Researchers consider new tools for gene editing at Newton conference

Brian Schneider, Sr. Regional Account Manager at Synthego, said we should optimize gene-editing and engineer biology at the fifth International Conference of Drug Discovery and Development at Crowne Plaza Boston on Friday, Nov. 22, 2019. Photo by Anna Stjernquist/ BU News Service

By Anna Stjernquist
BU News Service 

BOSTON — “We need to think of biology at a bigger scale — in comes automation,” Brian Schneider, senior territory account manager at Synthego said last week as he pitched the idea of making biology a data science with gene editing tools at the Fifth International Conference on Drug Discovery, Development and Lead Optimization.

With topics ranging from cutting-edge research on drug development for cannabis to HIV treatment and efficient gene editing, researchers and practitioners gathered from all over the world for a three-day conference in Newton to network and spark conversation.

Synthego, like other companies and startups on the forefront of biotech, uses CRISPR-Cas9, a gene editing tool that has been making waves in the science community since it was co-developed at the University of California Berkeley in 2012.

The development created controversy though, after a legal battle over whether the Massachusetts Institute of Technology was first to patent the groundbreaking tool.

Not all rumors have been good: In November 2018, Chinese scientist He Jiankui revealed his plans to gene edit babies, spreading ethical concerns about what gene editing could do when cells are passed down to future generations.

The potential of CRISPR-Cas9 is, nevertheless, indisputable. The bio-engineering tool allows genetic editing on DNA and even could be used to reverse life-threatening disease, including HIV and cancer, according to Synthego reports.

The conference convened at a time when technology in drug development is innovating fast.

“The year that has passed since the [Conference Chairman Harold Smith] and I decided that we wanted a therapeutics symposium here has been a year where everything really went off in the field. So I think it’s even more appropriate to have a session like this, compared to what it was just a year ago,” said Troels Koch, co-chairman of the conference.

Since 2019, several studies in the U.S., Europe and Canada have begun testing CRISPR-Cas9 on humans for the first time, NPR reported.

Researchers from Penn Medicine in Pennsylvania announced they would test CRISPR-Cas9 on cancer patients as early as this year. The most recent update was released on Nov. 6 and suggests it may be possible to re-engineer patients’ immune cells to fight cancer.

Similarly, Vertex Pharmaceuticals released positive results Tuesday from the first company-backed study that tested CRISPR-Cas9’s gene-edited treatment on two patients.

The patients, who had severe blood disorders were reported as having benefited from the temporary therapy with only minor side effects.

Representing the biotech engineering company Synthego, Schneider said making biology a data science is key in making gene-editing efficient.

“Our ability to study biology is very limited,” he said. “The next level is to create a screening platform where researchers design or experiment on their computers, send it to us and then we return insight data.”

Companies like Synthego aim to optimize gene editing by collecting data in a “biology insight factory,” that gives its users quantitative feedback immediately. 

“We collect a ton of data with thousands of experiments in parallel, trying to make biology a true data science that can shorten the drug discovery timeline,” Schneider said.

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