- Bayer has struck an alliance with U.S. biotech firm
Dimension Therapeutics to develop a gene therapy for the
treatment of haemophilia A, marking renewed interest in
an approach to tackle the cause of diseases at a
Gene therapy involves inserting corrective genes into malfunctioning
cells to get them to work again without further use of drugs.
Dimension has been looking into using viruses to carry the genes to
the affected cells.Gene therapy has seen more than 20 years of
experiments and a series of disappointments. However,
Amsterdam-based UniQure is about to start selling its gene therapy
drug Glybera in Europe as a treatment for the ultra-rare disease
lipoprotein lipase deficiency (LPLD) with partner Chiesi.
U.S. biotech firm Bluebird Bio last week reported positive results
with an experimental gene therapy for another blood disorder,
sending its shares soaring.
Dimension Therapeutics will receive an upfront payment of $20
million and may get development and commercialisation milestone
payments of up to $232 million. Bayer will also make royalty
payments based on future product sales.
Standard haemophilia A therapies are often administered
intravenously multiple times a week and may be required for life.
The German drugmaker's established haemophilia A therapy Kogenate
had 1.2 billion euros ($1.6 billion) in sales last year. It has two
more drug candidates against the type A of the hereditary bleeding
disorder in the third and last phase of testing on humans that is
required for regulatory approval. ($1 = 0.7366 Euros)