CF Foundation Affiliate Awards $3M to Advance Gene Therapy Candidate to Trials

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by Magdalena Kegel |

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4D Molecular Therapeutics (4DMT) announced it has secured funding to complete studies necessary for its gene therapy candidate for cystic fibrosis (CF), 4D-710, to be tested in people.

The therapy is composed of a what 4DMT describes as an advanced and customized adeno-associated virus (AAV) vector, which will carry a functional CFTR gene into the lungs of CF patients. The idea is to deliver the therapy through a nasal spray.

Cystic Fibrosis Foundation Therapeutics contributed with $3 million in additional support, bringing the non-profit’s total investment to more than $3.5 million. The organization is the drug discovery and development arm of the Cystic Fibrosis Foundation.

The financing will cover studies needed to file an Investigational New Drug (IND) application — required to test a new therapy in humans — with the U.S. Food and Drug Administration.

“We are very pleased and honored to have been selected to receive additional IND candidate development funding from Cystic Fibrosis Foundation Therapeutics Inc., a world leader in CF research and development,” David Kirn, co-founder and CEO of 4D Molecular Therapeutics, said in a press release.

“The CF Foundation’s support … further validates the potential of our Therapeutic Vector Evolution platform, and the products engineered from our optimized and proprietary AAV vectors,” Kirn added.

4DMT’s Vector Evolution approach attempts to address problems evident in delivering gene therapies using current viral carriers. Viruses are often used to deliver genetic material into cells, since they are naturally capable of entering cells.

Non-infectious and safe, AAV are viruses typically found as contaminants in laboratories or as monkey infections. While they have been shown to be well-tolerated by patients, their delivery yields are poor — meaning their effectiveness as a therapy remains low and can require aggressive dosing, 4DMT states in the release.

To overcome this, the company is working to develop second-generation viral vectors that are better at delivering the gene therapy to the target cells through a chosen route of administration.

Cystic Fibrosis Foundation Therapeutics (CFFT) granted 4DMT $525,000 in funding in September 2016.

“The CFFT is a critical partner for 4DMT’s efforts to bring our transformative gene therapy product to CF patients,” said David Schaffer, co-founder and acting CSO of 4DMT.

“In addition to financial support, CFFT clinicians, scientists, and patient advocates will work closely with 4DMT to advance 4D-710 as efficiently and effectively as possible,” added Schaffer, who is also a professor of bioengineering and chemical engineering at the University of California, Berkeley.