Genzyme, a Sanofi company, has agreed to use Ablynx's therapeutic proteins or “Nanobodies” to carry out in vitro and in vivo research on multiple sclerosis models, then develop therapies against the disease, under a collaboration whose value was not disclosed.

Genzyme will pay Ablynx an “exclusivity fee” for the Nanobodies, which are based on single-domain antibody fragments, and combine the features of conventional antibody drugs and small-molecule drugs.

The Nanobodies will be studied, Genzyme said, against an undisclosed “target that plays an important role in multiple sclerosis (MS) and specifically aligns with Genzyme's early-stage MS research programs involving neuroprotection and CNS repair.”

MS is one of Genzyme’s key therapeutic areas of interest, along with rare disease. Two monoclonal antibodies against MS are in the company’s pipeline. One is the preclinical compound GZ402668, subject of a Phase I first-in-human, single ascending dose study and a long-term follow-up study.

The other is Vatelizumab, indicated for patients with relapsing remitting multiple sclerosis (RRMS), and subject of the Phase II EMPIRE trial for which Genzyme enrolled its first patients in November. As of April 22, Genzyme was enrolling patients by invitation only, according to ClinicalTrials.gov.

Genzyme says its current research efforts on MS “aim to develop agents that could provide neuroprotection against immunological damage and promote remyelination of neurons (neural repair).

“Such approaches may have applicability to other inflammatory neurological disorders such as neuromyelitis optica (NMO) or chronic inflammatory demyelinating polyneuropathy (CIDP),” Genzyme adds.

Ablynx has already generated Nanobodies against the specific target of interest, and confirmed their activity in pre-clinical models. The neurodegeneration observed in MS is not directly targeted by existing treatments, Genzyme said.

Upon completion of studies, Genzyme will have the option to negotiate a license agreement. Genzyme said it seeks to target underlying causes of MS disease progression, as well as develop treatments to protect neurons and promote repair of CNS damage.

“The formatting flexibility of Nanobodies makes them promising candidates for combining antibody-like selectivity and multi-specificity in a single molecule. We are looking forward to working for the first time with Genzyme,” Ablynx CEO Edwin Moses, Ph.D., said in a statement.

Genzyme joins pharma giants such as AbbVie, Boehringer Ingelheim, Eddingpharm, Merck & Co, Merck Serono, and Novartis in partnering with Ablynx. The Belgian-based biotech says it has more than 30 stand-alone and partnered R&D programs under development in therapeutic areas that include inflammation, haematology, oncology, and respiratory disease.








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