Gene therapy is front and center in an agreement between MaxCyte Inc. and Northern Therapeutics Inc.

The companies entered a collaboration and license agreement to develop multiple nonviral, gene-based products to treat pulmonary diseases using MaxCyte's cell-loading system. MaxCyte's GT technology is designed to enable and improve nonviral therapeutic gene transfer. The parties will work to develop gene-based therapeutics for specific indications that can be administered at a patient's bedside.

Rockville, Md.-based MaxCyte will receive up-front and milestone payments and research and development funding, as well as royalties from resulting products. More specific financial terms were not disclosed.

"I think this is a very balanced deal," MaxCyte President and CEO Douglas Doerfler told BioWorld Today. "It really validates our business plan, which is working with companies that have a need for a nonviral, cell-loading platform. We provide them that platform in exchange for the funding required to move this program through the clinic and eventually through commercialization. At that point we both share in an improved product."

Northern Therapeutics has developed a cell-based gene therapy for pulmonary arterial hypertension and other chronic, life-threatening pulmonary disorders - a technology that provides a platform for the regeneration of damaged lung tissue by improving distal pulmonary arterial circulation.

"They have a gene on which we are working with them, and they are looking for a nonviral method to load cells that can be used directly in treating the patient," Doerfler said. "We're working together to develop the protocol and then the submission to regulatory authorities, and will work together in developing the program clinically."

Montreal-based Northern Therapeutics pointed to a synergy as well.

"This is a nonviral approach that can achieve very good efficiency and is very well suited to ex vivo transfection - exactly what we're doing," Duncan Stewart, Northern Therapeutics' chief scientific officer, told BioWorld Today. "As we move forward into clinical trials, their technology is going to greatly enhance the transfection efficiency without having to resort to viruses."

The companies said they plan to file with the FDA their first investigational new drug application later this year, positioning them to begin a Phase I/II study in pulmonary arterial hypertension patients during the third quarter. The companies plan to reference in the application material from a separate clinical program already using MaxCyte's GT technology.

Stewart, who will direct the program, said the timeline is manageable.

"We're working with regulatory authorities to achieve that goal," he said. "We think it is feasible."

Last summer, MaxCyte entered a research agreement with another Montreal-based partner, Angiogene Inc., to develop a recuperative treatment for congestive heart failure caused by myocardial infarction using the GT cell-loading system. In the agreement, the companies aim to load a patient's muscle precursor cells with a patented gene construct to stimulate angiogenesis in the diseased myocardium of the heart. (See BioWorld Today, July 11, 2002.)

Outside of its collaborative work, MaxCyte's pipeline includes one therapeutic, a cell-based oxygen product, in Phase I trials at the University of Cincinnati. A second Phase I trial, planned to begin in the next quarter, is designed to test another therapeutic to treat chronic lymphocytic leukemia. The study is being carried out in collaboration with the Baylor College of Medicine in Houston.

Several late-stage preclinical therapeutic candidates are focused on oncology, cardiovascular and genetic diseases.

Northern Therapeutics' core technology centers on cell-based regenerative therapies for lung diseases. Beyond its proof-of-principle work in pulmonary arterial hypertension, Northern Therapeutics also is exploring other applications, including adult respiratory distress syndrome, among others.

Its largest shareholder is Silver Spring, Md.-based United Therapeutics Corp., for which it is the Canadian distributor of Remodulin, HeartBar and other United Therapeutics products.