Gene Therapy Stocks Drug Candidates Gain Value as Therapies Reach Market

October 10, 2019

Difei Yang, Ph.D., is Managing Director of Equity Research at Mizuho Americas, Research Division. Dr. Yang has been covering the biotech and pharma sector for nearly a decade, most recently as Managing Director at Aegis Capital.

She also worked in senior equity analyst roles at Brean Capital, R.F. Lafferty, WallachBeth Capital and Auriga Global Investors. In addition, she has held senior science, program management and business development roles within the pharmaceutical industry and has authored many granted U.S. patents and peer-reviewed scientific publications.

She holds a Ph.D. in chemistry from UCLA, as well as an MBA from Georgia State University and a B.S. degree in physics from Peking University, China.

In this exclusive 2,716 word interview in the Wall Street Transcript, Dr. Yang explores the valuation of certain key biotech stocks in her coverage.

“…In gene therapy, we continue to see developments for treating neurological disorders, blood disorders and liver-mediated disorders.

It’s actually across multiple therapeutic areas. A few common themes we’re observing in that space is that, number one, we tend to see the highest probability of success for those companies where they target a single gene mutation, and secondarily, when these companies are vertically integrated, they’re able to control the manufacturing and basically make products to support clinical development as well as commercialization.”

In gene therapy, we continue to see developments for treating neurological disorders, blood disorders and liver-mediated disorders…”

One example is curing a dreaded genetic disease in infants:

“…We cover a company called Audentes Therapeutics (NASDAQ:BOLD). They have a therapy that is currently in late-stage development treating X-linked myotubular myopathy — XLMTM — an inherited form of a neuromuscular disorder.

These patients are typically diagnosed when they are babies.

The disease involves severe muscle weakness, so not only do these babies not reach developmental milestones, they probably never acquired the skills to walk.

Muscle weakness also impacts their lung function. And the result usually leads to babies not being able to breathe on their own and require ventilators for support, in some cases 24 hours per day.

Audentes Therapeutics has a gene therapy. And based on early clinical data, what we’re seeing is that not only the babies are alive, they started getting off ventilator support…”

Get more detail on this and other biotech winners from this exclusive 2,716 word interview in the Wall Street Transcript.