Member Spotlight: Q&A with bluebird bio

Apr 16, 2024

Every month, MassBio spotlights a member company and its efforts in advancing the life sciences industry and supporting the patients we serve. In April, we profile bluebird bio and speak with Tom Klima, chief commercial and operating officer of the Somerville-based gene therapy company. Tom, who joined bluebird in May 2021, has deep passion for patients and experience across multiple commercial roles in both rare diseases and oncology.

bluebird custom designs each of its investigational therapies with the goal of addressing the underlying cause of disease at the genetic level. bluebird is currently developing gene therapies for severe genetic diseases and is advancing research to apply new technologies to these and other diseases. More than 170+ patients have received bluebird’s therapies across 8 clinical trials.

The following is an edited transcript of the conversation between Kendalle Burlin O’Connell and Tom Klima filmed on Thursday, April 11, 2024.

Kendalle: Tom, bluebird is a Massachusetts homegrown company. Tell us a little bit about what bluebird is working on. Tell us a little bit about your role; share with the membership what you’ve got going on.

Tom: It’s a really exciting time at bluebird right now. And keep in mind that bluebird has been around for over a decade now, first leading the way in gene therapy R&D, but now making a successful transition to gene therapy commercialization. If you look at just our R&D history, we have an extensive, actually the largest ex vivo gene therapy database in the world with over 180 patients studied during our development programs. And now we’re transitioning to a commercial setting where we now have our three approved gene therapies First, back in August 2022, our therapy ZYNTEGLO® for β-thalassemia was approved by the FDA, obviously a huge milestone for patients with β-thalassemia who have to go through regular brain blood cell transfusions every couple of weeks just to survive.

Right after that, our second therapy was approved for cerebral adrenoleukodystrophy or CALD, and that obviously was a landmark for blue for a long time. It’s a very horrible neuro-degenerative disorder. So there’s a great option for patients that are suffering from CALD.

And then most recently in December, LYFGENIA™ was approved for sickle cell disease and patients have with sickle cell disease who have vaso-occlusive events and they’re in and out of the hospital. So just a great option that’s potentially curative for patients with sickle cell disease.

Kendalle: So you’re located in Somerville. Tell me a little bit about why Somerville? What attracted bluebird to Somerville and tell us some of the advantages of being there.

Tom: So it’s just outstanding being in Somerville. We have our office at Assembly Row, and then we have our lab space down in Charlestown under Hood. If you back up a few years ago, remember as we were kind

of evolving our needs as a company, we split into two companies. We had bluebird and we had 2Seventy. bluebird was focused on severe genetic disorders. As our needs change for our business, we were very excited to move to Somerville where we could accommodate this growing environment where we had people working remotely and hybrid, but also, once you look at our, our costs of being in Cambridge versus up in Somerville, we’re able to reduce our cost structure so that we can really focus on building our commercial infrastructure. So it’s great being up there. It’s been a great little community and our birds, as we call them, are very, very happy up there.

Kendalle: I love it. Assembly Row is so cool. When we think about all their clusters, what do they really need to build in? They’ve done all of that … work, life, play is all happening right over there.

Tom: That’s right. The transportation is easy, and then everything you can have from food to shopping is all right there. So it’s a great little commute.

Kendalle: So we’ve talked a lot about your focus on gene therapy, and I think Massachusetts is playing a really important role right now in the future of gene therapy. You’ve got three approved therapies. Talk to us a little bit about how Massachusetts has been helpful in bluebird accelerating these therapies, specific to gene therapies.

Tom: Oh, absolutely. If you think about gene therapy here, the product is really the process. So the process starts at cell collection, and the manufacturing process actually is a lot different than most therapies that we talk about historically. It starts with cell collection and the patient goes all the way through manufacturing and then numerous assays for testing for things like viability, dose for the gene therapy, and then through the infusion to the patient. So that whole process requires a network of partners–CDMOs that we work very closely with and we’ve had long relationships with. So gene therapy is really just a big process where it’s all involved with all the partners, locally and abroad.

Kendalle: What are some of your biggest challenges as it relates to innovating specifically in the gene therapy space? Is it the FDA? Is it payers? Is it just generally the regulatory landscape?

Tom: I wish I could say gene therapy was easy, but there are many challenges. You hit on a couple of big ones. I would say let’s start with the manufacturing process. It’s extremely complex and it takes time. It’s longer than most therapies and it’s our job really to make it as simple and seamless as possible for patients and for hospitals serving patients.

Secondly, obviously payers. The concept of a one-time potentially curative therapy in these incredibly devastating and costly conditions is somewhat of a new concept. You have to work with a lot of different audiences to make sure that they understand the value of a one-time potentially curative therapy.

And so finally, working with the FDA and regulators and making sure that we have a plan for the long-term commitments that come with gene therapies. You have to follow patients for up to 15 years after their therapy so it doesn’t stop with manufacturing. It doesn’t stop with the treatment. It actually lasts for an ongoing up to 15 years. So that’s an additional thing that is new and different and a challenge for gene therapy.

Kendalle: You just hit on really having to follow that sort of life cycle of your patients. Talk to me a little bit about the importance of patients to the bluebird community.

Tom: Everything we do is really founded based on patients and the patient experience and going all the way back to our founders and our former birds, patients are at the center of everything we do. Not only is it the center of the manufacturing process for our gene therapies, it’s really at the heart of all the decisions that we make. And I talked about CALD and SKYSONA®, that was really the mission of bluebird to bring that therapy forward. That’s only for tens of patients. So it’s a very small patient population, but it’s the passion behind what drove bluebird.

Kendalle: I’ve always known that about the bluebird community. We have a hashtag that we use here, #PatientDriven, and I think bluebird has truly always embodied that sentiment.

Tom: That’s outstanding. It drives the passion in every person at bluebird, and we call it the nest, a passion for patients.

Kendalle: So we’ve hit on this a few different times: Manufacturing. I’d love to get your perspective on the potential of the future particularly of biomanufacturing here in Massachusetts.

Tom: Yeah, as I mentioned before, the process and the product are synonymous. They’re one and the same. The process is the product, and the product is the process. So we have to work with the big CDMOs and other partners as we scale. And again, I mentioned that CALD really serves tens of patients, but as you think about larger indications like people living with sickle cell disease, there’s over a hundred thousand people in the United States who have sickle cell disease and about 20,000 who have severe sickle cell disease and has a history of VOEs where they might need a gene therapy. So really scaling the whole process becomes incredibly important as you reach different patient populations.

Kendalle: And I think that’s what is truly unique about Massachusetts particularly, right? Think about a patient population that had no therapy. And now because of the work that you are doing and others here in the Massachusetts ecosystem, we can say the sickle cell community, so many others now have a therapy. It’s pretty amazing stuff that’s happening here in the Commonwealth.

What is in store for bluebird in 2024 and beyond?

Tom: So 2024 and beyond, in the short term and the long term, right now, everything at bluebird is dedicated to bringing these three gene therapies to more patients in need, really focused on our three US launches right now. That’s where we’re stopping right now. We’ll see what the future brings beyond that, but we’re really busy with our three launches going on right now.

Kendalle: Well thank you so much for being here. Thank you to bluebird and the entire team for the incredible work you do each and every day to change the lives of patients, not just here in the Commonwealth but around the world.

Tom: Thank you for all you guys do as well. Thank you.

About bluebird bio, Inc.
bluebird bio is pursuing curative gene therapies to give patients and their families more bluebird days.

Founded in 2010, bluebird has been setting the standard for gene therapy for more than a decade—first as a scientific pioneer and now as a commercial leader. bluebird has an unrivaled track record in bringing the promise of gene therapy out of clinical studies and into the real-world setting, having secured FDA approvals for three therapies in under two years. Today, we are proving and scaling the commercial model for gene therapy and delivering innovative solutions for access to patients, providers, and payers.

With a dedicated focus on severe genetic diseases, bluebird has the largest and deepest ex-vivo gene therapy data set in the field, with industry-leading programs for sickle cell disease, β-thalassemia and cerebral adrenoleukodystrophy. We custom design each of our therapies to address the underlying cause of disease and have developed in-depth and effective analytical methods to understand the safety of our lentiviral vector technologies and drive the field of gene therapy forward.

bluebird continues to forge new paths as a standalone commercial gene therapy company, combining our real-world experience with a deep commitment to patient communities and a people-centric culture that attracts and grows a diverse flock of dedicated birds.

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