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Waksal’s Kadmon Spins Out Gene Therapy Group But Keeps Details Close

Xconomy New York — 

As it heads toward an IPO try, New York biotech Kadmon is lightening its load. The firm announced today it has spun out a gene-therapy subsidiary and expects it to have four products in clinical trials next year with a focus on eye disease.

The spinout, Kadmon Gene Therapy Holdings, or KGT, will be run by Alexandria Forbes, who was previously chief commercial officer at Kadmon and remains on the firm’s board of directors. Kadmon chairman and founder Samuel Waksal (pictured) told the audience at Xconomy’s New York biotech event in March that KGT would be an independent company and Kadmon would take an equity stake. From his description, the move was made with an eye not only toward developing the gene therapy platform, but also to streamline the parent company Kadmon as it prepares for an IPO.

“Kadmon has now exploded into a very large biotech company, getting ready to go public and do all the things biotech companies do in their growth periods,” Waksal said in March.

Waksal was the CEO of ImClone Systems, an antibody therapeutic company in New York at the center of the Martha Stewart-related insider trading scandal. Waksal was convicted of several felonies and spent nearly six years in jail. When he got out, he wasted little time declaring his intent to start a new biotech company. ImClone was sold to Eli Lilly for $6.5 billion in 2008.

His brother Harlan Waksal, who helped run ImClone, is Kadmon’s president and CEO.

Despite putting out a press release, the company declined to comment about the spinout or its gene therapy technology. [A previous version of this story quoted a spokeswoman to that effect.]

The release describes the gene therapy platform as “riboswitch technology.” That seems to differ from other high-profile gene therapy efforts that Xconomy has written about here, here, and here. As described elsewhere, such as this Nature paper, riboswitches naturally occur as a small part of bacterial RNA or ribonucleic acid, which delivers the genetic information that cells turn into proteins. In bacteria, riboswitches play a role in protein production.

Several researchers have published work related to blocking riboswitches in bacteria as an antibiotic drug strategy. But there is also a body of relatively new work that looks to create synthetic riboswitches as a way of turning up or down the amount of protein produced in a cell. Intrexon (NASDAQ: XON) is investigating what seems to be a similar approach, called “rheoswitch,” as a way to provide a more nuanced way to dial up or down the amount of protein expressed by a gene. The switch, in that case, is a pill given to the patient that regulates gene expression. Kadmon declined to elaborate today, but that description seems to jibe with Waksal’s description of Kadmon’s work from his talk in March.

“One of the other things that we just did…. is being able to control gene expression with small molecules, so [it’s] sort of a holy grail of what we’re supposed to do in the future of the world of gene therapy, putting a gene in for a protein, but being able to turn it on when you take a small pill, and just light up the expression of that protein,” he said. “We’ve just completed some of the work which showed we can really do that.”

Waksal formed Kadmon in 2010. The company is based out of the Alexandria Center for Life Science on Manhattan’s East Side—incidentally, the same complex that houses his old company, ImClone, which is now owned by Eli Lilly. Kadmon is developing therapies for a variety of diseases, among them autoimmune disorders and cancer, and it sells ribavirin, a staple of hepatitis C drug regimens.

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