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RNA Specialist Arcturus Therapeutics Gets $3M R&D Grant for CF Drug

Xconomy San Diego — 

San Diego-based Arcturus Therapeutics said today Cystic Fibrosis Foundation Therapeutics has agreed to provide $3 million to fund research and development of a new messenger RNA drug that could be broadly used to treat cystic fibrosis patients.

The four-year-old startup has previously raised $37 million in research grants and other “non-dilutive” funding, and another $13 million from investors, according to Arcturus spokeswoman Neda Safarzadeh.

Arcturus describes itself as “an RNA medicines company” with proprietary technology for developing so-called antisense drugs that are intended to prevent specific gene mutations from producing disease-causing proteins. Arcturus says its technology can be used to make all types of drugs that target RNA in this aberrant protein-making process, including messenger RNA, small interfering RNA, antisense RNA, microRNA, and gene editing therapeutics.

Cystic fibrosis is a genetic disorder caused by mutations in the gene that makes a key protein responsible for moving ions across the membranes of certain cells, which is important in mucus production. However, these abnormal proteins cause the body to produce mucus that’s thicker and stickier than normal. The buildup of this heavy mucus in the lungs and other organs results in a variety of health issues—especially difficulties breathing and lung infections.

There is no cure, but the field has been rapidly advancing, and Arcturus faces some strong competition.

The Vertex Pharmaceuticals (NASDAQ: VRTX) drug ivacaftor (Kalydeco) was approved to treat CF patients that have certain genetic mutations. Earlier this month, the FDA expanded the drug’s approval to cover 33 mutations.

Arcturus said its two-year agreement with the Cystic Fibrosis Foundation will enable the company to accelerate the development of its mRNA drug for treating more than 1,700 genetic variations associated with cystic fibrosis. Arcturus plans to apply its nanoparticle delivery technology to get its mRNA drug into a patient’s pulmonary system.