Innovative Solutions for Orphan Global Diseases – Phoenicia BioSciences, Inc.
PHOENICIA BIOSCIENCES is a biopharmaceutical company developing proprietary small molecules that target specific genes for treatment of serious hematologic disorders. Started by Susan P. Perrine, MD and Douglas V. Faller, MD, PhD, Phoenicia is dedicated to developing innovative solutions for Orphan Global Diseases. They discovered an effective Therapeutic for both Sickle Cell Disease and Beta-Thalassemia. Their lead drug (PB-04), identified for repurposing as a therapeutic for these hemoglobinopathies, is very active in highly-predictive animal models, and has a decades-long record of clinical safety. The drug candidate was recently selected as a project within the Therapeutics for Rare and Neglected Diseases program at the NIH’s National Center for Advancing Translational Sciences. Through our collaboration with TRND researchers, we gain access to preclinical drug development expertise. The goal of the TRND program is to work with collaborators to advance potential treatments to human clinical trials.
Phoenicia Biosciences is dedicated to the discovery and development of therapies for serious medical conditions for which there is currently inadequate treatment, with a special focus on diseases affecting global populations.
Phoenicia scientists have developed therapeutics from discovery to bedside, together submitted 20 INDs, and launched international clinical trials from academia, biotech or from pharma. Successful drugs have included the first monoclonal antibody, global development and launch of a hematopoietic stimulant, a therapy for viral-associated lymphomas, and a therapy to induce fetal genes to treat two genetic blood diseases. Our Therapeutic Discovery Programs are targeted to activate silenced genes to replace the function of mutant alleles, or disrupt pathologic signaling pathways in hematologic and oncologic diseases. We currently have two Phase 2 drug candidates in addition to discovery-phase and preclinical-stage programs.
Our Consulting Services were established to assist academic investigators in bringing their discoveries to clinical testing in patients worldwide. We consult on FDA-required manufacturing and toxicology studies and provide introductions to industry specialists in regulatory, manufacturing, business basics, intellectual property law and clinical investigations. We provide guidance on studies “required” vs. “nice to have,” and information regarding programs which fund or perform drug development. The goal of our service is to assist others in bridging the “valley of death” between discovery and clinical testing.
- Therapeutic activation of developmentally silenced genes to replace the function of mutated hemoglobin genes
- Targeting a pathway which allows cancer cells to escape normal cellular death signals
- Targeting the virus as a therapeutic approach to virus-associated malignancies
- Refining therapies to heal refractory wounds
- Enhancing proliferation of hematopoietic cells to accelerate recovery from acute radiation sickness, transplantation and chemotherapy, or inherited cytopenias