Advance Rare Disease Therapies With Us
Each of the over 7,000 identified rare diseases poses unique challenges in drug development. Northern Bio is here to help.
Rare Disease Research Expertise
At NorthernBio, we understand the unique challenges faced by researchers and foundations developing treatments and cures for rare diseases. We partner with these organizations to streamline and accelerate the path to getting these treatments to the clinic. Our experienced scientists have been working on rare diseases for over 20 years, and they understand the hurdles along the road to regulatory approval.
Consult a Scientist
We are a team of scientists who value open communication and collaboration. We work closely with our sponsors to provide customized solutions and expert support.
Targeted Delivery
Frequently Asked Questions (FAQs) about Rare Disease Research
What classifies a disease as rare or orphan?
The World Health Organization defines a rare disease as one that strikes fewer than 65 per 100,000 people.
What types of therapeutics are being developed for rare diseases?
There is a wide range of therapeutics being developed. These include but are not limited to Antisense oligonucleotides (ASOs), Adeno-associated viral vectors (AAVs), enzyme replacement therapies, small molecules, Small interfering RNA (siRNA), and CRISPR Gene editing.
What types of animal studies are required for regulatory approval?
The types of animal studies required are based upon the type of therapeutic being developed. For most therapeutics, regulatory agencies require studies to be conducted in two species that include a rodent and non-rodent species.
For more FAQs, visit the NIH Genetic and Rare Disease Information Center.
Partner With Us
Let’s work together to develop
life-changing treatments.
Join us in the pursuit of better lives through scientific innovation. Explore how Northern Bio can help you achieve your research goals.