GenEdit, a Silicon Valley startup founded by Koreans, has signed a research collaboration agreement with U.S.-based gene editing company Sarepta Therapeutics to develop gene editing therapeutics for the treatment of four rare neuromuscular diseases.

Under the agreement announced on Tuesday, Nasdaq-listed Sarepta will obtain exclusive option rights to license polymer nanoparticles developed by GenEdit in the collaboration for up to four neuromuscular indications selected by Sarepta. GenEdit may receive up to $57 million in near-term payments and is also eligible for significant future development, regulatory and commercial milestones and tiered royalties ranging from upper-single to low-double digits on future product sales, according to a joint press release from the two.

GenEdit’s co-founder Park Hyo-min told Maeil Business Newspaper that GenEdit has been collaborating with Sarepta since December 2020, and their partnership has recently yielded significant results for new drug development.

Sarepta is a biotech company founded in 1980 and currently has a market capitalization of approximately $6 billion. It is focusing on the treatment of muscle diseases using gene scissors.

Sarepta has intellectual property rights from the development of its own genetic modulator and the two companies have confirmed that GenEdit’s nanopolymer is delivered to the disease-causing cells targeted by Sarepta, Park said.

The two are planning to investigate whether a modulator to cure these diseases can be delivered through the nanopolymer platform and work as an effective therapeutic.

According to Sarepta, the treatment market for the four types of muscle disease is currently worth about $6 billion and is expected to continue to grow until 2030.

The two companies plan to wrap the gene editing modulator, which acts as a treatment for myopathy, with GenEdit`s nanopolymer, making it in the form of solution for intravenous injection.

Sarepta’s chief executive officer Doug Ingram said he has been impressed with the diversity of GenEdit’s NanoGalaxy platform and its screening and selection process, which has generated distinct polymers that deliver to muscle.

GenEdit’s co-founder and CEO Lee Kun-woo, Ph.D., said that GenEdit has demonstrated that the NanoGalaxy platform can overcome historic challenges in the field and achieve tissue-selective delivery of a broad range of genetic medicine cargos.

GenEdit is developing gene platform NanoGalaxy. The potential of mRNA, the basis of gene therapy, has already demonstrated its potential as Covid-19 vaccines through Moderna and Pfizer. GenEdit and Sarepta see its potential as a gene therapy. If it is loaded onto mRNA and delivered to human cells properly, it can be used as a cancer drug.

There are still hurdles in the development as delivering mRNA remains a challenging method. GenEdit is focusing on developing polymer-based nanoparticles, which are high molecular compounds instead of a viral vector. This makes it possible to understand the interaction of nanoparticles with specific cells on a database and to use strategies to selectively deliver genes to specific cells only. GenEdit aims to become a pharmaceutical platform by building information about 50,000 nanoparticles as data.

GenEdit also plans to set up an R&D center in Korea to strengthen collaboration with global pharmaceutical companies. The company decided to establish a research institute in Korea, where there are many talented people, for various collaborations, Park explained.

GenEdit is currently receiving offers from many global pharmaceutical companies for gene therapy-related collaborations. The research center is scheduled to be built in Pangyo, Gyeonggi and will begin operations from April.

By Lee Sang-duk, Shin Hyun-kyoo and Minu Kim

[ⓒ Pulse by Maeil Business Newspaper & mk.co.kr, All rights reserved]