSNL . I hope that discussing Axonis Therapeutics project at the #ISSRRDC , which established 'rapid 3D self-assembled iPSC-derived #human #brain models in microgravity', can inspire next generation neurological disorder models for #precision #neuroscience and #therapeutic development.

𝐒𝐩𝐢𝐧𝐚𝐥 𝐂𝐨𝐫𝐝 𝐈𝐧𝐣𝐮𝐫𝐲 𝐈𝐧𝐯𝐞𝐬𝐭𝐨𝐫 𝐒𝐲𝐦𝐩𝐨𝐬𝐢𝐮𝐦: Axonis Therapeutics ' CEO Joanna Stanicka discusses the importance of KCC2 inhibition in CNS disorders. She describes how Axonis is developing a small molecule that is KCC2-potentiating, with the idea to restore the role that KCC2 inhibition plays in neuronal function. Full video: https://lnkd.in/gCcbGZxM BiotechTV is brought to you by: Mispro | Breakthrough Properties | HSBC Innovation Banking | Theragent Inc | NYSE | Allucent

BiotechTV

Shane Hegarty is the Chief Scientific Officer of Axonis Therapeutics , a Boston-based Biotech targeting KCC2 to revive neurons and treat people suffering from neurological disorders. Shane joined me on the Careers in Discovery podcast to share the story of his transition from academic to Biotech entrepreneur, why communication, persistence, collaboration and focus are critical skills for success, his amazing track record of securing grant funding, and much more. Come listen to the interview now at https://lnkd.in/eWmf98mu - it's insightful stuff!

Tom Froggatt

Terrific discussion on what it takes to progress science with Teresa Bitetti Takeda Sekar (Sek) Kathiresan Verve Therapeutics John Lepore ProFound Therapeutics Søren Møller Novo Holdings Joanna Stanicka Axonis Therapeutics Brent Vaughan Cognito Therapeutics, Inc.

AXON

US:AXONIS

AXONIS Therapeutics, Inc.

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