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A CNS collaboration
May 2017
by Kelsey Kaustinen  |  Email the author
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GRANADA, Spain—nLife Therapeutics S.L and WAVE Life Sciences Ltd. have linked up in a research collaboration to investigate cell-specific targeting of nucleic-acid therapeutics in the central nervous system (CNS). The collaboration will run the next two years, with the companies working to determine if certain chemical moieties are capable of directing the distribution and uptake of nucleic acid cargo to different cell types such as neurons, glia and astrocytes.
 
Though specific financial details were not disclosed, WAVE will be funding the related research activities at nLife and will have an option to license the latter’s technology for development and commercialization rights across the WAVE portfolio.
 
“Following promising broad distribution of our existing CNS programs, and as we expand our portfolio into additional neurological disorders, we are exploring the ability of various technologies to selectively target certain cell types. This collaboration reflects WAVE’s long-term commitment to neurology and we expect will complement our oligonucleotide expertise within the CNS space,” said Dr. Paul Bolno, president and CEO of WAVE. “We are excited to initiate this research collaboration with nLife, a company that we believe is at the forefront of neuronal targeting technologies.”
 
Oligonucleotides are short nucleic acid polymers engineered to bind to specific complementary messenger RNA strands promoting their degradation. This in turn causes a decrease in the levels of specific proteins that may lead to diseases when they accumulate. By attaching small-molecule chemical ligands to oligonucleotides, it is possible to enable cell-specific targeting. In addition, conjugated oligonucleotides can be linked to un-druggable target proteins.
 
Andrés Montefeltro, senior vice president, Research, and founder at nLife, notes:Crossing the BBB [blood-brain barrier] is a big issue that has attempted to be addressed by nanoparticle formulations and antibody conjugates. However, using these technologies, less than 4 percent of the active molecule can cross the BBB with success, and a high systemic exposure remains unsolved.”
 
In an effort to get around this, “nLife’s approach is to selectively target disease affected cell populations rather than massive transfection along the brain. We believe that cell-specific delivery can increase the therapeutic margin, reduce the treatment dose and minimize the toxicity and side effects,” he explains.
 
That’s where the partnership with WAVE comes in.
 
“nLife has developed a unique delivery platform for small oligonucleotides to target specific neuronal populations. We use small-molecule drugs that interact with selected receptors or transporters on the cell membrane of the target cell. This combination of a small-molecule drug linked to an oligonucleotide facilitates intracellular delivery into targeted neuronal populations of interest for the treatment of specific neurodegenerative diseases,” explains Montefeltro. “Wave has a leading edge and strong proprietary position in chemistry for stereopure oligonucleotides for gene silencing. They can also target single point mutations in a mRNA with unprecedented results.
 
“There is good synergy in combining our targeted neuronal delivery approach with Wave’s stereopure oligonucleotide chemistry for development of gene-silencing therapeutics for the treatment of CNS disorders.”
 
“By combining WAVE’s stereopure molecules with our targeted delivery technology, we believe it will be possible to address genetic diseases that were previously not accessible and potentially expand development of safe and effective treatments for a host of CNS disorders,” said Errol De Souza, executive chairman of nLife.
 
In other news for WAVE, the company announced a share offering in mid-April of 4.2 million shares at $24 each. Approximately $93.4 million is expected in net proceeds, and there will be a 30-day option for underwriters to buy up to an additional 625,000 shares, which could bring in an additional $14.1 million. The company’s stock dropped roughly 12 percent following the news.
 
Maxx Chatsko wrote on The Motley Fool that this offering “will pad a strong end-of-2016 cash position of $150 million and greatly aid the effort to advance the first drug candidates from the company’s novel technology platform into clinical trials … the company is ramping up development of nucleic acid therapeutics targeting a wide range of rare genetic diseases. Despite their promise, nucleic acid therapeutics have struggled to advance in the clinic due to complex synthesis methods—something Wave Life Sciences claims to have solved with its novel chemistry platform that focuses on rational design. Proof-of-concept studies have demonstrated the approach results in more stable, active and specific therapeutic candidates than previous synthesis methods.”
 
Code: E051710

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