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12:00 AM
 | 
Oct 29, 2012
 |  BioCentury  |  Emerging Company Profile

Regenesance: Stopping the MAC attack

Regenesance developing anti-C6 mAbs for myasthenia gravis

Regenesance B.V. is developing mAbs against complement 6 that may halt nerve damage and provide a better safety profile than standard of care for the orphan neurological disease myasthenia gravis. The mAbs are in lead optimization and are expected to enter the clinic in 18 months.

Myasthenia gravis is an autoimmune disorder characterized by a loss of signaling between nerve and muscle cells that leads to muscle weakness. The disease can be life threatening, as muscle weakness in the diaphragm can lead to breathing difficulty and the need for respiratory support.

Available treatments provide only symptomatic relief, while immunosuppressants have serious side effects.

It was long believed that autoantibodies against acetylcholine receptors (AChRs) at the neuromuscular junction were responsible for blocking signal transmission, causing muscle weakness and damaging the receptors. But according to co-founder and CSO Frank Baas, that view has changed.

Rather than inhibiting neuromuscular signaling, Baas said the autoantibodies recruit effectors that damage the neuromuscular junction. The primary effector is thought to be the C5b-9 membrane attack complex (MAC), as dense deposits of MAC are found in the neuromuscular junctions of patients.

MAC - a terminal...

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