Moonwalk debuts with $57M and epigenetic toolkit to reprogram disease

04 Jan 2024
firstwordpharma.com
Gene Therapy
Moonwalk Biosciences is moving beyond genetic scissors to focus on our genomic software. With $57 million in funding and a suite of epigenetic technologies, the company aims to develop therapies that reverse disease by flipping their genetic on/off switches – no cuts necessary.
Moonwalk boasts an impressive roster of co-founders, including serial biotech entrepreneur and renowned scientist Feng Zhang of the Broad Institute of MIT and Harvard, and Alex Aravanis, the former chief technology officer of Illumina. Moonwalk is at least the sixth company Zhang has helped found.
The $57-million funding announced Thursday comprises seed and Series A rounds led by Alpha Wave Ventures. Additional participating investors include ARCH Venture Partners, Future Ventures, GV, Khosla Ventures and YK Bioventures.
Mapping methylation
While traditional gene editing usually involves cutting, nicking, or removing DNA sections, epigenetics regulate whether a gene is “on” or “off” without physically changing the DNA itself. Moonwalk aims to modify gene expressions associated with disease by modulating the methylation patterns that determine cell states.
In an interview with FirstWord, Aravanis, Moonwalk’s CEO, saidmethylation acts as  a kind of switch that determines which genes are allowed to be expressed, and which ones aren’t. “This is the single most important thing which controls which of many genes in the genome hardware are going to be used in any particular cell,” he said.
While the theory that epigenetics could produce disease-modifying therapeutics has been around for several years, actual drug development in the field has been limited by a lack of comprehensive knowledge on the 28 million methylation sites in the genome that control expression. Much as the Human Genome Project in the early 2000s helped jump-start the genetic revolution, Aravanis said a methylome mapping study he helped co-author, which published last January in Nature, was key to uncovering the “epigenome recipes” that determine cell type.
“Before – for most cells in the human body and the vast majority of their methylation sites – we didn’t know their status. There wasn’t the information to understand what these signals look like and how significant they were,” Aravanis said. The methylome atlas project uncovered methylation patterns that control specific sets of genes that in turn define whether a cell becomes a T cell, or an epithelial cell, and so on.
Unlocking epigenetic treatments
Moonwalk is leveraging this knowledge of methylation patterns to first find epigenetic differences between healthy and diseased cells, and then create compounds that manipulate the methylation of a diseased cell back into a healthy state. The biotech calls this process its “read-and-write” platform.
For the “read” portion, Moonwalk is deploying AI and machine learning technologies to predict which of the 28 million methylation sites contribute to a cell’s healthy state, which ones are going to be the most causally related to disease, and which are the most optimal targets.
Moonwalk then plans to “write” a compound that fuses a proven homing mechanism – whether a Cas protein with a guide RNA, a TALE protein, or a zinc finger – to an enzyme to either methylate, demethylate, or modify the histones at the target site, depending on whether a gene needs to be turned on or off.
The approach, Aravanis says, offers two potential benefits over gene editing technologies used today. First, because the DNA isn’t being snipped, there’s less of a chance for genome toxicity, and thus a better safety profile. Second, while these editing methods are adept at removing genes, it’s much harder for them to activate genes. Modulating methylation is particularly suited to induce gene expression, he added.
With the Series A funding in hand, Aravanis said the company plans to spend the next couple of years exploring the disease areas in which epigenetic therapeutics can have the biggest impact.
“Because epigenetic changes are profound and they occur in every pathological process, there are lots of opportunities, particularly in complex, chronic conditions like cancer, heart disease, metabolic disease, and neurodegeneration,” Aravanis said. “There's the ability there to try to change those states back and reverse disease.”
For more details,please visit the original website
The content of the article does not represent any opinions of Synapse and its affiliated companies. If there is any copyright infringement or error, please contact us, and we will deal with it within 24 hours.
Organizations
Indications
Targets
Drugs
-
Get started for free today!
Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.
Start your data trial now!
Synapse data is also accessible to external entities via APIs or data packages. Leverages most recent intelligence information, enabling fullest potential.