Therapies 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.   Founded by Susan P. Perrine, MD and Douglas V. Faller, MD, PhD, Phoenicia is dedicated to developing innovative solutions for Orphan Global Diseases. Phoenicia discovered  a new generation therapeutic for both Sickle Cell Disease and Beta-Thalassemia.

Their lead candidate (PB-04), identified for repurposing as a therapeutic for these hemoglobinopathies, is highly active in highly-predictive animal models,and has a decades-long record of clinical safety. The drug candidate was selected as a project by the Therapeutics for Rareand Neglected Diseases program at the NIH’s National Center for Advancing Translational Sciences,  which conducted studies required for initiating clinical trials in patients.

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 multiple and launched international clinical trials from academia, biotech or from pharma. Successful drugs included the first monoclonal antibody, global developmentand launch of a hematopoietic stimulant, a viral gene activation therapy in trials for viral-associated lymphomas,and a therapy to induce fetal genes to treat two genetic blood diseases.   Therapeutic Discovery Programs are targeted to activate silenced genes to replace the function of mutant alleles, or disrupt pathologic signaling pathways in hematologicand oncologic diseases. Currently the Founders are seeking partners or licensors for 2 candidates.

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Our Consulting Services were established to assist academic investigators in bringing their discoveries to clinical testing in patients worldwide. We consult on FDA-required manufacturingand toxicology studiesand provide introductions to industry specialists in regulatory, manufacturing, business basics, intellectual property lawand 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 discoveryand clinical testing.

Research / Discovery Programs

  • 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, transplantationand chemotherapy, or inherited cytopenias