Natural medicines

Aiming for on/off switch of genes, Chroma corrals $125M for new wave of medicines – MedCity News

Summary

 

Cells have the same DNA, but the body has a built-in way to effect what any given cell becomes, whether it’s a liver cell or a neuron. That system, called the epigenome, takes what is in the DNA and makes sense of it, explained Catherine Stehman-Breen, CEO of biotech startup Chroma Medicine. If you think of DNA as hardware, then the epigenome is the software that tells the hardware what to do, she said.

Genetic medicines research is marching forward with therapies…….

npressfetimg-1267.png

 

Cells have the same DNA, but the body has a built-in way to effect what any given cell becomes, whether it’s a liver cell or a neuron. That system, called the epigenome, takes what is in the DNA and makes sense of it, explained Catherine Stehman-Breen, CEO of biotech startup Chroma Medicine. If you think of DNA as hardware, then the epigenome is the software that tells the hardware what to do, she said.

Genetic medicines research is marching forward with therapies that treat disease by cutting and editing the genome, or by introducing genetic material intended to fix faulty genetic code. But Stehman-Breen said tapping into the epigenome offers an alternative approach, and one with potential advantages. Instead of trying to correct a mutated gene, Chroma is targeting the software that controls it in order to regulate its expression.

“Leveraging this mechanism allows us to have a tremendous amount of breadth in terms of therapeutic application,” Stehman-Breen said. “We think it’s ultimately a really powerful tool.”

Cambridge, Massachusetts-based Chroma is building a pipeline of epigenetic medicines that turn genes on or off and the startup has raised $125 million to support its research. The Series A round of funding announced Wednesday was led by Cormorant Asset Management.

Chroma’s approach mimics what the body already does, said Vic Myer, the company’s president and chief scientific officer. There are some genes, such as retroviruses in the genome, that remain dormant for life because the epigenome turns them off. Similarly, a Chroma drug would silence a toxic or mutant gene, allowing a healthy gene to do its job. For haploinsufficiencies, diseases in which one gene of a gene pair is not functioning properly, Chroma would take the opposite approach and turn on gene expression.

The drugs themselves are large molecules engineered to go to a specific location in the genome, Myer said. The goal is for these therapies to be one-time treatments. After the drug does its work, when the cell divides, the epigenomic system understands that a particular gene is supposed to be turned off. The daughter cells that follow retain the gene silencing. Myer, a former executive at gene-editing biotech Editas Medicine, said he left that company after nearly five years wanting to do something different. He was persuaded to return to genomic medicines by Chroma’s technology.

“It’s an elegant approach, and sort of a natural, normal approach to turn off a gene if you want to turn off a gene, and turn on a gene if you want to turn on a gene,” Myer said.

Chroma is based on the research from its six scientific founders: Luke Gilbert, Keith Joung, David Liu, Angelo Lombardo, Luigi Nadine, and Jonathan Weissman. According to Stehman-Breen, the basis of Chroma was research from Weissman, Gilbert, and others, published in the journal Cell in 2016, showing how a “CRISPRoff” fusion protein could turn off gene …….

Source: https://medcitynews.com/2021/11/aiming-for-on-off-switch-of-genes-chroma-corrals-125m-for-new-wave-of-medicines/