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Pictured left to right at the Voyager Therapeutics ribbon cutting are Steven Paul, Susan Windham-Bannister, Mark Levin, Michael Collins, Terence Flotte and Guangping Gao.
Although Cambridge-based biotech startup Voyager Therapeutics has only been in existence for one year, its developments in gene therapy so far could place it at the forefront of medical innovation for years to come.
Gene therapy is a technique in which defective or toxic disease-causing genes are either replaced with normal ones or silenced—using RNA interference technology developed at University of Massachusetts Medical School (UMMS)—by purposely infecting patients with a harmless virus. By using gene therapy and advancing adeno-associated viruses (AAV) as a carrier to develop vital proteins in the brain, Voyager Therapeutics aims to develop life-changing treatments for central nervous system diseases such as Parkinson’s, ALS and Friedreich’s ataxia.
While it may be a few years before one of Voyager’s gene-therapy drugs is approved, the firm’s research shows such potential that venture capital firm Third Rock Ventures invested $45 million in Series A funding to launch the startup in February 2014.
The startup, born out of research conducted at the University of Massachusetts, is now well on it’s way to becoming a leader in gene therapy innovation.
With the proper financing, Voyager and Third Rock are fully committed to advancing AAV gene therapy by conceiving and investing in vector optimization, new dosing techniques, and process development and production.
Voyager’s commitment to excellence in gene therapy stems from its founders, who are experts in AAV gene therapy, RNA interference and neuroscience. Each member of the group — made up of Krystof Bankiewicz, Guangping Gao, Mark Kay and Phillip Zamore — is as a leading researcher responsible for pioneering a number of medical advancements.
Gao and Zamore have strong ties to one of the world’s leading research institutes, as Gao is the director of UMMS Gene Therapy Center & Vector Core and a Penelope Booth Rockwell chair in Biomedical Research, and Zamore is a Howard Hughes Investigator, the Gretchen Stone Cook Chair of Biomedical Sciences at UMMS as well as a professor of biochemistry and molecular pharmacology and co-director of the RNA Therapeutics institute at UMMS.
The University of Massachusetts has been critical in furthering Voyager’s developments, as Voyager and MassBiologics of UMMS entered into a strategic collaboration in December 2014 to establish scalable processes for manufacturing Voyager’s recombinant AAV vector products at MassBiologics’ South Coast Manufacturing Center in Fall River.
With the proper financing, Voyager and Third Rock are fully committed to advancing AAV gene therapy by conceiving and investing in vector optimization, new dosing techniques, and process development and production.
The collaboration may seem curious considering Voyager is still a startup, but advancements in Voyager’s gene therapy treatment for Parkinson’s and the need for a new drug outlines why the partnership is necessary.
The key to the underlying biology of Parkinson’s is the progressive loss of neurons in the brain which produce dopamine. Neurons rely on the enzyme aromatic L-amino acid decarboxylase (AADC) to convert levodopa into dopamine. When neurons drop, so do AADC levels, and levodopa cannot produce dopamine at the required level, causing tremors, rigidity and slowness of movement.
Patients can take levodopa in a pill form, but as Parkinson’s progresses they require higher and more frequent doses to maintain stability, and side effects such as dyskinesia set in.
Voyager’s gene therapy treatment for Parkinson’s is aimed at replacing AADC enzymes and lowering the required daily dose of levodopa patients need. This would provide more reliable control of symptoms and improve patients’ overall quality of life.
Due to the promise of Voyager’s Parkinson’s therapy, the company must be able to produce large quantities of the drug for clinical trials. Access to MassBiologics’ manufacturing expertise and infrastructure allows Voyager to deliver large-scale production.
Voyager’s collaboration with MassBiologics also furthers the startup’s commitment to utilizing the best minds and resources, as MassBiologics is the only publicly owned, nonprofit FDA-licensed manufacturer of vaccines and other biologic products in the nation.
In just under a year of existence, Voyager Therapeutics has positioned itself as one of the leading biotech firms in the nation, garnering Voyager recognition as one of the “Fierce15” Biotech Companies of 2014 by FierceBiotech.
It may be a few years before we see one of Voyager’s treatments approved, but its name will certainly be heard often as the firm continues to drive the advancement of gene therapy.
Image via the University of Massachusetts
