CELLECTA, INC. Receives Phase II NIH SBIR Grant to Screen for Therapeutic Targets for Blood Cancers

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Cellecta receives a two-year $1.85M Phase II NIH/NCI SBIR grant to identify novel drug targets which interact with small molecules specifically lethal to hematopoietic cells. It is anticipated that this study will provide a basis to develop therapeutic treatments for blood cancers.

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Discovery of pharmacological approaches to eliminate targeted cell populations related to specific tissues or lineages will open up a range of treatment options for life-threatening tissue-specific diseases such as leukemia.

Cellecta, Inc., a biotechnology contract services provider that specializes in genetic screening to identify novel therapeutic targets, announced receipt of a two-year $1.85M Phase II NIH/NCI Small Business Innovation Research (SBIR) grant to identify novel drug targets which interact with small molecules specifically lethal to hematopoietic cells. It is anticipated that this study will provide a basis to develop therapeutic treatments for blood cancers.

The grant, funded by the National Cancer Institute (NCI) at the National Institutes of Health (NIH), provides resources to enable Cellecta to apply its RNA interference (RNAi) screening platform to identify genes essential for the growth and proliferation of hematopoietic cells--genes that might be good therapeutic targets. This study is unique, however, because, in addition to the genetic screens, Cellecta will collaborate with Tartis, Inc. in Buffalo, NY and Dr. Andrei Gudkov’s lab at the Roswell Park Cancer Institute, also in Buffalo, to identify chemical compounds lethal to the same set of hematopoietic cells. Combined analysis of the chemical screening results with the genetic data will enable prediction and evaluation of both the biological mechanism of action of the small molecules and the effect of various genes on the cellular response to the molecules. It is anticipated that these findings will provide a basis to develop therapies focused on blood cell populations, from non-invasive myeloablative approaches to finely targeted treatments to eliminate specific oncogenic hematopoietic variants.

"Discovery of pharmacological approaches to eliminate targeted cell populations related to specific tissues or lineages will open up a range of treatment options for life-threatening tissue-specific diseases such as leukemia," stated Dr. Gudkov. "We have clinical approaches to replace lost cells for essential tissues--such as blood cells--but do not have cell-specific or tissue-specific chemotherapeutic approaches to eliminate diseased cells. This grant will allow us to continue our initial research developing targeted pharmacological approaches to eliminate diseased cell populations."

"We are very excited to begin this work which I believe is the first to combine loss-of-function RNAi and small molecule compound screens to evaluate the interaction between genetic and chemical responses in a disease model," said Cellecta President & CEO Dr. Alex Chenchik. "In addition to the potential impact on the development of blood cancer therapies, I anticipate that the results will validate this new experimental approach for the discovery and characterization of novel anti-cancer drugs. The preliminary results from the first year of the grant allowed us to reveal several classes of small molecules with unique mechanisms of action. We are very much looking forward to analyzing the full dataset from all the screens."

About Cellecta, Inc.

Cellecta is a privately owned contract research company that provides high-throughput (HT) RNAi-based genetic screening services for the discovery and functional characterization of novel therapeutic targets. Using their unique platform of lentiviral, shRNA, HT sequencing, and bioinformatics technologies, they offer shRNA library screening and analysis by HT sequencing, pooled lentiviral shRNA libraries, and stable reporter, overexpression, or knockdown constructs and cell lines. Cellecta’s scientists have developed some of the most advanced technologies available, developed in collaboration with opinion leaders and with funding from 12 NIH SBIR grants. For more information, visit the Cellecta website, call 1-877-938-3910, or email us at info(at)cellecta(dot)com.

About Tartis, Inc.

Tartis is a privately owned biotechnology company engaged in the R&D of novel therapeutics for treatment of hematological malignancies in children and adults, and safe preconditioning of recipients of hematopoietic stem cell transplantation. Tartis' strength is based on a close interaction of innovative biologists and chemists who have had prior experience in drug discovery and development. The team works in close collaboration with Dr. Andrei Gudkov and other scientists from the Roswell Park Cancer Institute in Buffalo, NY. Using high-throughput screens of small molecules, Tartis' scientists have identified several structural classes of compounds that are selectively toxic to cells of various hematological malignancies but nontoxic to other tissues in the body. These compounds are currently undergoing hit-to-lead optimization and characterization of their physicochemical and pharmacological properties.

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Paul Diehl
Cellecta, Inc.
(650) 938-4050
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