Origami Therapeutics is taking a precision medicine approach to discover disease-modifying treatments for neurodegeneration caused by toxic protein misfolding.

Meet Origami Therapeutics

Origami Therapeutics, a discovery-stage company based in San Diego, is taking a precision medicine approach to discover disease-modifying treatments for neurodegeneration caused by toxic protein misfolding. Leveraging the Founder's experience in discovering transformational therapies for Cystic Fibrosis that modulate CFTR conformation, the Company's focus is to treat neurodegeneration by directly modulating the pathogenic proteins that cause disease. Our platform enables discovery of both protein degraders and conformation correctors, allowing us to match the best drug to treat each disease by using patient-derived disease models to ensure success in clinical trials. Origami plans to generate a pipeline of small molecule therapeutics that prevent or delay the onset and the progression of neurodegenerative diseases by targeting the underlying genetic cause of disease. Currently, we are selecting the optimal protein degrader molecule to advance into preclinical testing for Huntington's disease, a devastating fatal disease that strikes at the prime of life. We are seeking seed funding for our lead Huntington's disease asset in addition to research collaborations to initiate a program for a second disease indication.

Why is your company the best option to solve the problem you're solving?

Origami Therapeutics is the best option for generating a transformational therapy for Huntington's disease because we are targeting the underlying genetic and mechanistic cause of the disease using small molecules, a therapeutic modality consistent with simple, efficient delivery to all cells in the body including the central nervous system. The use of a small molecule avoids the need for invasive delivery, a trained neurosurgeon and a neurosurgical suite, a distinction from our competitors. By targeting the root cause of disease, we mitigate the risk of side effects caused by targeting more general cellular pathways. This increased benefit to risk ratio should enable treatment before symptoms develop, and more importantly, before brain cells die. These factors contribute to a therapy with the potential to prevent, halt or slow progression of disease, resulting in a normal quality of life and normal productivity for the patient.

What nuggets of info make you interesting? Why do people care about what you're doing?

Origami is a little unusual in the San Diego biotech ecosystem in that we arose neither from academic research nor as a spin-out/license from another company. Instead, we are a boot-strapped, home-grown San Diego company leveraging the founders' previous successes in multiple therapeutic areas with multiple companies. We do feel it is important to exploit the terrific expertise available in San Diego and that is why we collaborate and partner with local companies whenever possible. Besides the great entrepreneurial environment (such as JLABs where we are a proud resident), San Diego also provides a terrific opportunity to engage with patients and their families. We have long-standing interactions with the San Diego Chapter of the Huntington's Disease Society of America, the premier patient advocacy group, as well as key clinicians and researchers in the therapeutic space. We believe that the greatest successes arise from accepting the biggest challenges. That is why we were not afraid to strike out on our own to seek the best solution for the patients using all of the cutting-edge science available.

What else do you want us to know?

You never know where something seemingly trivial might lead. While I was a postdoctoral fellow at the National Institute of Mental Health (NIMH), I had the honor of presenting at a laboratory meeting in front of Julius Axelrod, our resident Nobel Laureate. You knew that you were giving a great presentation if you managed to keep Julie engaged and awake. Of course, reviewing the molecular cloning of the molecules responsible for the biology for which he won the Nobel Prize probably helped! Challenge met! Little did I know that this presentation would lead to a special speaking opportunity at Julie's 80th Birthday Symposium. That presentation in turn would lead to a series of drug-hunting opportunities in pharma and biotech, from the East Coast to the Heartland (Eli Lilly & Co) to Southern California (Amgen). Eventually I was recruited to the San Diego site of Vertex Pharmaceuticals, Inc. as Vice President of Discovery Biology. My experience contributing to the small molecule protein conformation modulators that have transformed the treatment for Cystic Fibrosis combined with my expertise in Neuroscience drug discovery led to the founding of Origami Therapeutics. Who would have thought that the challenge to keep a Nobel Laureate awake during a lab meeting would lead to becoming the Founder and CEO of a start-up company in San Diego?

BEHIND THE COMPANY

Founder/CEO: Beth Hoffman

What is your company's secret sauce?

Origami's secret sauce is our demonstrated ability to identify and develop protein conformation modulators combined with neuroscience drug discovery expertise.

What sets your company culture apart?

Blink by Malcolm Gladwell focuses on the phenomenon that your brain integrate multiple experiences without you even realizing it and that we use this "knowledge" without even thinking about it. It's the essence of going with your gut (by the way, your gut has its own brain). In my experience, the best decisions come from "going with your gut". This is what you intuitively understand. Whenever I've acted contrarily, the outcome is less than desirable. In the end, you have to trust yourself.

Origami Therapeutics in the News

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