Dr. Ben Wolozin, M.D., Ph.D., CSO and co-founder of Aquinnah Pharmaceuticals, pioneered the groundbreaking connection between stress granules and neurodegenerative diseases — a discovery now recognized as one of the most important in the field, offering a beacon of hope for patients and their families. Aquinnah technology leverages its extensive expertise in unraveling the intricate interplay of RNA-binding proteins to form biomolecular condensates, such as stress granules. Aquinnah is strategically targeting its technology to remove the pathologic biomolecular condensates (containing particularly “sticky” proteins) that contribute to disease progression in Amyotrophic "Lateral Sclerosis (ALS), also known as Lou Gehrig’s disease, Alzheimer’s disease, and other neurological disorders.

Our profound understanding of this biological landscape has empowered us to identify and develop new innovative small molecule therapeutics, administered as a pill, to address the pressing unmet medical needs for effective neurodegenerative disease treatments.

The robust foundation of our scientific approach is forged through a longstanding commitment to neurodegenerative research, unwavering standards, and a pioneering, out-of-the-box mindset. We've garnered support from major pharmaceutical companies including Abbvie, Pfizer, and Takeda, in addition to securing numerous peer-reviewed grants from the National Institute of Health and other leading foundations supporting innovative brain research.

While neurodegenerative drug development traditionally carries inherent risks, our seasoned and successful drug discovery and development team has made remarkable progress, introducing novel strategies to surmount these challenges and transform possibilities into realities.

As the population ages, the prevalence of neurodegenerative diseases in the United States is poised to experience a significant surge. Without groundbreaking advancements in treatment, projections indicate that the number of individuals affected by Alzheimer’s disease will double by the year 2050. Similarly, ALS is anticipated to witness a staggering 70% increase by 2040.

A diagnosis of ALS or Alzheimer’s disease presents patients with limited therapeutic options. Currently, there are only a handful of approved treatments for these debilitating conditions, providing only modest improvements.

increase in the 65+ U.S. Population with Alzheimer’s Dementia

ALS stems from the loss of motor neurons in the brain and spinal cord, crucial for controlling muscle movement. The deterioration of these neurons leads to the gradual loss of one’s ability to walk, talk, swallow, and ultimately breathe. ALS typically progresses rapidly, claiming the lives of most patients within just three to five years following diagnosis. 

In the case of Alzheimer’s disease, symptoms manifest as a gradual decline in memory and executive function—the capacity for complex reasoning and decision-making. Few ailments evoke such profound fear — fear of its onset, fear of losing one's identity and connection to loved ones, and fear of relinquishing control over one's life.

For more information:

• National Institute of Health

• ALS Association

• Alzheimer's Association