Targeted Approaches to Treat

Neurological and Psychiatric Disorders

About Us

Welcome to Redpin Therapeutics. We are a privately held, preclinical stage gene therapy company based in New York City. We are developing a proprietary chemogenetics platform for targeted cell therapies that address currently intractable diseases of the nervous system. Our approach integrates powerful principles from synthetic biology, gene therapy, and traditional pharmacotherapy.

Our science is based on the groundbreaking discovery and development of an ultrapotent ion channel-based chemogenetics platform that enables targeted cell activation or inhibition controlled by low doses of the anti-smoking drug varenicline (CHANTIX®). We have a worldwide exclusive license from the Howard Hughes Medical Institute for the therapeutic use of this technology, and we are working with urgency to apply it to advance important medicines for patients in desperate need of effective therapies.

Science

The Need: Targeted Approaches to Treat Neurological and Psychiatric Disorders

Central and peripheral nervous system function is built around electrical activity. Neurological and psychiatric diseases and disorders are caused by local neuronal dysfunction, typically either over-active or under-active neural circuits.

Current treatment of neurological and psychiatric diseases and disorders typically involves the use of systemic drugs to address local neuron dysfunction. However, traditional systemic therapeutics for targeted dysfunctions can have limited efficacy due to adverse, off-target side effects.

At Redpin, we are pioneering a novel targeted cell therapy approach to address neural circuit dysfunction. Our technology is designed to selectively activate or silence disease-causing neurons, modulating only dysfunctional neurons while leaving normal functioning cells alone.

Our approach has the potential to deliver effective treatments for a wide range of currently intractable neurological and psychiatric diseases and disorders, bringing much needed help to patients.

Our Ion Channel-Based Chemogenetics Platform: A New Paradigm of Targeted Cell Therapies

Chemogenetics is a ground-breaking approach to selectively control cell function by installing an engineered receptor that renders any targeted cell population sensitive to modulation by an agonist designed to target that receptor. At Redpin, we are building an innovative ion channel-based chemogenetics platform that introduces a new paradigm for tunable, targeted cell therapies.

Ion channels are proteins that are responsible for electrical activity in cells. We are leveraging ion channels as neuromodulation tools to either stimulate under-active neurons or inhibit over-active ones.

Using our synthetic biology platform, we have designed proprietary chimeric ligand-gated ion channels as targeted cell therapies to selectively stimulate or inhibit dysfunctional neuronal circuits implicated in a given disease.

Our ion channel receptors are designed to only modulate neuron activity in the presence of the orally delivered small molecule drug varenicline. Varencline (CHANTIX®) is approved by the U.S. Food & Drug Administration (FDA) as an anti-smoking agent. We believe varenicline is particularly attractive for chemogenetic applications in the central nervous system because it is well tolerated by patients at low doses and has excellent brain penetration.

Our Approach

1. Design

Rationally design a proprietary chimeric ion channel to activate or silence specific neurons.

2. Deliver

Insert the engineered ion channel gene into a viral vector (adeno-associated virus, or AAV) and administer to the patient. The AAV releases the encapsulated ion channel gene into the disease-causing neurons which then expresses the chimeric ion channel selectively in those neurons.

3. Activate

The patient takes the oral drug varenicline. This activates our ion channel receptors on targeted neurons, stimulating or silencing their activity to treat a given disease.

Publications

Pioneering research led by our scientific co-founder Scott Sternson, Ph.D., Group Leader at the Janelia Research Campus of the Howard Hughes Medical Institute (HHMI), serves as the scientific foundation for Redpin’s platform.

Sternson, SM et al, Ultrapotent Chemogenetics for Research and Potential Clinical Applications, Science, 14 Mar 2019 (online publication)
Sternson, SM et al, Chemical and Genetic Engineering of Selective Ion Channel–Ligand Interactions, Science, VOL 333 pp. 1292 – 1296, 2 September 2011

Leadership

Redpin Management Team

Elma S. Hawkins, Ph.D., MBA

President and CEO, Co-Founder

David Bleakman, Ph.D.

Chief Scientific Officer

Redpin Founders

Jeffrey M. Friedman, M.D., Ph.D.

Marilyn M. Simpson Professor, The Rockefeller University; Investigator, Howard Hughes Medical Institute

Scott Sternson, Ph.D.

Group Leader, Howard Hughes Medical Institute, Janelia Research Campus

Michael Kaplitt, M.D., Ph.D.

Vice Chair, Research, Neurological Surgery, Professor of Neurological Surgery, Director, Movement Disorders and Pain, Weill Cornell Medicine

Sarah Stanley, MBBCh, Ph.D.

Assistant Professor, Diabetes, Obesity and Metabolism Institute, Icahn School of Medicine Mount Sinai

Jonathan S. Dordick, Ph.D.

Howard P. Isermann Professor of Chemical and Biological Engineering, Center for Biotechnology & Interdisciplinary Studies, Rensselaer Polytechnic Institute

Elma S. Hawkins, Ph.D., MBA

President and CEO, Redpin Therapeutics

News

Recent Media Coverage

Neurons Controlled Using Anti-Smoking Drug

Genetic Engineering & Biotechnology News | March 15, 2019

Researchers Use Pfizer’s Anti-Smoking Drug Chantix to Control Neurons

BioSpace | March 15, 2019

Using Pfizer’s Smoking-cessation Treatment Chantix to Control Neurons for Drug Research

FierceBiotech | March 14, 2019

Chemogenetics Method Uses Anti-Smoking Drug to Control Cells

The Scientist | March 14, 2019

A New Chemogenetic Toolset

American Association for the Advancement of Science | March 14, 2019

Using An Anti-Smoking Drug to Control Neurons

Howard Hughes Medical Institute | March 14, 2019

Eli Lilly’s David Bleakman joins Redpin as Chief Scientific Officer

Xconomy | October 16, 2018

New York Biotech Snaps Up Eli Lilly Neuro Veteran as CSO

FierceBiotech | October 15, 2018

Contact Redpin

Please direct all inquiries to Elma S. Hawkins, Ph.D., M.B.A.