A biotech company developing cell and gene therapies for blood cancers and hematological disorders is partnering with a European company to develop genome-editing technology for genetic diseases.
Cambridge, Massachusetts-based bluebird bio and Danish drugmaker Novo Nordisk said Wednesday they had started a three-year research collaboration focused on developing in vivo genome editing treatments for genetic diseases, including hemophilia, with a particular focus on developing a gene therapy for hemophilia A. The companies will use bluebird’s RNA-based megaTAL technology and Novo Nordisk’s hemophilia portfolio.
Although better known as a company that makes treatments for diabetes, Novo Nordisk also markets several treatments for hemophilia, including recombinant factors VIIa, VIII and XIII, among others. IT also has a monoclonal antibody, concizumab, in Phase II development for hemophilia A and B. megaTAL is related to TALENs, or transcription activator-like effector nucleases, a form of gene-editing technology distinct from the other two dominant technologies, CRISPR and zinc finger nucleases. Bluebird’s website describes megaTALs as fusion proteins that combine homing endonucleases with TALENs’ modular DNA binding domains in order to precisely target DNA sequences.
“Bluebird has made tremendous progress on enabling an in vivo gene editing platform based on our megaTAL technology, including important advances in high-quality mRNA production and purification,” bluebird Chief Scientific Officer Philip Gregory said in a statement, adding that the company considers the technology to have potential to create a “highly differentiated” approach to treating severe genetic diseases. “Moreover, we are thrilled to be able to combine this new platform technology with Novo Nordisk’s deep expertise in hemophilia research and therapeutics.”
Bluebird’s first approved product was the gene therapy Zynteglo, which the European Medicines Agency approved earlier this year for transfusion-dependent beta-thalassemia.
San Rafael, California-based BioMarin is developing BMN 270 (valoctocogene roxaparvovec), an adeno-associated viral vector-based therapy in Phase III development for hemophilia A. Another company, Philadelphia-based Spark Therapeutics, is also developing an AAV-based gene therapy for hemophilia A, SPK-8011, in addition to SPK-9001 (fidanacogene elaparvovec) for hemophilia B. In February, Swiss drugmaker Roche announced it would buy Spark for $4.3 billion, but the deal has lately hit regulatory speedbumps.
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.
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