The gene editing system known as CRISPR-Cas9 has electrified the world of science and led to a slew of startups, including two—Editas Medicine and Intellia Therapeutics—that have already gone public. The latest to join the fray is Agenovir, a company incubated at Johnson & Johnson’s JLABS accelerator in South San Francisco that aims to use CRISPR technology to develop antiviral drugs.
Agenovir announced today that it’s raised $10.6 million in a round led by Data Collective. Summit, NJ-based cancer drugmaker Celgene (NASDAQ: CELG) also participated in the round, marking its second investment in a CRISPR company, following its 2015 backing of CRISPR Therapeutics. Lightspeed Venture Partners and other unnamed individuals and investors also provided the cash, the company wrote in a press release. Agenovir says it plans to use CRISPR-Cas9 and other methods to try to disrupt viral DNA, hoping to treat and eliminate diseases caused by latent or persistent viral infections that currently have no treatment. It didn’t specify these diseases, or say how far away its experimental treatments are from clinical testing.
The company’s founding CEO, Bruce Hironaka, said in the statement that the company plans to use the cash to hire a leadership team and push its research forward. Hironaka is on the company’s board with Data Collective co-founder Matt Ocko and William Smith, the general counsel for Fluidigm (NASDAQ: FLDM).
Agenovir is based on the work of scientific founder Stephen Quake, a Stanford University bioengineering professor. Quake used Agenovir’s approach to treat cells infected with Epstein–Barr virus, which can lead to a type of cancer called Burkitt lymphoma (as well as mono), in a study published in PNAS.
Recent research has shown that tiny fragments of DNA circulating in a person’s blood can allow scientists to monitor cancer growth and even get a sneak peek into a developing fetus’ gene sequences. But isolating and sequencing these bits of genetic material renders little insight into how that DNA is used to generate the dizzying array of cells, tissues and biological processes that define our bodies and our lives.
Now researchers at Stanford University have moved beyond relying on the static information delivered by DNA sequences in the blood. Instead, they’ve generated a much more dynamic picture by monitoring changing levels of another genetic material — RNA — in the blood. It’s the biological difference between a still photo and a video when it comes to figuring out what the body is doing, and why.
“We think of this technique as a kind of ‘molecular stethoscope,’” said Stephen Quake, professor of bioengineering and of applied physics, “and it’s broadly useful for any tissue you care to analyze. There are many potential practical applications for this work. We could potentially use it to look for things going wrong in pregnancy, like pre-eclampsia or signs of preterm birth. And we hope to use it to track general health issues in various organs.”
Neuroscientists and bioengineers at Stanford are working together to solve a mystery: How does nature construct the different types of synapses that connect neurons – the brain cells that monitor nerve impulses, control muscles and form thoughts.
In a paper published in the Proceedings of the National Academy of Sciences, Thomas C. Südhof, M.D., a professor of molecular and cellular physiology, and Stephen R. Quake, a professor of bioengineering, describe the diversity of the neurexin family of proteins.
Neurexins help to create the synapses that connect neurons. Think of synapses as switchboards or control panels that connect specific neurons when these brain cells must work together to perform a given task.
Neurexins play a key role in the formation and functioning of synaptic connections. Past human genetics studies have linked neurexins to a variety of cognitive disorders, such as autism and schizophrenia.
Südhof, the Avram Goldstein Professor in the School of Medicine and a winner of the 2013 Nobel Prize in Medicine, has spent years studying the many different forms, or isoforms, of neurexin proteins. He has postulated that different isoforms of neurexins may help to create different types of synaptic connections with distinct properties and functions, and thus enable neurons to do so many complex tasks.
But Südhof had no way to know exactly how many isoforms of neurexins existed until he sat down last year with Quake, the Lee Otterson Professor in the School of Engineering. Quake has pioneered new ways to sequence DNA – the master blueprint that nature follows when making proteins.
Neurexin gene-splicing graphicThe study being published in PNAS represents the results of a year-long collaboration between neuroscientists and bioengineers to better understand how different neurexin proteins affect the behavior of synapses and, ultimately, normal brain functions and neurological conditions such as autism.