CAMBRIDGE, Mass. – Hunterian Medicine LLC, a gene-editing and gene therapy company, has been awarded a Therapeutics Development Award by the Cystic Fibrosis Foundation. The award, through the Foundation’s Path to a Cure initiative that seeks to replace, restore or repair cystic fibrosis transmembrane conductance regulator (CFTR) genes, will support Hunterian’s concentrated efforts in advancing its innovative platform technology to develop a gene therapy for cystic fibrosis.
“We are especially grateful to receive the Cystic Fibrosis Foundation’s support for our cystic fibrosis gene therapy program,” said Dr. Vinny Jaskula-Ranga, President and CEO of Hunterian Medicine. “Working with the Foundation not only provides important financial support in our quest to find a cure for the condition but also connects us to leading experts in the field. The vision behind the Path to a Cure initiative is just another example of what sets the Cystic Fibrosis Foundation apart.”
As part of the award, Hunterian will collaborate with cystic fibrosis experts from the Johns Hopkins University School of Medicine and the Johns Hopkins Cystic Fibrosis Center. The co-investigators from Johns Hopkins are Drs. Garry Cutting, Christian Merlo, and Neeraj Sharma.
Hunterian’s platform technology addresses major therapeutic hurdles for replacing genes (gene therapy) and repairing genes (CRISPR gene-editing). The technology enables large genes to fit inside adeno-associated virus (AAV) vectors, the gold standard for gene delivery. In the case of cystic fibrosis, Hunterian’s technology will enable the packaging of the full-length CFTR gene, or alternatively a CRISPR gene-editing system, within a single AAV vector. While the company is pursuing both approaches for the condition, Hunterian’s Therapeutics Development Award is initially focused on the gene therapy approach using the full-length CFTR gene.
About Cystic Fibrosis
More than 30,000 people in the U.S. have cystic fibrosis, a debilitating and ultimately fatal disease caused by mutations in the CFTR-encoded chloride channel. More than 1,800 distinct CFTR mutations have been identified. Despite remarkable therapeutic advances which have increased life expectancy, many patients, in particular those carrying nonsense or splicing mutations, do not respond to current medications. Treatments benefiting all cystic fibrosis patients, especially those who do not respond to current therapies, are urgently needed.
About Hunterian Medicine LLC
Hunterian Medicine, headquartered in Cambridge, Massachusetts with a laboratory in the LifeBridge Health BioIncubator in Baltimore, Maryland, is a pre-clinical gene-editing and gene therapy company working to cure genetic diseases using its innovative delivery technology. The company’s platform technology provides a solution to the AAV gene delivery problem for key areas of (1) CRISPR gene-editing and (2) gene therapy applications. The company is developing a pipeline of gene-editing and gene therapy therapeutics to address diseases of significant unmet need.
Researchers across the world continue to demonstrate the revolutionary potential of gene-editing and gene therapy technologies to cure vast numbers of genetic diseases affecting millions of people; however, broad therapeutic development has been hindered by one universally recognized challenge: Delivery.
Although the industry standard for in vivo gene delivery is through an AAV vector, the primary barrier to delivery of CRISPR and other large genes is AAV’s limited packaging capacity. Hunterian’s innovative technology is based on the discovery of novel genetic elements – compact promoters – that significantly reduce the length of the sequence required for in vivo expression, enabling delivery via a single AAV for key areas of CRISPR gene-editing and gene therapy applications.
For CRISPR gene-editing, Hunterian’s technology enables delivery of SpCas9, high-fidelity variants with undetectable off-targets, Cas12a (Cpf1), PAM variants, and other CRISPR systems through a single AAV. This makes possible the targeting of more regions in the human genome – several billion more sites than existing technologies – meaning that many more diseases can now be addressed.
For gene therapy applications, Hunterian’s technology enables delivery of genes previously deemed too large to fit into AAV, such as CFTR. Competing approaches using truncated and less functional genes, or synthetic viral promoters and enhancers with known safety issues, can be avoided entirely.
The company’s extensive portfolio of novel endogenous promoters enables robust expression following the packaging of large genes through a single AAV vector. Hunterian’s novel promoters provide the unique ability to fine-tune expression levels for the safest and most effective gene therapy and gene-editing applications.
For more information, please visit www.hunterian.com.
Mark Button (for Hunterian Medicine LLC)