What happened

After updating investors on its wide-ranging gene therapy research program at a key industry conference early in the month, shares of bluebird bio (NASDAQ:BLUE) surged 20.7% higher in January, according to S&P Global Market Intelligence.

So what

At the J.P. Morgan Healthcare Conference in early January, Bluebird Bio's management outlined how it hopes to transform treating rare disease, including cerebral ALD (CALD), transfusion dependent beta thalassemia (TDT), and sickle cell disease.

A man speaking into a megaphone in front of a rising stock price chart

Image source: Getty Images.

The company provided an outlook for 2022 that includes a goal of having two gene therapies on the market, two other therapies near commercialization, and four or more research programs in clinical studies.

In 2017, Bluebird Bio's plans include prepping a filing of its TDT therapy, LentiGlobin, for approval in Europe, and developing a pathway to regulatory approval of its CALD therapy, Lenti-D.

The company is also going to continue early stage research into its CAR-T program, including its work on bb2121, a BCMA-targeting therapy that's being developed with Celgene (NASDAQ:CELG) for multiple myeloma.

Now what

The potential to significantly reduce, or eliminate, the need for blood transfusions in TDT patients has industry watchers estimating that LentiGlobin could reshape patient treatment. If so, this gene therapy could be a nine-figure (or higher) top-seller. A similar opportunity exists for Lenti-D.

Perhaps most compelling, however, is the potential market opportunity for bb2121. Although a number of new multiple myeloma treatments have been launched over the past few years, the need for new treatment options remains high. Roughly 30,000 people are newly diagnosed with myeloma in the U.S. each year, and sadly, the five-year survival rate is just 48.5%, according to the National Cancer Institute. Clinical trials for bb2121 are early stage studies, so a lot could go wrong from here. But successfully targeting BCMA and improved outcomes without a lot of safety risks could significantly change how doctors treat their patients. If that happens, bb2121 could become a billion-dollar blockbuster someday.

 

Todd Campbell owns shares of Celgene. His clients may have positions in the companies mentioned. The Motley Fool owns shares of and recommends Celgene. The Motley Fool recommends Bluebird Bio. The Motley Fool has a disclosure policy.