BioBlast Pharma, a clinical-stage biotechnology firm focused on developing orphan disease drugs, recently announced it has received an allowance from the United States Patent and Trademark Office (USPTO) regarding a method to address oculopharyngeal muscular dystrophy (OPMD) with Cabaletta. The Company showed in preclinical cell and animal models with Cabaletta positive results that led to a therapeutic program. It is currently being assessed in human clinical trials to treat two indications.
Colin Foster, who is the President & CEO of BioBlast said in a press release: “We are pleased to have received this notice of allowance which covers the significant innovations we have made in the stabilization of protein and prevention of protein aggregates within cells. Cabaletta is our most advanced platform, and is currently in Phase 2/3 for OPMD and Phase 2 for spinocerebellar ataxia type 3 (SCA3). We have developed this platform based upon a unique formula of trehalose, a well-known and safe disaccharide. We look forward to making further progress toward providing a therapy to help OPMD patients in need of treatment.”
OPMD is a hereditary myopathy that is characterized by difficulty in swallowing, known as dysphagia, and by the loss of muscular strength that results in weakness in several parts and regions of the body. As the dysphagia worsens and is more severe, patients tend to become malnourished, dehydrated, lose weight and suffer a major loss in quality of life because of recurrent aspiration pneumonia issues. Both aspiration pneumonia and dehydration are the common causes of death among these patients. The cause of the disease is related to a genetic mutation that is responsible for the establishment of a mutant unstable protein (PABPN1) that becomes jointed and aggregated within muscle cells.
Cabaletta is a chemical substance that has protective abilities against some pathological processes in cells. It prevents the pathological aggregation of proteins, a phenomenon that occurs inside cells, and has been identified in several studies to be the cause of a number of diseases.
Notes About BioBlast Pharma
BioBlast Pharma was founded in 2012 and is developing therapies for rare and ultra-rare genetic diseases. The Company retains a diverse and powerful portfolio of candidates with high potential to respond to such incurable genetic diseases.