AbbVie and Voyager Therapeutics have extended their collaboration to co-develop and commercialize antibodies that target the toxic forms of alpha-synuclein to treat Parkinson’s disease and other synucleinopathies.
The collaboration will combine AbbVie’s expertise in monoclonal antibodies with Voyager’s gene therapy platform to deliver antibodies across the blood-brain barrier using a one-time injection into the blood. The blood-brain barrier is a semipermeable membrane that protects the brain against the external environment, and is a major barrier for the efficient delivery of certain therapeutics that need to reach the brain and central nervous system.
Researchers will use a modified and harmless adeno-associated virus (AAV) with a high capacity to penetrate the blood-brain barrier to deliver genes that carry the information for producing therapeutic antibodies against toxic forms of alpha-synuclein.
“The expansion of AbbVie’s partnership with Voyager represents the potential we see in the ability of its vectorized antibody platform to surpass the blood-brain barrier and more effectively deliver biologic therapies,” Jim Summers, PhD, vice president, discovery neuroscience research, AbbVie said in a press release. “We are hopeful that Voyager’s technology will enable further development of transformative treatments for patients with neurodegenerative diseases,” Summers said.
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Parkinson’s disease belongs to a class of neurodegenerative disorders called synucleinopathies, characterized by the accumulation of misfolded alpha-synuclein aggregates. These abnormal protein aggregates are toxic and trigger the death of dopamine producing-nerve cells — those responsible for releasing the neurotransmitter dopamine, a critical neurotransmitter that regulates brain cell activity and function.
Given the key role of alpha-synuclein in the development and progression of Parkinson’s disease, many efforts have been made to find ways to effectively prevent its toxicity, such as using antibodies targeting the toxic forms of alpha-synuclein.
However, delivering antibodies to the brain is not an easy task. This is because the central nervous system (CNS) — comprised by the brain and spinal cord — is protected from the circulatory blood system by the very selective blood-brain barrier.
Current delivery of antibodies to the brain requires frequent injections and large amounts of antibodies. This new approach is expected to result in higher therapeutic antibody levels in the brain.
“Our scientific platform allows us to develop unique AAV gene therapies that are designed to knock down disease-causing gene expression, increase the expression of missing proteins, or enable the expression of therapeutic antibodies through vectorization,” said Andre Turenne, president and CEO of Voyager Therapeutics.
“We are excited to expand our efforts towards pathological species of alpha-synuclein given its role in the progression of disease, and AbbVie is the ideal partner to advance this new target and therapeutic modality,” Turenne added.