Orpheris, Inc. was founded in 2015 as a spin-out of Ashvattha Therapeutics, LLC. It was created to develop the dendrimer technology to address unmet medical needs in orphan diseases. Orpheris licensed the exclusive rights for up to three products based on the dendrimer technology.
The dendrimer platform technology was exclusively licensed for all fields by Ashvattha from Johns Hopkins University, Wayne State University, National Institutes of Health, and the Kennedy Krieger Institute.
The dendrimer technology used in our products has been developed over several years of research and a significant amount of funding from the National Institutes of Health. Our founders, Sujatha Kannan, MD, and Kannan Rangaramanujam, PhD, have demonstrated that the hydroxyl poly (amidoamine) dendrimers used in our products selectively target neuroinflammation. In addition, our first selected product, OP-101, has the ability to treat brain injury in several small and large animal models. The selective targeting of this product to the activated microglia in the brain, from systemic administration, results in no detectable toxicity and rapid clearance from non-target organs. This enables ease of administration, increased efficacy due to targeting, and reduced side effects.
Orpheris intends to initially develop OP-101 for the treatment of childhood cerebral andrenoleukodystrophy (ccALD) and other neuroinflammatory diseases such as Parkinson's disease, ALS or Huntington's disease. Orpheris has met with the US FDA in pre-IND and Type C meetings and has established a development plan to treat ccALD patients that are not eligible for bone marrow transplant. These patients typically die within 2-5 years of diagnosis due to progressive neurodegeneration caused primarily by neuroinflammation and oxidative stress. There are no currently approved products for these initial indications for OP-101 and both indications represent a significant unmet medical need.
Orpheris in collaboration with Ashvattha is also evaluating additional product candidates for the treatment of cystic fibrosis (CF), Rett syndrome and autism spectrum disorders as well as other orphan diseases. The dendrimer platform technology has the ability to be effective in many disorders with an inflammatory component. Orpheris intends to advance these products after the initial clinical trial results from OP-101.