Hansa Biopharma has granted an exclusive license to Sarepta Therapeutics for developing and promoting a pretreatment aimed to make a gene therapy to treat muscular dystrophy available to people that are naturally resistant to the treatment.

Gene therapy can be used to treat genetic conditions by delivering a healthy copy of the faulty gene using harmless recombinant viruses. The adeno-associated virus (AAV) is one of the most commonly used in gene therapy, and the basis of Sarepta Therapeutics gene therapy technology.

However, up to 70% of people naturally carry neutralizing antibodies against AAV. This prevents the transfer of the healthy gene sequence and raises safety concerns for the patient. Swedish company Hansa Biopharma is developing a pretreatment called imlifidase that works to eliminate these neutralizing antibodies prior to gene therapy treatments.

Imlifidase is an enzyme from the bacterium Streptococcus pyogenes that breaks down the antibodies that are involved in generating an immune response against external pathogens, inhibiting their activity within hours after administration.

Imlifidase is completely unique in development, said Emanuel Bjorne, VP Business Development at Hansa Biopharma. There is no product in the market that has this mode of action.

Sarepta Therapeutics aims to use imlifidase to extend its gene therapies for Duchenne muscular dystrophy and Limb-girdle muscular dystrophy to those patients that are naturally resistant to the AAV vectors used to deliver these treatments. According to the agreement, Hansa will get 8.8M ($10M) upfront and is eligible for a total of 350M ($397M) in development, regulatory, and sales milestone payments, in addition to royalties to future sales.

Sarepta Therapeutics will conduct preclinical evaluation of imlifidase as a pretreatment for gene therapy with Hansa providing our imlifidase expertise to the collaboration, said Bjorne. If everything goes according to plan, the company will start a clinical study of imlifidase in combination with Sareptas gene therapy in the second half of next year.

In preclinical models, Hansas technology has been successful in clearing the antibodies that prevent the success of gene therapies. If successful, this could offer the potential of extending existing gene therapy treatments to patients who would otherwise not be able to benefit from them. Hansa Biopharma is also investigating the use of this drug as a way to prevent the rejection of transplanted organs, and to treat cancer and rare autoimmune conditions.

Antara Mazumdar is a computational biologist based in Groningen, The Netherlands. She is also a freelance science writer who writes about various areas of biological research. Prior to that, she studied biomedical science and bioinformatics in New Delhi, India. Outside of work, she enjoys organizing scientific and cultural events, singing and is a traveling enthusiast.

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Hansa Biopharma Gets up to 350M to Make Gene Therapy Work in Resistant... - Labiotech.eu