Express In view of the common genetic risk of Parkinson's disease, Xinrui has reached a gene therapy cooperation

Neurocrine Biosciences and Voyager Therapeutics today announced a new strategic collaboration to advance multiple gene therapies
for the treatment of neurological disorders.
The collaboration includes a Voyager gene therapy program
for the treatment of Parkinson's disease and other GBA1 gene-mediated diseases.
The gene therapy is administered intravenously and is in the preclinical stage
.
It uses a new capsid from Voyager's proprietary TRACER platform loaded with a payload capable of replacing the GBA1 gene
.
In addition, the two companies will collaborate on three new gene therapy projects targeting rare central nervous system (CNS) targets, all utilizing Voyager's novel TRACER capsid.

Voyager's TRACER technology platform is a widely applicable RNA-based screening platform for the rapid and efficient discovery of adeno-associated virus (AAV) capsids that efficiently cross the blood-brain barrier (BBB) and have enhanced CNS tropism in a variety of species, including non-human primates
.
These capsids have higher specificity, require lower doses, and have less off-target risk
than traditional AAV serotypes in non-human primates.
The capsids produced by TRACER exhibit higher and broader gene expression
in the CNS, including brain regions that are typically difficult to reach, compared to conventional AAV capsids.
For example, when administered intravenously, a capsid produced by TRACER increases transgene expression by a factor of 1,000-10,000 in a wide range of brain regions compared to conventional AAV9
vectors.

GBA1 is a gene encoding the lysosomal enzyme glucocerebrosidase (GCase), a gene whose mutations have been linked to
a variety of diseases.
About 10% of Parkinson's disease patients carry this mutation, which is the most common genetic risk factor for Parkinson's disease and increases the risk by about 20 times
.
When this mutation is homozygous, a decrease in GCase activity can cause lysosomal diseases such as Gaucher disease
.
Gene replacement therapy using AAV capsids that can cross the BBB is expected to continue to correct such diseases
affecting the CNS.
In preclinical models of GBA loss of function, Voyager has demonstrated that using its CNS-targeted intravenous capsid is able to deliver a payload to replace the gene and generate treatment-relevant levels of the GCase enzyme
.

The collaboration builds on the two companies' long-term strategic partnership and will continue to combine Voyager's expertise in novel capsid discovery, payload design and neuropharmacology with Neurocrine's extensive experience in clinical and commercial development of neuroscience and therapies to provide new treatment options
for patients with severe neurological conditions.

Image source: 123RF

Under the terms of the agreement, Voyager will receive an upfront payment of $175 million, including a $39 million equity investment, in addition to the possibility of potential development milestones of up to $1.
5 billion, additional potential commercial milestones, tiered royalties on net sales, and project funding
.

Dr.
Jude Onyia, Chief Scientific Officer of Neurocrine Biosciences, said: "This new collaboration with Voyager covers GBA1-mediated diseases like Parkinson's disease and Gaucher disease, complementing our existing collaboration
around Friedreich ataxia and other CNS targets.
We believe GBA1 gene therapy holds the potential to play a transformative role
in the treatment of Parkinson's disease and other serious neurological disorders in the future.
”

Dr.
Alfred W.
Sandrock, CEO of Voyager, said, "This collaboration is a great opportunity for Voyager to demonstrate its value by combining our novel TRACER capsid platform with our in-depth understanding of neuropharmacology and payloads to advance next-generation gene therapies
for CNS diseases.
We look forward to expanding our collaboration
with Neurocrine Biosciences, which already has a strong relationship.
" We anticipate that the opportunities presented by this collaboration will allow us to continue to invest in our platform and pipeline and advance cutting-edge research initiatives
.
" ”

As WuXi AppTec's cell- and gene-focused CTDMO, WuXi Biologics accelerates and transforms the development, testing, manufacturing, and commercialization
of gene and cell therapies and other advanced therapies.
WuXi Biologics can help customers around the world bring more innovative therapies to market for the benefit of patients
.
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