JPM17: Why the Key to Gene Therapy Could be in an Old Bay Area Warehouse

JPM17: Why the Key to Gene Therapy Could be in an Old Bay Area Warehouse January 13, 2017
By Alex Keown, Breaking News Staff

SAN FRANCISCO -- Bay Area-based gene therapy development-company Audentes is expecting a big 2017. The company anticipates entering the clinic this year with two gene therapies, Audentes Chief Executive Officer Matt Patterson told BioFlash, a podcast by the San Francisco Business Times.

Audentes is developing gene therapy treatments for X-Linked Myotubular Myopathy. The company, unlike many others, is building its own manufacturing capacities rather than using a third party contract manufacturing organization. In November, Audentes announced it had successfully initiated large-scale cGMP (current Good Manufacturing Practice) production runs in its new, state-of-the-art manufacturing facility. At the time, Patterson said the company having its own manufacturing facility will allow the company to retain “full control over costs, timelines and intellectual property associated with manufacturing our products.”

Audentes will manufacture two products, AT132 for X-Linked Myotubular Myopathy and AT342 for Crigler-Najjar Syndrome, at this facility. Both products are anticipated to enter the clinic this year. Additionally, the company announced it plans to establish internal cGMP manufacturing for our Pompe disease and CPVT programs this year as well. On Jan. 9, Patterson said the company should have preliminary data from its two lead products by the end of the year.

Patterson, who has worked in rare diseases for more than 20 years, said the science of making gene therapy treatments is very complex. He told BioFlash that doing so in a large scale was even more complex.

“You couldn’t just go to a CMO and say please make this drug,” he said. Patterson added that the contract manufacturing organizations just didn’t have the resources to make these medicines.

The company found a facility in San Francisco that had been used in biological manufacturing that needed some work, but Patterson said it was able to be modified to fit the company’s needs. What made this challenging from an investment standpoint was the company did not have any human data to take to its board for approval. But, a belief in Audentes’ research led to the approval by the board, Patterson said.

“This has given the company a whole new look… to have your own manufacturing,” he said.

Patterson said 2017 will build on the successes the company saw in 2016. In addition to the construction of its manufacturing facility, Audentes also saw its stock go public in July 2016. Shares of Audentes are currently trading at $17.64, which is down from its year-high of $20.74 in October. Also the company began enrolling patients in its Phase I/II trial X-Linked Myotubular Myopathy. As of Jan. 1, there were about 15 patients enrolled. XLMTM is a rare monogenic disease characterized by extreme muscle weakness, respiratory failure and early death, with an estimated 50 percent mortality rate by 18 months of age. AT132 is an AAV8 vector containing a functional copy of the MTM1 gene for the treatment of XLMTM.

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