Business Corpus: This Biotech Stock Is Flying On Its Promising Gene TherapyUltragenyx Pharmaceutical stock jumped Friday after its gene therapy showed promise in patients with an inherited disease that causes low blood sugar. The biotech studied three patients. The post This Biotech Stock Is Flying On Its Promising Gene Therapy appeared first on Investor's Business Daily.

[Collection]

Ultragenyx Pharmaceutical (RARE) jumped Friday after its gene therapy showed promise in patients with an inherited disease that causes low blood sugar.

On the stock market today, Ultragenyx stock leapt 7.9%, to 43, in higher than average volume. Meanwhile, shares of biotech stocks collectively rose just under 5%.

The biotech stock tested its gene therapy, DTX401, in three patients with glycogen storage disease. In this disorder, patients synthesize glycogen incorrectly, leading to low blood sugar. Ultragenyx studied three patients over the course of 12 weeks.

All three patients showed improved glycogen control — two of the results were clinically meaningful. Research suggests patients with low blood sugar can eat raw cornstarch before bedtime to help preserve glycogen in their bodies.

Two patients "experienced a sufficient increase in time to hypoglycemia (low blood sugar) such that they may be able to sleep through the night without taking supplemental cornstarch," Eric Crombez, chief medical officer of Ultragenyx's gene therapy unit, said in a written statement.

Gene Therapy Improves Glycogen Control

The three patients also showed improved glycogen control throughout the day, Ultragenyx said in a news release. That allowed them to decrease their daily cornstarch intake by about half.

"We are extremely encouraged by these results and look forward to beginning enrollment in the second (group) this month, with results expected in mid-2019," Crombez said.

As of the cutoff date on Nov. 28, there were no infusion-related side effects. Two patients had elevated liver enzymes, which can occur with this type of gene therapy. Both completed tapering steroid regimens in response.

Ultragenyx expects to dose the first patient in a new study this month. The new study will look at a higher dose of the gene therapy. An independent data monitoring committee has given the biotech stock the go ahead to begin the next round of testing.

YOU MIGHT ALSO LIKE:

Get Notifications For Live IBD Videos By Subscribing On YouTube

3 Gene Therapy Companies In Hot Pursuit Of A 'One-And-Done' Cure

Exclusive Q&A: AveXis President David Lennon Talks Gene Therapy In 2019

Looking For Growth? Take A Look At These Biotechnology Stocks

The post This Biotech Stock Is Flying On Its Promising Gene Therapy appeared first on Investor's Business Daily.