Our Science

Built on Rigorous Science

ProJenX is a clinical-stage biotechnology company developing novel, targeted brain penetrant therapies to address neurodegenerative diseases, with an initial focus on ALS.

At the heart of ProJenX’s approach is an innovative, patient-specific, cell-based drug discovery platform. Through a long-term research collaboration between Project ALS and researchers at Columbia University, our team drove breakthroughs in stem cell biology and created the first ALS patient-derived disease models. These models drove insights into what goes wrong with motor neurons, the brain cells affected by ALS – and how to rescue them.

At ProJenX, we are guided by ALS biology. Our lead candidate, prosetin, was identified in patient-derived models, developed for motor neuron protection, and optimized for brain penetrance.

Scientific Research

Read more about the scientific breakthroughs behind ProJenX.

January 2019 | The American Society of Gene and Cell Therapy

Prosetin: Changing the Trajectory of ALS

Prosetin is the first brain-penetrant MAP4K inhibitor therapy to enter clinical trials. ProJenX scientific co-founders at Columbia University have shown that prosetin is highly effective across multiple laboratory models of ALS.

What sets prosetin apart?

Penetrating the
Blood-Brain Barrier
Even though ALS is a brain disease, most drugs developed for ALS cannot cross the “blood-brain barrier” to reach motor neurons. We developed prosetin to preferentially reach the brain and target motor neurons directly.
ALS in a Dish
Prosetin’s inventors at Columbia University were the first to identify how to make a motor neuron from a skin or blood sample from someone with ALS. Their world-leading research means that prosetin was discovered using patient-derived laboratory models, and developed specifically to treat ALS.
While ALS is a complicated, heterogeneous disease, endoplasmic reticulum (ER) stress is a well-established common feature across ALS subtypes. By blocking MAP4K, we have shown that prosetin can rescue ER stress-mediated motor neuron loss in ALS models.

Pipeline Development:

Novel, brain-penetrant kinase inhibitors

Developing prosetin for people with ALS is our top priority. At the same time, we are exploring the role of MAP4K inhibition in treating other neurodegenerative disorders that trigger endoplasmic reticulum (ER) stress-mediated degeneration of neurons, and in other debilitating brain diseases in which this pathway play an established biological role.

Target Candidate Indication Discovery Preclinical Clinical
MAP4K Prosetin Parkinson’s disease
Undisclosed Undisclosed ALS

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