Mines D`or Orbec Inc
🇺🇸United States
- Country
- 🇺🇸United States
- Ownership
- Public
- Established
- 1993-01-01
- Employees
- 375
- Market Cap
- $121.4M
- Website
- http://www.bluebirdbio.com
- Introduction
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.
CRISPR Gene Editing Breakthrough Saves Baby with Ultra-Rare Metabolic Disorder
• Doctors at Children's Hospital of Philadelphia successfully treated a baby with severe CPS1 deficiency using a personalized CRISPR base-editing therapy, marking a first-of-its-kind approach for this rare metabolic disorder.
• The experimental treatment, developed within just six months of diagnosis, corrected the infant's specific genetic mutation by delivering edited DNA to liver cells via lipid nanoparticles, allowing him to reduce medication and process more dietary protein.
• This breakthrough demonstrates the potential for creating customized gene therapies for millions with rare genetic diseases, with researchers suggesting costs comparable to liver transplantation and possibilities for treating numerous other conditions.
Leap Therapeutics Halves Workforce and Refocuses Cancer Drug Development Amid Market Challenges
• Leap Therapeutics has announced a significant restructuring, reducing its workforce by approximately 50% and narrowing the development focus of its lead cancer drug candidate in response to challenging market conditions.
• The strategic pivot aims to extend the company's cash runway while concentrating resources on the most promising clinical applications of its lead oncology asset, potentially improving its chances for regulatory success.
• This move follows similar restructuring trends across the biotech sector, with companies like Arcturus, NGM Bio, and Erasca all recently announcing staff reductions and pipeline reprioritizations to navigate the difficult funding environment.
Eli Lilly Commits $250 Million to Expand Purdue University Research Alliance Through 2032
• Eli Lilly has extended its research collaboration with Purdue University through 2032, investing up to $250 million over the next eight years in what could become the largest industry-academic partnership in the United States.
• The expanded alliance will facilitate researcher exchanges between Purdue's campus and Lilly's Indianapolis facilities, while also utilizing Indiana's LEAP Research and Innovation District to accelerate drug discovery and development.
• This partnership represents a significant investment in pharmaceutical innovation infrastructure, focusing on improving both drug discovery processes and manufacturing capabilities in the U.S.
Pfizer Withdraws Sickle Cell Drug Oxbryta Globally Following Safety Concerns
• Pfizer has voluntarily withdrawn its sickle cell disease therapy Oxbryta (voxelotor) from all global markets after discovering an imbalance in deaths during clinical trials.
• The withdrawal marks a significant setback for the sickle cell community as Oxbryta was the first approved medication that directly prevented sickle hemoglobin polymerization, the molecular basis of the disease.
• The decision follows Pfizer's $5.4 billion acquisition of Global Blood Therapeutics in 2022, with Oxbryta having generated $328 million in sales last year and previously projected to reach $2 billion in peak annual revenue.
Next-Generation Gene Therapies: Evolving Beyond Viral Vectors Towards More Affordable, Sustainable Solutions
• Despite 32 approved gene therapies globally, the industry faces significant challenges in safety, efficacy, and affordability, prompting development of novel delivery systems beyond traditional viral vectors.
• Companies are advancing non-viral delivery platforms including exosomes, lipid nanoparticles, and hydrophilic nanoparticles that offer cost-effective alternatives with reduced immunogenicity and potential for repeat dosing.
• Next-generation gene editing technologies like Prime Editing and CRISPR variants are emerging as more precise alternatives to traditional CRISPR-Cas9, with Prime Medicine's PM359 for chronic granulomatous disease advancing to clinical trials.
FDA's Cell and Gene Therapy Champion Peter Marks Departs, Leaving Industry at Critical Juncture
• Peter Marks, head of FDA's Center for Biologics Evaluation and Research since 2016, has resigned, leaving cell and gene therapy developers without their biggest regulatory advocate during a challenging investment period.
• Under Marks' leadership, the FDA approved dozens of cell and gene therapies including the first gene therapy, first cellular treatment for cancer, and first CRISPR gene editing medicine, establishing flexible regulatory frameworks for these novel treatments.
• Despite concerns about regulatory uncertainty following Marks' departure, incoming FDA Commissioner Marty Makary has signaled support for conditional approval pathways for rare disease treatments where randomized controlled trials aren't feasible.
FDA Rejects Hengrui-HLB Liver Cancer Combination Therapy for Second Time
• The U.S. Food and Drug Administration has issued a second rejection for the liver cancer combination therapy developed by Jiangsu Hengrui Medicine and HLB, dealing a significant setback to their oncology program.
• The combination therapy, targeting advanced hepatocellular carcinoma, failed to meet regulatory standards despite previous promising clinical data in Asian populations.
• This rejection highlights the ongoing challenges in developing effective treatments for liver cancer, which remains one of the deadliest cancer types with limited therapeutic options.
New York Man Becomes First in State to Be Cured of Sickle Cell Disease with Lyfgenia Gene Therapy
• 21-year-old Sebastien Beauzile has been successfully treated for sickle cell disease at Cohen Children's Medical Center using Bluebird Bio's Lyfgenia gene therapy, becoming the first New Yorker to receive this breakthrough treatment.
• The FDA-approved therapy works by extracting the patient's blood stem cells, genetically modifying them to produce functional hemoglobin, and reinfusing them after chemotherapy, effectively curing the disease rather than just managing symptoms.
• After suffering from severe pain rated "10 out of 10" since infancy, Beauzile is now symptom-free, calling the treatment "like a second birthday," and plans to pursue a medical career to help others with the condition.
GSK's Blenrep Secures UK Approval for Multiple Myeloma Treatment in Combination Therapy
• The UK's medicines regulatory body has approved GSK's Blenrep (belantamab mafodotin) in combination with other drugs for multiple myeloma patients whose first treatment failed or caused severe side effects.
• This approval marks a significant comeback for Blenrep, which was withdrawn from markets in 2022 after failing to outperform existing treatments when used as monotherapy.
• Clinical trials demonstrated Blenrep's combination therapy extended progression-free survival and overall survival compared to standard care regimens, including those based on Darzalex (daratumumab).
Bristol Myers Squibb Acquires 2seventy bio for $286 Million to Gain Full Control of CAR-T Therapy Abecma
• Bristol Myers Squibb has agreed to acquire 2seventy bio for $286 million, ending the profit-sharing arrangement for the BCMA-targeted CAR-T therapy Abecma used in multiple myeloma treatment.
• The acquisition represents an 88% premium over 2seventy's closing share price but marks the end of a challenging journey for the cell therapy company, which was spun out from bluebird bio in 2021.
• BMS's strategic move aligns with its cost-cutting initiatives aimed at saving $3.5 billion through 2027, as the company faces patent expirations for key products including Opdivo, Yervoy, and Eliquis.
Bluebird Bio Goes Private in $29M Deal with Carlyle and SK Capital Partners
• Gene therapy pioneer Bluebird Bio agrees to private acquisition by Carlyle and SK Capital Partners for $3 per share, with potential additional payments of up to $6.84 per share through CVR.
• Despite having three FDA-approved gene therapies, Bluebird Bio has faced significant financial challenges, including European market withdrawal and workforce reductions of 55% since 2022.
• The company's struggles intensified after failing to secure an expected $103M FDA priority review voucher for Lyfgenia in 2023, leading to critical cash flow issues.
Hemgenix Gene Therapy Shows Sustained Efficacy in Hemophilia B Patients After Four Years
• Data from the HOPE-B trial demonstrates that Hemgenix (etranacogene dezaparvovec-drlb) provides long-term bleed protection for adults with hemophilia B.
• Patients treated with Hemgenix experienced a 90% reduction in adjusted annualized bleeding rate (ABR) at year four compared to the lead-in period.
• 94% of patients remained free of continuous prophylaxis treatment at year four, indicating sustained independence from regular infusions.
• The gene therapy maintained a favorable safety profile, with most adverse events occurring within the first six months post-treatment.
Biopharma Industry Faces Continued Layoffs in 2025 Amid Strategic Realignments
• Biopharma companies continue to announce layoffs in 2025, driven by strategic realignments, pipeline reprioritizations, and challenging financial conditions.
• Several companies, including Intellia Therapeutics and IGM Biosciences, are cutting a significant portion of their workforce to focus on key programs and extend cash runways.
• These workforce reductions are often accompanied by discontinuation of certain drug development programs and a shift in strategic focus towards core assets.
• The layoffs reflect a broader trend in the industry as companies adapt to evolving market dynamics and prioritize efficiency and long-term sustainability.
Breakthroughs and Challenges in Gene Therapy: A 2024 Overview
2024 has seen significant advancements and challenges in the field of gene therapy, including promising results from Novartis' spinal muscular atrophy treatment, India's first successful gene therapy for haemophilia, and the development of a new prenatal gene therapy technique. However, the year also highlighted the risks associated with gene therapy, as seen in the death of a clinical trial participant and the development of blood cancer in children treated with Bluebird bio's Skysona.
J&J's Carvykti Shows Promise in Earlier Myeloma Treatment
• The CARTITUDE-4 study indicates that Carvykti (ciltacabtagene autoleucel) significantly improves progression-free survival in multiple myeloma patients with 1-3 prior lines of therapy.
• Carvykti, a BCMA-directed CAR-T therapy, may soon be used earlier in the treatment pathway, potentially leapfrogging Bristol-Myers Squibb's Abecma.
• The study compared Carvykti to standard three-drug regimens, showing a significant benefit that led to unblinding of the trial.
• Expansion of Carvykti's use is a key component of J&J's strategy in multiple myeloma, alongside other therapies like Darzalex and bispecific antibodies.
Corcept Therapeutics Submits New Drug Application for Relacorilant to Treat Cushing's Syndrome
• Corcept Therapeutics has submitted a New Drug Application to the FDA for relacorilant, a selective cortisol modulator designed to treat patients with endogenous hypercortisolism (Cushing's syndrome).
• The application is supported by positive results from multiple clinical trials, including the pivotal GRACE trial, Phase 3 GRADIENT study, and long-term extension studies, demonstrating improvements in various symptoms with an acceptable safety profile.
• Relacorilant showed no instances of serious adverse events common in current treatments, such as drug-induced adrenal insufficiency, hypokalemia, or QT prolongation, positioning it as a potential new standard of care.
Kymriah Receives EU Approval as First CAR-T Therapy for Follicular Lymphoma
• Novartis' Kymriah is the first CAR-T therapy approved in the EU for relapsed/refractory follicular lymphoma, offering a new option for patients after multiple treatments.
• The approval was based on the ELARA trial, demonstrating an 86% response rate and 69% complete response rate in patients treated with Kymriah.
• Kymriah provides durable treatment effects, with 87% of complete responders remaining in remission for at least nine months after initial response.
• This approval expands Kymriah's indications in the EU, adding to its existing approvals for diffuse large B-cell lymphoma and acute lymphoblastic leukemia.
FDA to Review Belantamab Mafodotin and Linvoseltamab Combinations for Multiple Myeloma
• The FDA has accepted a BLA for belantamab mafodotin combinations with bortezomib plus dexamethasone and pomalidomide plus dexamethasone for multiple myeloma treatment.
• Regeneron's linvoseltamab BLA resubmission has been accepted by the FDA, with a decision expected by July 10, 2025, for relapsed/refractory multiple myeloma.
• Clinical trials DREAMM-7 and DREAMM-8 support the belantamab mafodotin BLA, while LINKER-MM1 supports the linvoseltamab BLA, showcasing improved progression-free survival.
• Both belantamab mafodotin and linvoseltamab are under review by other regulatory authorities, potentially expanding treatment options for multiple myeloma patients.
GSK's Blenrep Demonstrates Significant Survival Benefit in Multiple Myeloma Trial, Paving Way for Potential Comeback
• GSK's Blenrep, combined with chemotherapy and a steroid, reduced the risk of death by 42% compared to a standard-of-care treatment in relapsed or refractory multiple myeloma.
• The DREAMM-7 trial data showed a projected median overall survival of 84 months for the Blenrep combination versus 51 months for the comparator arm.
• Blenrep, an antibody-drug conjugate targeting BCMA, is under regulatory review in multiple regions, potentially offering a new treatment option for myeloma patients.
• Despite previous market withdrawal due to a failed trial, Blenrep's new data suggests a possible paradigm shift in treating relapsed or refractory multiple myeloma.
© Copyright 2025. All Rights Reserved by MedPath