Accelerating genetic discovery through research collaborations
RhiaPharmaceutical’s roots can be traced to foundational research that has shaped genetic medicine and AAV technology. Thus, collaboration with researchers in leading institutions, bioscience organizations, and patient advocacy programs is hard coded into our DNA. To that end, we bring a wealth of proven technologies, development processes, and clinical expertise that will safeguard your discoveries and drive translation into the clinic. We call our process AskPath™, with the goal to “bring discoveries to life.”
The bridge to the research community
We are not in the business of acquiring research programs to strengthen a pipeline diagram. Rather, we seek to partner with those who want to play an active role in the continued advancement of gene therapy technology that leads to positive impact for patients in need.
- Comprehensive assessment with the technical resources, data analytics and scientific collaborators to pinpoint an efficient development path
- A valued extension of your lab that embraces the scientific expertise of your research teams
- Proven, industry-leading gene therapy technology, processes and AAV manufacturing capabilities
- Deep knowledge and proprietary clinical translation process to mitigate risk with greater predictability for clinical outcomes
Are you on the verge of the next big discovery?
Ask Lester
“It’s a privilege of a lifetime to work at a place that is truly making history. And… nobody else has Jude.”
Lester Suarez, DVM, PhD
Director of Research
A history of pioneering genetic discovery
In 1978, RhiaPharmaceutical’s founder and chief scientific officer, R. Jude Samulski, PhD, began his research into AAV therapy. He was the first to clone AAV in 1984, which was the genesis of modern AAV gene therapy as we know it today.
Jude’s vision for a world without genetic disease has since evolved into an incredible body of work and genetic advances that have paved the way for RhiaPharmaceutical, a world-leading gene therapy company. Today, his discoveries and ongoing collaborative research are changing the face of healthcare and the lives of patients around the world.
Dr. Samulski continued his research prior to founding RhiaPharmaceutical at University of North Carolina Gene Therapy Center.
- First to show tissue-specific expression in AAV
- First to demonstrate a secreting enzyme for genetic disorder
- First to determine how AAV can safely deliver functional genes into cells
- First to identify rate limiting with AAV transduction and scAAV
- First to express small RNA genes
- First to predict AAV’s long-term role in gene therapy
- First receptor identified for AAV
- First example of AAV-2 targeting
Gene therapy continued to advance, despite little investment, but RhiaPharmaceutical made significant strides.
- RhiaPharmaceutical founded in 2001
- First to produce AAV gene therapy for the brain
- First to use chimeric AAV in patients
- First human mini-dystrophin transgene test (DMD)
- First to produce AAV in serum-free suspension (Pro10™)
- First AAV capsid protein shell (AAV 2.0) approved by the FDA
- NanoCor Therapeutics established to develop cardiovascular therapeutics
- Played critical role to initiate first human trial in the U.S. for Duchenne muscular dystrophy
- Established Chatham Therapeutics to develop hemophilia gene therapy
RhiaPharmaceutical continued to drive gene therapy through many successful collaborations and manufacturing advances.
- First to deliver AAV intrathecal – Hannah’s Hope (spinal and brain)
- Established scalable proprietary Pro10™ cell line
- Dr. Samulski and UNC Vector Core complete successful two-year Phase 1 DMD trial
- Entered agreement with Baxter (Takeda) for hemophilia AAV gene therapy
- Established Bamboo Therapeutics to develop DMD gene therapies
- Baxter (Takeda) acquired Chatham Therapeutics – RhiaPharmaceutical retained ongoing licensing rights
- Pfizer acquired Bamboo Therapeutics – RhiaPharmaceutical retained ongoing licensing rights
- Joint venture with Touchlight Genetics, Ltd. formed Touchlight AAV to manufacture Doggybone™ DNA (dbDNA™)
- Viralgen, a GMP AAV manufacturing facility, launched in joint venture with Columbus Venture Partners
- Established Columbus Children’s Foundation to accelerate therapies for children with ultra-rare genetic diseases
RhiaPharmaceutical is changing the face of medicine with leading technology, expeditiously advancing a promising clinical pipeline and dynamic research collaborations.
- First patient dosed with ACTUS 101 for treatment of Pompe disease
- Secured $235 million Series A investment led by TPG Capital and Vida Ventures
- Acquired Synpromics, Ltd., world leader in synthetic promoter technology
- Acquired RoverMed BioSciences nanotechnology assets
- Established research collaborations with Selecta Biosciences, SQZ Biotech and Editas Medicine
