What happened

After bluebird bio (NASDAQ:BLUE) updated investors on the presentations it will be making at a key industry conference soon, its shares jumped 24.2% in November, according to S&P Global Market Intelligence

So what

The company is on the cutting edge of gene-therapy research, and in November, management unveiled data that it will be presenting this month at the high-profile American Society of Hematology conference, which runs from December 9 to December 12.

Business people cheer on a beach.


Investigators will discuss updated results for bluebird bio's LentiGlobin in transfusion-dependent beta-thalassemia and severe sickle cell disease. They'll also update data for bb2121, their investigational chimeric antigen receptor T-cell therapy (CAR-T) for late-stage multiple myeloma. 

Transfusion-dependent beta thalassemia is a rare and severe genetic blood disease requiring lifelong supportive care, blood transfusions, and chelation treatments that remove heavy metals. These patients can suffer organ damage from the disease and iron overload, so there's an important need for new treatment approaches beyond allogeneic hematopoietic stem cell transplant (HSCT). HSCT can treat the underlying cause of beta-thalassemia, but it exposes patients to risks, including treatment-related mortality, graft failure, graft versus host disease, and infection.

The need for new treatments for sickle-cell disease is big, too. Patients with severe sickle-cell disease face anemia, vaso-occlusive crisis, infections, stroke, and sometimes, early death. Currently, treatment centers on preventing infection and disease management, which may include hydroxyurea drugs and, in certain cases, regular transfusions. HSCT can be used in these patients, too, but it still carries the same risks as in beta-thalassemia.

In addition to listening in to what bluebird bio has to say about LentiGlobin's safety and efficacy in those two important indications, industry watchers will also be listening carefully to the update provided for bb2121.

In June, bluebird bio stole the show at the American Society of Clinical Oncology conference when it reported that bb2121 delivered a 100% response rate in evaluable, heavily pre-treated, multiple myeloma patients. It's likely that response rates will slip over time, as more patients are evaluated, but it appears that the trial was a success given that bluebird bio's collaboration partner, Celgene (NASDAQ:CELG) is set to begin enrolling patients in a phase 2 trial soon that could allow for an accelerated Food and Drug Administration approval.

Now what

Gene therapy is a major advance in patient treatment, and bluebird bio's therapies have the potential to reshape patient standard of care in these indications. It's anyone's guess if data presented at the ASH conference will be confirmed in larger studies that can enable regulatory approvals, but there's good reason for industry watchers and investors to be buzzing and hopeful. After all, if these therapies are effective and safe, they could generate billions of dollars in combined sales -- if they eventually make it to market.