A newly formed gene therapy startup is taking gene delivery technology from ReGenX and a $5 million investment from Fidelity Biosciences to develop new treatments for rare diseases, starting with hemophilia.
ReGenX and Fidelity Biosciences announced the launch of Dimension Therapeutics at the end of last month and disclosed the $5 million Series A fundraise in an SEC filing this week.
Dimension Therapeutics, for the time being led by Dr. Thomas Beck of Fidelity, is developing drugs based on ReGenX’s novel adeno-associated viral vectors, which are designed to enter cells and deliver their DNA genome to the nucleus. Beck said in the company announcement that safely and effectively delivering replacement genes to the correct target cells has been a core challenge for gene therapies in the past.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
Currently Dimension is working on IND-enabling studies of a drug candidate for hemophilia B, a hereditary bleeding disorder characterized by a lack of one of the proteins needed to form blood clots in the body. It can cause a person to experience life-threatening bleeding after an injury. Whereas current treatments for severe hemophilia focus on routine factor replacement therapy, Dimension hopes that its technology could correct the faulty gene that causes the disorder in the first place.
Dimension is the latest company to emerge in the revival of gene therapies. Last month, Children’s Hospital of Philadelphia spun out Spark Therapeutics to commercialize a gene therapy for inherited blindness. In June, Audentes Therapeutics launched with $30 million in VC funding to advance treatments for rare muscle diseases.
Meanwhile, Baxter is also in early development of a gene therapy for hemophilia B.
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