REGENXBIO Banks $70.5 Million As Gene Therapy Enjoys Revival
May 20, 2015
By Riley McDermid and Mark Terry, BioSpace.com Breaking News Staff
Washington, D.C.-based REGENXBIO, announced today that it had completed a $70.5 million funding round, as investors go shopping for the hottest new technologies in gene therapy and are willing to bet on even tiny companies to do it.
REGENXBIO has only 18 employees but has raised $100.5 million since the beginning of January, in two capital raises. The latest one included investors Venrock, the hedge fund Brookside Capital and a mutual fund run by Janus Capital Group Inc.
The company said in January that it would use that earlier round to make new hires, but said Wednesday it would use this round to boost its plans for clinical studies and round out its pipeline.
"With this financing, we are well positioned to support our lead programs through initial clinical studies and to establish a number of new clinical candidates in order to grow our pipeline of NAV Technology-based treatments in development," said Ken Mills, president and chief executive of REGENXBIO, in a statement.
Investors had long been wary of gene therapies, but new technologies have made the area a much more attractive bet in recent years, particularly as biotech’s bull market has continued.
“We have much safer ways to deliver DNA into tissue,” Daniel Krizek, a director at Brookside Capital, told Bloomberg, adding that recent trials have shown efficacy. “The science has advanced.”
Joshua Schimmer, a closely followed biotech analyst at Piper Jaffray, said his data showed that gene therapy companies have raked in $2 billion in public offerings since 2013, and at least $400 million from private sources during the same period.
“Everything has changed,” Schimmer told Bloomberg. “Now the capital is aplenty, the technologies are ready and companies are being formed quickly.”
REGENXBIO has certainly reaped the rewards of that renewed confidence. In January the company closed a $30 million Series C financing. The round was led by Enrick and Brookside Capital. Also participating were Deerfield Management and an unnamed new investor.
Also participating were existing investors, FoxKiser and Fidelity Biosciences. Two individuals will be joining REGENXBIO’s board of directors, Camille Samuels, a partner at Venrock, and Michael Gelman.
“This Series C financing further validates our NAV Technology platform and facilitates our ability to bring life-changing gene therapies to patients suffering from serious diseases for which better treatment options are needed,” said Mills at the time.
“The proceeds will enable us to initiate clinical proof of concept data for our lead programs, which we plan to advance rapidly to registration, and allow us to remain opportunistic in regards to in-licensing new programs,” he said. “In addition, the funds will be used to strengthen our clinical and manufacturing capabilities and team, making us well-positioned to develop and commercialize our pipeline of AAV gene therapy treatments.”
Founded in 2009, REGENXBIO has the goal of driving the broad development of AAV gene therapy. The company acquired exclusive proprietary rights to NAV Technology, which is inclusive of recombinant AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. This was researched developed by the company’s scientific founder, James Wilson, and his team at the Gene Therapy Program of the University of Pennsylvania. In November 2014, Wilson joined the company as chief scientific advisor.
In 2014 REGENXBIO developed a pipeline of treatments for lysosomal storage disorders and ocular diseases. It has licensed its NAV Technology to eight gene therapy companies for possible treatment for more than 12 different diseases. Those companies include Baxter Healthcare Corporation , Fondazione Telethon, Audentes Therapeutics, Lysogene, Esteve, AveXis, AAVLife and Voyager Therapeutics.
The NAV Technology is used to develop treatments for diseases such as Hurler Syndrome, Hunter Syndrome, Wet Age-Related Macular Degeneration, X-Linked Retinitis Pigmentosa, and various Lysosomal Storage Disorders and ocular diseases.
Adeno-associated virus (AAV) vectors are considered some of the most promising approaches to gene therapy. In this technology, adeno-associated viruses vectors are used to directly deliver functional genes to the target cells. NAV Technology utilizes natural isolates of AAV that are mostly non-immunogenic, allowing for gene therapy applications with fewer immune responses from subjects.
“With its technological lead, IP, and strong leadership, REGENXBIO is uniquely positioned to lead the next generation of gene therapy companies,” said Camille Samuels in a statement. “I’m delighted to be joining the REGENXBIO board and to be given the opportunity to work with a great company to help patients.”
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