bluebird bio, Inc. is a clinical-stage biotechnology company, which engages in researching, developing and commercializing potentially transformative gene therapies for severe genetic diseases. It also offers ZYNTEGLO or beti-cel and SKYSONA or eli-cel gene therapies. The company was founded by Philippe Leboulch and Ronald C. Dorazio on April 16, 1992 and is headquartered in Somerville, MA.
Clinical Trials
45
31 active
Approvals
0
Total approvals
Agencies
0
Regulatory bodies
Founded
1993
Active, not recruiting
30
66.7%
Completed
9
20.0%
Enrolling By Invitation
1
2.2%
Recruiting
4
8.9%
Terminated
1
2.2%
No approval data available
- PK MED appoints François Romaneix as Chairman and adds gene therapy pioneer Prof. Philippe Leboulch to its Board of Directors and Scientific Advisory Board. - Prof. Alain Fischer, internationally renowned immunologist and former President of the French Academy of Sciences, becomes Chairman of the Scientific Advisory Board. - The governance strengthening supports ARTHRELIS's upcoming direct entry into Phase 2 for refractory gout flares following FDA regulatory discussions. - The company aims for Phase 2 clinical results and a strategic licensing or M&A deal by 2028, while advancing ENGRAFTIS toward preclinical partnerships by 2027.
- CAR-T therapy Carvykti, originating from China's Legend Biotech, has produced durable long-term remissions in heavily pretreated multiple myeloma patients, with 33% remaining disease-free at five years. - The therapy uses llama-derived nanobodies targeting BCMA, offering superior efficacy over traditional antibody-fragment CAR-T approaches, with 100% response rates in early trials versus 80% for competitors. - China's streamlined clinical trial ecosystem enabled Legend to move from first patient dosing in 2016 to stunning data presentation by 2017, outpacing American counterparts. - The Carvykti story signals a broader shift in global drug discovery, with roughly half of all major drug licensing deals in 2026 involving Chinese-originated therapies.
- Chantez Sanford Jr., a 24-year-old from Southfield, became the first Michigan patient to receive Lyfgenia gene therapy for sickle cell disease at Children's Hospital of Michigan in December 2025. - The $3.1 million treatment involves extracting patient stem cells, genetically modifying them to produce healthy hemoglobin, and reinfusing them after chemotherapy to eliminate diseased cells. - Ninety-nine days post-treatment, Sanford has experienced no painful episodes or need for blood transfusions, showing significant health improvements including increased energy and better physical appearance. - Children's Hospital of Michigan is now preparing a second sickle cell patient for the same treatment, marking expansion of this breakthrough therapy in the state.
- Dr. Christian Loss, CEO of Neovii Pharmaceuticals AG, will retire after a distinguished career and be succeeded by Frédéric Prince PhD effective January 1st, 2026. - Prince brings extensive experience from senior roles at Roche's Pharma Research and Early Development, where he served as Head of Strategy and Chief of Staff to the Head of Oncology. - The new CEO previously led gene therapy programs at bluebird bio (now Genetix Biotherapeutics) and held leadership positions at Novartis Oncology across multiple countries. - Loss will continue his involvement with Neovii as a member of the Board of Directors following his transition from the CEO role.
- Agios Pharmaceuticals' mitapivat (Pyrukynd) met one of two primary endpoints in the Phase 3 Rise Up trial, significantly improving hemoglobin levels in 41% of sickle cell disease patients compared to 3% on placebo. - The drug failed to achieve statistical significance in reducing annualized sickle cell pain crises, with treated patients experiencing 2.6 crises per year versus 3.1 in the placebo group. - Despite mixed results, Agios plans to meet with FDA in early 2026 to discuss a supplemental new drug application for sickle cell disease indication. - The company's stock price fell nearly 50% following the announcement, while awaiting a December 7 FDA decision on mitapivat's approval for beta thalassemia.
- SOTIO Biotech has appointed Amy Jensen-Smith as Chief Scientific Officer and Ulrich Moebius as Chief Development Officer to strengthen its leadership team and advance its pipeline of antibody-drug conjugates and immunotherapies. - Dr. Jensen-Smith has been instrumental in developing SOTIO's lead preclinical ADC candidates SOT106 and SOT109, which target mesenchymal tumors and colorectal cancer respectively. - Dr. Moebius brings over 25 years of experience in drug development and previously served as SOTIO's chief of preclinical development from 2017 to 2024. - The appointments support SOTIO's focus on next-generation ADCs targeting solid tumors and its ongoing Phase 1 VICTORIA-01 study of SOT201 immunocytokine.
- St. Louis Children's Hospital is now administering Lyfgenia, an FDA-approved gene therapy developed by Bluebird Bio for treating sickle cell anemia patients. - The therapy involves extracting patient stem cells, genetically modifying them with a healthy hemoglobin gene, and reinfusing them after chemotherapy treatment. - Nearly 100,000 Americans suffer from sickle cell anemia, with the disease affecting 1 in every 265 African American births and reducing average life expectancy to approximately 40 years. - The treatment offers hope for patients like 20-year-old Martin Mwita Jr., who has endured decades of blood exchanges and pain crises associated with the genetic disorder.
- Bluebird bio has rebranded to Genetix Biotherapeutics after a $50 million private buyout by Carlyle and SK Capital Partners, returning to its original 1992 name. - The company plans to expand manufacturing capacity within the next year and improve access to its three approved gene therapies: Lyfgenia, Zynteglo, and Skysona. - New CEO David Meek aims to streamline treatment access for thousands of patients who could benefit from the company's genetic therapies despite being the market leader. - The rebrand comes amid regulatory challenges, including FDA restrictions on Skysona due to blood cancer risks and broader scrutiny of the cell and gene therapy sector.
- Kriya Therapeutics has secured $313 million in new funding to advance its pipeline of single-dose gene therapies targeting chronic conditions including geographic atrophy and trigeminal neuralgia. - The company's lead candidate KRIYA-825 for geographic atrophy has completed IND-enabling studies and is ready to enter clinical testing, while KRIYA-748 for trigeminal neuralgia is also in advanced development. - The funding comes amid regulatory challenges facing the gene therapy sector, including recent safety concerns with AAV-based therapies and FDA restrictions on other approved gene therapies.
- Half of hematologists now favor Casgevy, a CRISPR/Cas9-based gene therapy, over Zynteglo for treating transfusion-dependent β-thalassemia, according to new physician survey data from Spherix Global Insights. - Casgevy uses gene editing to reactivate fetal hemoglobin production, while Zynteglo employs gene addition via lentiviral vectors to restore normal hemoglobin, with physicians citing different advantages for each approach. - Access barriers including limited treatment center capacity, geographic distance, high costs, and lengthy insurance approvals continue to challenge equitable gene therapy delivery for this severe inherited blood disorder.
