Is Gene Therapy Medicine's Next Big Thing?

Bluebird Bio (BLUE) is advancing gene therapies through clinic using technology that Celgene (CELG) thinks could someday be used to treat cancer.

Jun 14, 2014 at 1:05PM

Bluebird Bio (NASDAQ:BLUE) is a small company with big ideas. The clinical stage biotechnology company is researching novel treatments that seek to insert missing genes into patient stem cells.

The science behind  Bluebird's approach is so compelling that Celgene (NASDAQ:CELG) inked a deal early last year that could prove worth hundreds of millions of dollars to Bluebird if the two succeed in developing cancer treatments using Bluebird's technology.

More recently, Bluebird's shares soared again last month, after the company reported that it will present data from a study involving its gene therapy treatment for beta-thalassemia patients at the 19th Annual Congress of the European Hematology Association, to be held in Milan later this week. Since that presentation is likely to spark renewed interest in the company, let's take a closer look at Bluebird, its clinical pipeline, and research surrounding gene therapy.

BLUE Chart

BLUE data by YCharts

Innovative treatment for rare disease
Bluebird has two clinical product candidates. The first is Lenti-D, a treatment for childhood cerebral adrenoleukodystrophy, or CCALD, that is in phase 2-3 trials.

CCALD is an ultra-rare, hereditary neurological disorder that affects young boys. Over time, the myelin sheath surrounding brain cells breaks down and the patient's adrenal glands fail to work properly, eventually resulting in death.


Source: Bluebird Bio.

Currently, patients are treated with stem cell transplants, in which stem cells are introduced to the body that contain the missing gene associated with the disease. However, fewer than 30% of donors are ideal matches and patients often suffer from complications tied to infection, graft failure, or graft versus host disease when non-matching donors are used.

Lenti-D circumvents those non-matching donor risks by modifying patients' own stem cells to include a functioning version of the missing gene. So far, Lenti-D has succeeded in four patients, all of whom have seen disease progression halted.

Bluebird is also studying LentiGlobin as a treatment for beta-thalassemia and sickle cell disease, and LentiGlobin will be the subject of this weekend's presentation.

LentiGlobin works similarly to Lenti-D by inserting a fully functional human beta-globin gene into patient stem cells to prevent rejection. Bluebird treated its first patient with Lenti-D in 2010 and started a phase 1-2 study in the U.S. in March.

Gene therapies' appeal
It's a bold approach, but gene therapy has been gaining momentum following the approval of Dendreon's (NASDAQ:DNDN) immunotherapy drug Provenge in 2010. While Provenge differs from gene-modifying drugs like Bluebird's (and its commercial success or failure is an ongoing debate), the approval of the therapy, which involves removing patient blood cells, modifying them to contain CD54+ T cells, and returning them to the patient, has given hope to researchers that cell-based therapies are viable. Provenge, which costs $93,000 per patient, posted $325 million in sales during 2012 and $284 million in sales last year.

Provenge's approval was followed in 2012 with the approval of uniQure's Glybera in the European Union. Glybera also targets an ultra-rare disease, familial lipoprotein lipase, by replacing a missing enzyme. While Glybera has yet to produce material sales for uniQure, its approval reinforces that gene therapies can win over regulators.

Amgen (NASDAQ:AMGN) hopes that regulators' willingness to consider gene therapies may help it secure approval of its own gene therapy candidate, T-VEC, a treatment for metastatic melanoma.

Amgen reported in March 2013 that 16% of 400 patients treated with T-VEC responded to the therapy, far outpacing the 2% response rate for patients treated in the control group. Amgen acquired T-VEC in its $425 million purchase of Biovex, a company formerly run by uniQure's President of U.S. operations, and Amgen is currently compiling evidence in support of a potential FDA filing later this year.

Fool-worthy final thoughts
Celgene paid Bluebird Bio $75 million upfront and agreed to pay Bluebird up to $225 million in fees and milestones for therapies resulting from its collaboration on modified T-cell gene therapies for cancer. That's a big endorsement; however, gene therapy remains in its infancy, and the two companies have yet to move any drugs from their collaboration into human trials. That suggests that Bluebird, while intriguing, remains highly speculative and suited only for the most aggressive investors. 

Leaked: This coming blockbuster will make every biotech jealous
The best biotech investors consistently reap gigantic profits by recognizing true potential earlier and more accurately than anyone else. Let me cut right to the chase. There is a product in development that will revolutionize not how we treat a common chronic illness, but potentially the entire health industry. Analysts are already licking their chops at the sales potential. In order to outsmart Wall Street and realize multi-bagger returns you will need to Motley Fool’s new free report on the dream-team responsible for this game-changing blockbuster. CLICK HERE NOW.


Todd Campbell has no position in any stocks mentioned. Todd owns E.B. Capital Markets, LLC. E.B. Capital's clients may or may not have positions in the companies mentioned. Todd owns Gundalow Advisors, LLC. Gundalow's clients do not have positions in the companies mentioned. The Motley Fool recommends Celgene. Try any of our Foolish newsletter services free for 30 days. We Fools don't all hold the same opinions, but we all believe that considering a diverse range of insights makes us better investors. The Motley Fool has a disclosure policy.

1 Key Step to Get Rich

Our mission at The Motley Fool is to help the world invest better. Whether that’s helping people overcome their fear of stocks all the way to offering clear and successful guidance on complicated-sounding options trades, we can help.

Feb 1, 2016 at 4:54PM

To be perfectly clear, this is not a get-rich action that my Foolish colleagues and I came up with. But we wouldn't argue with the approach.

A 2015 Business Insider article titled, "11 websites to bookmark if you want to get rich" rated The Motley Fool as the #1 place online to get smarter about investing.

"The Motley Fool aims to build a strong investment community, which it does by providing a variety of resources: the website, books, a newspaper column, a radio [show], and [newsletters]," wrote (the clearly insightful and talented) money reporter Kathleen Elkins. "This site has something for every type of investor, from basic lessons for beginners to investing commentary on mutual funds, stock sectors, and value for the more advanced."

Our mission at The Motley Fool is to help the world invest better, so it's nice to receive that kind of recognition. It lets us know we're doing our job.

Whether that's helping the entirely uninitiated overcome their fear of stocks all the way to offering clear and successful guidance on complicated-sounding options trades, we want to provide our readers with a boost to the next step on their journey to financial independence.

Articles and beyond

As Business Insider wrote, there are a number of resources available from the Fool for investors of all levels and styles.

In addition to the dozens of free articles we publish every day on our website, I want to highlight two must-see spots in your tour of

For the beginning investor

Investing can seem like a Big Deal to those who have yet to buy their first stock. Many investment professionals try to infuse the conversation with jargon in order to deter individual investors from tackling it on their own (and to justify their often sky-high fees).

But the individual investor can beat the market. The real secret to investing is that it doesn't take tons of money, endless hours, or super-secret formulas that only experts possess.

That's why we created a best-selling guide that walks investors-to-be through everything they need to know to get started. And because we're so dedicated to our mission, we've made that available for free.

If you're just starting out (or want to help out someone who is), go to, drop in your email address, and you'll be able to instantly access the quick-read guide ... for free.

For the listener

Whether it's on the stationary exercise bike or during my daily commute, I spend a lot of time going nowhere. But I've found a way to make that time benefit me.

The Motley Fool offers five podcasts that I refer to as "binge-worthy financial information."

Motley Fool Money features a team of our analysts discussing the week's top business and investing stories, interviews, and an inside look at the stocks on our radar. It's also featured on several dozen radio stations across the country.

The hosts of Motley Fool Answers challenge the conventional wisdom on life's biggest financial issues to reveal what you really need to know to make smart money moves.

David Gardner, co-founder of The Motley Fool, is among the most respected and trusted sources on investing. And he's the host of Rule Breaker Investing, in which he shares his insights into today's most innovative and disruptive companies ... and how to profit from them.

Market Foolery is our daily look at stocks in the news, as well as the top business and investing stories.

And Industry Focus offers a deeper dive into a specific industry and the stories making headlines. Healthcare, technology, energy, consumer goods, and other industries take turns in the spotlight.

They're all informative, entertaining, and eminently listenable ... and I don't say that simply because the hosts all sit within a Nerf-gun shot of my desk. Rule Breaker Investing and Answers contain timeless advice, so you might want to go back to the beginning with those. The other three take their cues from the market, so you'll want to listen to the most recent first. All are available at

But wait, there's more

The book and the podcasts – both free ... both awesome – also come with an ongoing benefit. If you download the book, or if you enter your email address in the magical box at the podcasts page, you'll get ongoing market coverage sent straight to your inbox.

Investor Insights is valuable and enjoyable coverage of everything from macroeconomic events to investing strategies to our analyst's travels around the world to find the next big thing. Also free.

Get the book. Listen to a podcast. Sign up for Investor Insights. I'm not saying that any of those things will make you rich ... but Business Insider seems to think so.

Compare Brokers