Rgenix is developing first-in-class drugs that target key pathways in cancer progression. Using a discovery platform developed by Rgenix founding scientists at Rockefeller University, we have discovered several novel cancer targets that drive tumor growth and cancer progression. These targets regulate key components of the tumor micro-environment including immune cells and cancer metabolism pathways.

Our discovery platform - which can be applied to virtually any solid tumor type - has yielded novel therapeutic targets for several high-unmet cancer types to date. We aim to develop effective therapies against these targets that will provide durable and meaningful responses for cancer patients that lack effective therapies.

Our lead therapy RGX-104 is a small molecule immunotherapy that blocks the ability of tumors to evade the immune system. RGX-104 has obtained FDA Orphan Drug Designation in several solid tumor types of high-unmet need and will enter clinical trials in the second half of 2016.


Science underlying Rgenix’s technology and pipeline has been published in leading scientific journals.


Rgenix has multiple therapeutic programs in development, impacting several prevalent cancer types

Rgenix's product pipeline addresses cancers diagnosed in more than 500,000 patients annually in the US, including drug-resistant cancer types. These cancers result in more than 250,000 deaths per year in America, with 5-year survival rates often as low as 10%-20% for patients with metastatic disease. Rgenix has exclusive worldwide rights to each of its programs below.


RGX-104 is an orally bioavailable small molecule immunotherapy that targets LXR. Rgenix in-licensed RGX-104 from GlaxoSmithKline as part of a world-wide exclusive licensing agreement. RGX-104 targets a key immune cell type in the tumor micro-environment, reversing immune evasion and resulting in robust anti-tumor activity in established animal models of various cancer types, including drug-resistant tumors.  In pre-clinical models, RGX-104 has demonstrated remarkable single-agent activity as well as synergy with approved therapies, such as immune check-point inhibitors. RGX-104 has obtained FDA Orphan Drug Designation in several solid tumor types of high-unmet need, and will enter clinical trials in the second half of 2016.


RGX-202 is a small molecule that inhibits a novel cancer metabolism pathway involved in supplying energy to cancer cells. The target of RGX-202 is genetically amplified in several prevalent cancer types, including colon cancer, lung cancer and lymphoma. RGX-202 has demonstrated anti-tumor activity in several tumor models in pre-clinical studies, both as a single agent as well as in combination with standard-of-care therapies. RGX-202 will enter clinical trials in 2017.


RGX-014/019 are two monoclonal antibodies that target independent components of a key pathway that drives tumor progression and metastasis of several cancer types, including triple-negative breast cancer.


Lung Cancer


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Colorectal Cancer


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Trip Neg Breast Cancer


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Addressable Cancers


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Rgenix’s management comprises biotech professionals with deep scientific, clinical and operational expertise.


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Jun 15, 2016

Rgenix Announces $33 Million Series B Financing to Develop First-in-Class Cancer Therapeutics

Jun 15, 2016

Novo A/S and Sofinnova Partners are leading this latest financing round

NEW YORK, New York ― June 15, 2016 ― Rgenix, a cancer therapeutics company developing first-in-class drugs targeting novel cancer pathways, announced a $33 million Series B financing led by Novo A/S and Sofinnova Partners, with participation from existing investors including Partnership Fund for New York City, Alexandria Venture Investments, and Conegliano Ventures LP. The financing will support clinical development of Rgenix’s lead drug candidates, RGX-104 and RGX-202, as well as further development of its therapeutics pipeline. 

“We are thrilled to have attracted top-tier investors to advance development of our novel cancer therapeutics,” said Masoud Tavazoie, M.D. Ph.D., Chief Executive Officer and co-founder of Rgenix. “This financing validates the potential of our lead immunotherapy RGX-104, which will be entering clinical trials this fall, and also demonstrates the strength of our discovery platform, developed in the laboratory of Rgenix co-founder Dr. Sohail Tavazoie at The Rockefeller University. The funding will enable our team to deliver an innovative therapy to cancer patients while simultaneously pushing forward our pipeline of other novel drug candidates.”  

Antoine Papiernik, Managing Partner at Sofinnova Partners, commented: “We are very excited to back such a high quality team at Rgenix. We also believe that RGX-104 could revolutionize treatment to cancer patients that today lack effective therapies.”

Despite recent advances in cancer therapy, most patients will eventually succumb to their disease due to drug resistance and immune evasion. RGX-104 is a small molecule that reverses immune evasion and drug resistance by targeting immunosuppressive cells in the tumor microenvironment via a novel pathway, resulting in strong anti-tumor activity in several drug-resistant cancer types in pre-clinical models, both as a single agent and in combination with approved immunotherapies such as PD-1 inhibitors. The target of RGX-104 was discovered using Rgenix’s miRNA platform that has yielded several new cancer targets across multiple prevalent cancer types.

“We are excited to support Rgenix’s novel approach to treating cancers of high unmet need,” said Nilesh Kumar, Senior Principal of Novo Ventures*. “Rgenix has an exciting platform founded on strong science from The Rockefeller University; the lead program is a first in class opportunity addressing a key mechanism in tumor immunosuppression in various cancer types.”

In connection with the financing, Nilesh Kumar of Novo Ventures and Antoine Papiernik of Sofinnova Partners will join the Rgenix Board of Directors together with existing members, including Executive Chairman Eric Rowinsky, M.D., Masoud Tavazoie, M.D. Ph.D., Nancy Chang, Ph.D., and Saeed Tavazoie, Ph.D.

Jun 15, 2016

Rgenix Raises $33M, Preps Cancer Trial For Drug Shelved By GSK

Jun 15, 2016

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Jun 15, 2016

Rottweiler tested, Novo and Sofinnova back $33M round for Rgenix

Jun 15, 2016

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Aug 31, 2015

In New York, Rgenix Rises From Rockefeller and Takes Aim at Cancer

Aug 31, 2015

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