Redpin Therapeutics Inc., of New York, plans to take its newly secured $15.5 million series A financing to continue developing its ion-channel based chemogenetics platform for addressing neural circuit dysfunctions such as epilepsy, neuropathic pain and Parkinson’s disease.

It’s a big step forward for the company, founded in 2017, that leverages a system developed by the lab of Scott Sternson, a group leader at Howard Hughes Medical Institute’s Janelia Research Campus. The system facilitates the targeted creation of engineered ion channels. The channels, created using specially designed proteins encoded by DNA delivered via adeno-associated virus vectors, can then be modulated through low doses of varenicline, also known as Chantix, which is approved by the FDA as a smoking cessation agent. The company said varenicline is particularly attractive for chemogenetic applications in the CNS because it is well-tolerated at low doses and has excellent brain penetration.

Though early, potential therapeutic applications of the approach could open new avenues to treating patients with challenging neurological issues without the side effects experienced with other treatments.

“The majority are marginally effective,” Elma S. Hawkins, Redpin’s president, CEO and co-founder, said of currently available therapies. “They have a lot of side effects associated with them. In some cases, epileptic drugs can cause tremendous drowsiness. It has a real impact” on patients’ lives.

Elma S. Hawkins, president, CEO, co-founder, Redpin

Hawkins said the company’s preclinical research demonstrated the application of its technology has a host of other, different indications, such as appetite control, anxiety, pain relief and movement disorders such as Parkinson’s disease.

The company and its platform are a good investment, according to Dmitry Kuzmin, managing partner at 4Bio Capital, because it’s time for a new vision of gene therapy.

“We think gene therapy has come to a place where you can take from its first generation and apply its learnings into creation of a second generation,” Kuzmin told BioWorld. “The key is better control to change the identity of gene therapy, better targeting.”

Kuzmin said he is bullish on the application of new CNS technology, such as Redpin’s.

“If you go back over 30 years of drug development, the track record is abysmal,” he said. “I think the key reason is small molecules don’t have persistence in the brain.”

That lack of persistence has led to drugs with unwanted side effects, he added.

Hawkins was reticent to say much in terms of timing estimate for getting into the clinic, but she said she was pleased with the company’s progress.

“What has been gratifying is knowing that we’re working on such a solid base,” she told BioWorld. “It makes our lives as development scientists a lot of easier going from research to development. As I look back over the past year and a half, I’m happy with both the quality and quantity of work we’ve accomplished. It positions us well on the road to the clinic.”

She also said the financing will allow Redpin, which takes its name from the red pin used in GPS apps to mark a targeted destination, to add staff members to its roster. While she declined to give a number, she said the hires will be in the “general area of development. We’re really past the research phase and so the next development of the company is product development.”

In March 2019, Redpin announced it planned to use the Howard Hughes Medical Institute’s chemogenetics platform to develop ion channel gene therapy for neurological and psychiatric disorders worldwide. The institute’s Sternson and Jeffrey Friedman of Rockefeller University shouldered much of the research that has produced Redpin’s technology.

The funding round was led by 4Bio Capital, a London-based fund investing exclusively in cell and gene therapies and RNA drugs, and Arkin Bio Ventures. The effort was joined by a new Redpin investor, Takeda Ventures Inc., and existing investors from the seed round, New York Ventures and Alexandria Venture Investments.

4Bio’s Kuzmin will join Redpin’s board as a nonexecutive director.

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