Carmine Therapeutics has completed series A financing to support the development of its innovative, non-viral gene therapy for cystic fibrosis (CF) and other diseases.

The company is working to advance a novel type of disease-modifying gene therapy, which is accomplished using its proprietary Red Cell EV Gene Therapy or REGENT platform. This technology allows the delivery of therapeutic payloads of pieces of genetic information both DNA and RNA to several tissues, including in the central nervous system (involving the brain and spinal cord).

It also enables the transport of shorter and larger segments of genetic information, as well as multiple payloads simultaneously, without inducing an immune or inflammatory response that often render gene therapies less effective, according to Carmine.

REGENTs ability to deliver very large genetic medicines to a broad variety of tissues, without triggering an immune response or excessive inflammation has the potential to extend the promise of gene therapy to numerous human diseases that have been inaccessible to viral based approaches, Don Haut, PhD, Carmines CEO, said in a press release.

CF is causedby mutations in the geneCFTR, which result in an abnormally thick mucus in the bodys organs, particularly in the lungs, pancreas, liver, and intestine.

Gene therapy involves correcting a genetic defect by introducing a normal copy of the affected gene into the patients cells or by silencing a faulty gene. Current gene therapies commonly involve the use of adeno-associated viruses, which are modified in the lab not to cause disease, as delivery vehicles. However, existing approaches pose manufacturing challenges, limit the size of the therapeutic gene, and may induce an immune response.

The REGENT platform uses red-blood-cell-derived extracellular vesicles (RBCEVs), tiny sacs surrounded by a fat layer that can be used to carry genetic material. Since red blood cells naturally lack genetic material, RBCEVs do not represent a risk of triggering an immune response and therefore enable re-dosing, according to Carmine.

Also, large amounts of RBCEVs can be obtained from a single unit of blood and are highly amenable for manipulation. The REGENT platform was based on research led by the companys co-foundersMinh Le, PhD, and Jiahai Shi, PhD, at theNational University of Singapore, andHarvey Lodish, PhD, at the Massachusetts Institute of Technology.

The Cystic Fibrosis Foundation and Huagai Capital are two new investors in this financing round. They join existing investors EVX Ventures and Simcere Pharmaceutical.

We are delighted to bring a terrific group of new investors who share our vision of the next generation of gene therapies into Carmine, said Haut.

The funding will first be used to advance the clinical development of the gene therapy for retinal (eye) and pulmonary diseases.

We are thrilled to join [the] Carmine family, said Deng Liang, of Huagai Capital. As an early-stage investor in biotech industry, we value Carmines RBCEV platform as a novel and advantageous delivery system in gene therapies, especially considering its payload capacity, costs, and safety. Combined with Carmines patented payload engineering methods, we believe Carmine would bring patients with promising curative therapies.

Added XQ Lin, Carmines chairman and founder: We are pleased to welcome Huagai Capital and the Cystic Fibrosis Foundation as new investors into Carmine and thank our existing shareholders for their continued support.

Non-viral gene therapy is a promising new modality with the potential to address many unmet medical needs, he said.

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Carmine Gets Series A Funding to Develop Non-viral Gene Therapy |... - Cystic Fibrosis News Today