
We can reverse the course of disease.
Generian is working to restore cellular function and modulate natural signaling networks to reverse the course of disease.


Aging is the predominant risk factor for most diseases.
Over time, cells no longer function as they should – impacting signaling networks that drive cellular stress, leading to chronic disorders. Generian is using insights about the natural process of cellular aging to develop therapies that restore signaling through the body’s natural biological networks.

The Approach
Generian is utilizing a proprietary, high-throughput screening platform that enables the discovery of monovalent small molecules that alter the activity or stability of previously undruggable proteins. We are focused on developing therapeutic candidates for chronic diseases that have limited treatment options.

The Programs
Leveraging decades of research from some of the foremost experts in age-related diseases and E3 ligase biology, Generian has identified network regulators (TFEB and AMPK), which are validated therapeutic targets essential to cellular function that diminish with age, disease or genetic mutations.
TFEB (or Transcription Factor EB)
plays an essential role in cellular quality control by regulating autophagic flux and lysosomal biogenesis.
AMPK (or AMP-activated protein kinase)
helps to maintain cellular fitness, playing an essential role in nutrient sensing and cell metabolism.

Perspectives from our Leadership team
“Our work at Generian is focused on translating research into drugs that can reverse the course of disease in humans. The goal is to help people live better longer.”
Toren Finkel, MD, PhD
CSO

“The Generian discovery platform we have built enables rapid and scalable target discovery through the use of proprietary tools and assays that pinpoint relevant target proteins, identify specific E3 ligases and develop small molecules for further studies.”
Bill Chen, PhD
VP, Drug Discovery

“The therapies we are developing are designed to inhibit specific E3 ligases, thereby preventing the degradation of beneficial target proteins to enhance cellular response and exert a beneficial effect.”
Yuan Liu, PhD
VP, Biology

News
June 29, 2022
Generian and Astellas Enter into Collaboration and Exclusive License Agreement to Discover and Develop Novel Small Molecules For Undruggable Targets
Generian and Astellas Enter into Collaboration and Exclusive License Agreement to Discover and Develop Novel Small Molecules For Undruggable Targets
Pittsburgh, PA and Cambridge, MA, June 29, 2022 – Generian Pharmaceuticals, Inc. (“Generian”) and Mitobridge, Inc. (a wholly owned subsidiary of Astellas Pharma Inc.) announced today that they have entered into a collaboration and exclusive license agreement to discover and develop novel small molecules for undruggable therapeutic targets by using a proprietary drug discovery platform for diseases that have limited treatment options.
Under the terms of the agreement, the companies will jointly conduct research and preclinical development activities to identify novel monovalent small molecules that modulate target proteins through activation, stabilization or degradation as potential development candidates. Astellas will be solely responsible for clinical development, manufacturing and commercialization of all products arising from the joint research activities. Generian will receive an upfront payment and is eligible to receive success-based milestone payments that could result in payments of over $180 million, along with single digit royalties on global net sales of those products.
“We are excited to collaborate with Astellas and believe our strategy can successfully mine for candidate drugs for therapeutically relevant targets that are currently considered undruggable,” said Hank Safferstein, PhD, JD, Chief Executive Officer of Generian. “Our small molecule discovery approach allows us to rapidly screen and identify potential drug candidates in an entirely new way in order to develop first-in-class medicines.”
“Our collaboration with Generian is an exciting and transformative opportunity to accelerate our drug discovery activities in the area of undruggable target space,” said David Barrett, Ph.D., Division Head and President of Mitobridge. “We are very excited to work with a world-class team of collaborators to deliver significant new treatment options for patients suffering from currently intractable or poorly served diseases.”
About Generian
Generian Pharmaceuticals, which was formed by UPMC Enterprises, the innovation, commercialization and venture capital arm of UPMC, is a private biotechnology company that is focused on developing small molecules to therapeutically modulate protein stability. By utilizing a proprietary platform, the company is advancing a pipeline of first-in-class drugs for clinically meaningful, difficult-to-drug targets. Learn more at www.generian.com or follow us on LinkedIn.
About Mitobridge
Mitobridge Inc. was founded in 2013 with financing from MPM Capital, Longwood Fund, and Astellas Pharma’s Venture Management team, all sharing a vision for the promise of mitochondrial-targeted therapeutics. In January of 2018, Mitobridge was acquired by Astellas Pharma and is now Mitobridge, an Astellas Company. At Astellas, mitochondrial biology is one of our Primary Focuses, and this biology platform drives our commitment to bring new biological concepts into the clinic for rapid proof of concept.
About Astellas
Astellas Pharma Inc. is a pharmaceutical company conducting business in more than 70 countries around the world. We are promoting the Focus Area Approach that is designed to identify opportunities for the continuous creation of new drugs to address diseases with high unmet medical needs by focusing on Biology and Modality. Furthermore, we are also looking beyond our foundational Rx focus to create Rx+® healthcare solutions that combine our expertise and knowledge with cutting-edge technology in different fields of external partners. Through these efforts, Astellas stands on the forefront of healthcare change to turn innovative science into value for patients. For more information, please visit our website at https://www.astellas.com/en.
Astellas Cautionary Notes
In this press release, statements made with respect to current plans, estimates, strategies and beliefs and other statements that are not historical facts are forward-looking statements about the future performance of Astellas. These statements are based on management’s current assumptions and beliefs in light of the information currently available to it and involve known and unknown risks and uncertainties. A number of factors could cause actual results to differ materially from those discussed in the forward-looking statements. Such factors include, but are not limited to: (i) changes in general economic conditions and in laws and regulations, relating to pharmaceutical markets, (ii) currency exchange rate fluctuations, (iii) delays in new product launches, (iv) the inability of Astellas to market existing and new products effectively, (v) the inability of Astellas to continue to effectively research and develop products accepted by customers in highly competitive markets, and (vi) infringements of Astellas’ intellectual property rights by third parties. Information about pharmaceutical products (including products currently in development) which is included in this press release is not intended to constitute an advertisement or medical advice.
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Generian Contact:
Media and all other inquiries
info@generian.com
Astellas Contact:
Astellas Pharma Inc.
Corporate Advocacy & Relations
+81-3-3244-3201