The development of approved biotherapeutic agents is generally long, costly, complex, and fraught with risk. And while the rewards are tempting, necessary funding to validate therapeutic product candidates and protein-based molecule through target validation, lead identification, and optimization, as well as secure intellectual property (IP) and patent protection, are required to increase the chances for success.

To advance the development of innovation therapoeutic agents and foster collaboration opportunities and innovation between scientists and entrepreneurs, MilliporeSigma, the Life Science business of Merck KGaA, Darmstadt, Germany whioch operates in the United States and Canada, has established grant programs in different regions of the world. This year Combined Therapeutics, a Cambridge, Massachusetts, based biotechnology company, was selected as the North American winner of MilliporeSigma’s 2020 Advance Biotech Grant Program.

The company was selected for its innovative nucleic acid platform which, combined with virotherapy*. This novel approach targets cancerous cells while protecting healthy tissue of key organs, has the potential to treat liver cancer by using a lipid nanoparticle delivery system.

Liver cancer
According to the American Cancer Society, in 2020, an estimated 42,810 adults (30,170 men and 12,640 women) in the United States will be diagnosed with primary liver cancer. Statiics have shown that the incidence of liver cancer has tripled, is the early 1980s. The same data shows that between 2007 and 2016, the number of people diagnosed with primary liver cancer has increased by approximately 2% annually. Men are about 3 times more likely than women to be diagnosed with the disease, making it the fifth most common cause of cancer death. For women, primary liver cancer is the seventh most common cause of cancer death among women.[1]

The overall, 5-year survival rate of hepatocellular carcinoma, the most common type of primary liver cancer, occurs as a results of chronic liver cirrhosis and has a five-year survival rate of only 18%, compared to just 3% 40 years ago.[1]

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Ethnicity is a major factor in the development of the disease, and Asians and Pacific Islanders have the highest risk of developing liver cancer, but the risk is also elevated for African-American, American Indian and Latino populations as well, compared to non-Hispanic whites.

Novel platform
When used in treating cancer, virotherpy includes viruses that have been genetically modified to selectively replicate within cancer cells and express specific transgenes that alter the tumor microenvironment to inhibit tumor progression. The products of these transgenes induce cell death, make the virus less virulent, compromise tumor vascularity, and are capable of modulating or enhancing the immune system-such as cytokines and chemokines.[2]

“We help pioneering biotech companies bring lifesaving drugs to market, faster,” noted Andrew Bulpin, head of Process Solutions at MilliporeSigma. “Combined Therapeutics’ platform shows promise to treat liver cancer, which affects thousands of people across the world each year.”

As part of the award, Combined Therapeutics will receive in-kind MilliporeSigma products and consultation in downstream processing, formulation excipients and sterile filtration.

Pre-clinical deveopment
Combined Therapeutics Inc. is in pre-clinical development of a platform to treat cancer by targeting solid tumors while protecting the healthy tissue of key organs. Virotherapy is treatment that uses a modified virus to target and destroy cancer cells. This approach, however, can often lead to low patient response and safety concerns due to poor targeting. Combined Therapeutics aims to direct and enhance virotherapies while preventing off-target effects. With its potential to target specific cells only, this combined approach shows promise to be a safe and effective therapy for cancer.

“MilliporeSigma’s expertise in large biomolecule filtration, scale-up and extensive range of excipients will play a pivotal role in moving this project forward to GMP and clinical trials,” said Romain Micol, Chief Executive Officert, Combined Therapeutics

“We are honored to have been selected as the winner of MilliporeSigma’s Advance Biotech Grant, and we look forward to the collaboration,” Micol added.

Since 2014, MilliporeSigma’s Advance Biotech Grant Program has awarded technologies and consultation to 28 biotechnology companies. These grants have been used to support biotech companies whose missions are to help patients suffering from cancer, brain tumors, osteoarthritis and cardiovascular disorders.

* Virotherapy is a treatment using biotechnology to convert viruses into therapeutic agents by reprogramming them to treat diseases.

[1] Key Statistics About Liver Cancer. American Cancer Society. Online. Last accessed on November 2, 2020
[2] Berkey SE, Thorne SH, Bartlett DL. Oncolytic Virotherapy and the Tumor Microenvironment. Adv Exp Med Biol. 2017;1036:157-172. doi: 10.1007/978-3-319-67577-0_11. PMID: 29275471.

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