pushing the boundaries
of neuroscience
About
Nido Bio is translating today’s neuroscience breakthroughs into tomorrow’s treatments for severe neurological diseases. Leveraging human genetics, we develop precision medicines that address the fundamental biology of disease and restore healthy cell function.
Led by dedicated drug development and neuroscience experts, world-class scientific advisors and an industry-leading investor syndicate, we aim to improve the daily lives of patients suffering from these conditions.
Approach
Most result in a progressive and debilitating loss of function with significant impact on patients’ daily lives. With no effective treatments available, Nido Bio is elevating transformative medicines from a nest of possibilities.
Neurological diseases are on the rise, posing a complex healthcare issue for doctors and scientists everywhere. Nido Bio is relentlessly pursuing real solutions on behalf of patients.
Programs
Nido Bio’s multiple programs have the potential to improve the daily lives of patients suffering from debilitating neurological disease.
Spinal and Bulbar Muscular Atrophy (SBMA), also known as Kennedy’s disease, is a rare inherited neuromuscular disorder that manifests in men causing progressive weakness and wasting of limb, facial and swallowing muscles, which results in impaired mobility, speech and swallowing.
1 of 4Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s Disease, is a progressive and rare neurological disease that results in the brain’s loss of control over voluntary movements.
2 of 4 Rare Tauopathies are a group of progressive neurodegenerative diseases characterized by aggregation and dysfunction of tau protein in the brain leading to cognitive, behavioral, language and movement disorders. 3 of 4Frontotemporal Dementia (FTD) is a progressive and rare neurological disorder that often begins at a younger age than other dementias and leads to marked changes in personality, behavior and/or ability to communicate.
4 of 4
Spinal and Bulbar Muscular Atrophy (SBMA), also known as Kennedy’s disease, is a rare inherited neuromuscular disorder that manifests in men causing progressive weakness and wasting of limb, facial and swallowing muscles, which results in impaired mobility, speech and swallowing.
1 of 4Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s Disease, is a progressive and rare neurological disease that results in the brain’s loss of control over voluntary movements.
2 of 4 Rare Tauopathies are a group of progressive neurodegenerative diseases characterized by aggregation and dysfunction of tau protein in the brain leading to cognitive, behavioral, language and movement disorders. 3 of 4Frontotemporal Dementia (FTD) is a progressive and rare neurological disorder that often begins at a younger age than other dementias and leads to marked changes in personality, behavior and/or ability to communicate.
4 of 4
Our clinical stage program, NIDO-361, is in development for the treatment of Spinal and Bulbar Muscular Atrophy (SBMA).
SBMA, also known as Kennedy’s disease, is a rare monogenic neuromuscular disorder that manifests in men. It causes progressive weakness and wasting of limb, facial and swallowing muscles, which results in impaired mobility, speech, and swallowing. For more information on SBMA, visit The Kennedy’s Disease Association website.
SBMA is caused by an X-linked genetic mutation of the androgen receptor (AR) gene that results in the loss of skeletal muscle and motor neuron function. NIDO-361 is a novel small molecule that binds to the BF3 site – a distinct site from where typical AR targeting small molecules bind – to restore healthy cell function.
Terry Thompson
President
Kennedy’s Disease Association
Additional pipeline programs center around a novel target with the potential to address multiple disease mechanisms and that has broad clinical application across neurodegenerative and peripheral inflammatory diseases.
The combined mechanisms of protein aggregation, neuroinflammation, and neuronal cell death create a self-sustaining pathological cycle that underpins many neurological and peripheral inflammatory diseases. We are focused on breaking this cycle by developing small molecule modulators of a novel target that can simultaneously activate the cell’s ability to clear protein aggregates (autophagy) while inhibiting neuroinflammation and cell death.
Platform
The opportunities to discover new precision medicines with our approach are significant.
We utilize a functional genomics discovery platform based on human iPSC-derived neurons and tailored screens to identify novel therapeutic targets for neurological diseases. We are collaborating with recognized world leaders in neurology to build this platform. Our initial screens are focused on amyotrophic lateral sclerosis (ALS) and frontotemporal disorders (FTD) and we are leveraging this platform to further build out Nido Bio’s pipeline.
Team
Chief Medical Officer
Scientific Director and Executive Vice President
St. Jude Children’s Research Hospital
President
Light Horse Therapeutics
Co-founder and Chairman
Rubedo Life Sciences
Chief Scientific Officer
Denali Therapeutics
Professor, Interdisciplinary Research Center on Biology and Chemistry
Shanghai Institute of Organic Chemistry
Senior Vice President, Neurodegeneration and Genetic Medicines
Eli Lilly and Company
Professor, Cambridge Institute for Medical Research, Department of Medical Genetics and UK Dementia Research Institute Group Leader
University of Cambridge
- & additional investors including Eli Lilly and Company
News
Nido Biosciences Presents Clinical Data Supporting the Continued Development of NIDO-361 in SBMA Patients at the 2023 International Kennedy’s Disease Patient Conference
Nido Biosciences Emerges as Clinical Stage Company Raising $109 Million to Advance Treatments for Debilitating Neurological Diseases
May 15, 2023
Join Us
Tackle scientific problems to make a difference in the lives of patient communities by asking the right questions, leveraging world-class talent, and striving for the highest level of scientific rigor.
Believe in our own and others’ words and actions. Openness to give and receive those words and actions without judgement.
COMMUNICATION
Create a safe environment where people feel encouraged to generate ideas and to voice their opinion.
FEARLESS INNOVATION
COLLABORATION
Effectively work together, utilizing the diverse skills and knowledge of the group to accomplish a common objective.
DIVERSITY
Encourage and respect heterogeneity of ideas, experiences, identities, and beliefs.
Testimonials
“The opportunity to impact patients while also deepening our understanding of neuroscience motivates me to work at Nido Bio. Taking each result and strategizing on what to do next in an open, attentive environment has afforded me many learning opportunities. At Nido Bio, we strive for excellence and challenge ourselves to answer difficult questions in pursuit of transformative therapeutics.”
Shawn Nelson
Senior Scientist, Medicinal Chemistry
“I love being around such talented, intelligent individuals every day. Our office environment is positive, collaborative, and team oriented. I chose Nido Bio because it’s exciting and rewarding to see the possibility of finding therapies to help patients combat crippling diseases.”
Loan Kraine
Accounts Payable/Payroll Manager
