Industry Updates from the Field of Stem Cell Research and Regenerative Medicine in April 2022

Abstract

Latest developments in the field of stem cell research and regenerative medicine compiled from publicly available information and press releases from non academic institutions in April 2022.

Keywords::

• industry
• regenerative medicine
• stem cells

Business development

Collaboration agreement: Artiva & Merck

Artiva Biotherapeutics (CA, USA; www.artivabio.com), an oncology company whose mission is to deliver highly effective, off-the-shelf, allogeneic natural killer (NK) cell therapies to treat cancer, has entered into an agreement with Merck (Germany; www.merck.com), known as MSD outside the USA and Canada [1]. Under the agreement, Merck will evaluate the therapeutic potential of combinations of tri-specific NK-cell engager candidates with clinical grade AB-101 NK cells generated from Artiva’s AlloNK™ platform.

Artiva and Merck originally entered into a collaboration and exclusive license agreement in January 2021 for the discovery, development, manufacture and commercialization of chimeric antigen receptors (CAR)-NK cells that target certain solid tumor-associated antigens. The collaboration leverages Artiva’s off-the-shelf allogeneic NK cell manufacturing platform, along with its proprietary CAR-NK technology, and includes two CAR-NK programs with an option for a third, none of which are in Artiva’s current or planned pipeline. Artiva’s platform supports large-scale production and cryopreservation of off-the-shelf allogeneic NK and CAR-NK therapeutics.

Collaboration agreement: EdiGene & Peking University

EdiGene (China; www.edigene.com), known for its proprietary ex vivo genome-editing platforms for hematopoietic stem cells (HSC) and T cells, in vivo therapeutic platform based on RNA base editing, and high-throughput genome-editing screening to discover novel targeted therapies, has entered into a research collaboration with Peking University Cancer Hospital (China; www.bjcancer.org) to understand gene mutation frequency and clinicopathologic features of patients with advanced colorectal cancer in China, and to advance the study of gene mutations relating to varying response rates to specific targeted therapies [2]. The research result is expected to enable the development of precision medicine for patients prescreened by specific mutation patterns.

Partnership agreement: A*STAR & WuXi

WuXi Advanced Therapies WuXi Advanced Therapies (WuXi ATU; PA USA; https://advancedtherapies.com), a wholly owned subsidiary of WuXi AppTec (China; www.wuxiapptec.com) and A*STAR’s Bioprocessing Technology Institute (BTI; Singapore; www.a-star.edu.sg/bti), announced a new partnership to bring WuXi ATU’s proprietary Tetracycline-Enabled Self-Silencing Adenovirus (TESSA™) technology to the Asia-Pacific region [3]. This partnership aims to foster scientific innovation in cell and gene therapy and establish a joint professional development program to train and develop the next generation of scientists and engineers in current Good manufacturing practices (cGMP) manufacturing for the cell and gene therapy industry.

TESSA technology is a novel process for transfection-free, scalable manufacture of adeno-associated viruses (AAV) and represents a major advancement in AAV manufacturing by improving AAV yields and particle quality. TESSA technology produces significantly more AAV than plasmid-based manufacturing, generating enough material to address the growing demand in healthcare needs for patients suffering from cancer and other life-threatening diseases.

As part of this collaboration, WuXi ATU will supply the materials, equipment, funding and training required to further develop and commercialize TESSA technology in Singapore. BTI will contribute research expertise, facilities and access to its network of partners to support WuXi ATU in its research and development in cell and gene therapy in Singapore.

Partnership agreement: Chimeric & WuXi

Chimeric Therapeutics (Australia; https://chimerictherapeutics.com), a clinical-stage cell therapy company, has entered into a strategic manufacturing partnership with WuXi Advanced Therapies (WuXi ATU; PA USA; https://advancedtherapies.com), the advanced therapies business unit of WuXi (China; www.wuxiapptec.com) and a global contract testing and manufacturing organization [4].

Under the agreement, Chimeric will transfer certain manufacturing and analytical testing technologies to WuXi ATU, who will support process development, analytical development and cGMP manufacturing and testing activities for Chimeric’s CAR T cell programs. The new partnership will enable Chimeric to accelerate clinical manufacturing readiness for new CAR T assets and to scale CAR T manufacturing to support multiple, simultaneous, multicenter CAR T clinical trials in the future.

The initial focus under the agreement will be on Chimeric’s two autologous CAR T-cell therapies for solid tumors:

• CHM 2101 (CDH17 CAR T) – currently in late preclinical development for multiple gastrointestinal cancers, including colorectal cancer, gastric cancer, pancreatic cancer and gastrointestinal neuroendocrine tumors.

• CHM 1101 (CLTX CAR T) – currently being evaluated in a single-site Phase I clinical trial to treat patients with recurrent or progressive glioblastoma.

Partnership agreement: Porton & Sinorda

Porton Advanced Solutions (China; www.portonbio.com/English) and Guizhou Sinorda Biomedicine (China; www.sinorda.com/changeEn/index.html) have announced a long-term strategic partnership in cell and gene therapy R&D and platform development [5].

Porton Advanced offers an end-to-end gene and cell therapy contract manufacturing organization service platform covering plasmids, cell therapy, gene therapy, oncolytic virus, mRNA therapy and bacterial therapy. With broad experience in domestic and international new drug filing, Sinorda Biomedicine focuses on the R&D of innovative drugs for digestive tract diseases, tumor immunotherapy and oncolytic virus therapy and has several new drug projects in clinical stage

Under the agreement, Porton Advanced will provide services to accelerate the development, production and registration of breakthrough cell therapies currently being developed by Sinorda Biomedicine, including its T-cell immunotherapy product for colorectal cancer, Sentinel lymph node T cell (SLN-T) and other pipelines. Porton Advanced will also work with Sinorda Biomedicine to support its cell and gene therapeutic R&D and production.

The first collaborated project is SND002 from SLN-T program. SND002 has received approval for the initiation of Phase II clinical trials in Europe. Previously, the product was also designated as a ‘Breakthrough New Drug’ by the Chinese National Science and Technology Major Project. The launch of this critical first project allows the integration of resources and capabilities from both Porton Advanced and Sinorda and promises greater synergy to accelerate the clinical development and approval of Sinorda’s SLN-T program.

Partnership agreement: Quell & Cellistic

Quell Therapeutics (UK, https://quell-tx.com), a developer of engineered Treg cell therapies for serious medical conditions driven by the immune system, and Cellistic (Belgium; www.cellistic.com), the induced pluripotent stem cell (iPSC) focused cell therapy process development and manufacturing partner recently launched by Ncardia (Belgium; www.ncardia.com) to make large-scale allogeneic cell therapy production a reality, announce they have entered into a strategic collaboration for the co-development of an iPSC derived Treg cell therapy platform [6]. The goal of the partnership is to facilitate the future expansion of Quell’s autologous Treg cell therapy pipeline by adding off-the-shelf, allogeneic Treg cell therapy products, leveraging Cellistic’s expertise in differentiation and scale-up of iPSC processes for allogeneic cell therapy applications.

Achievements

Crown Bioscience & HUB Organoids

Crown Bioscience (CA, USA; www.crownbio.com), a JSR Life Sciences company (CA, USA; www.jsrlifesciences.com) and HUB Organoids (the Netherlands; https://huborganoids.nl/) have published a study on a bispecific antibody which prevents the onset of metastasis and slows the growth of primary tumors in experimental models of cancer [7,8]. This is the first study that has used organoids exclusively for cell-based compound screening and characterization, leading to a compound entering clinical trials.

Launching new products, services

Akron

Akron BioProducts (FL, USA; https://akronbiotech.com), a supplier of cGMP-compliant materials and technologies for cell and gene therapy manufacturing, has launched its Closed System Solutions™ product line, aimed at functionally closing critical unit operations in cell and gene therapy manufacturing workflows [9].

Cimeio

Versant Ventures (CA, USA; www.versantventures.com) has announced the debut of Cimeio Therapeutics (Switzerland; www.cimeio.com), a biotechnology company developing a novel approach to cell therapies [10]. Versant has made a US$50 million Series A commitment to Cimeio, which is the most recent start-up to emerge from the firm’s Ridgeline Discovery Engine in Basel, Switzerland.

Cimeio’s platform has the potential to transform the treatment of patients with rare genetic diseases, hematologic malignancies and autoimmune disorders. The company’s initial focus is on a novel approach to HSC transplants and adoptive cell therapy (ACT).

HSC transplants are the only curative treatments for certain debilitating and life-threatening diseases, but many patients are ineligible due to the intensive chemotherapy and radiation conditioning required. While targeted therapeutics have recently emerged as alternatives to harsh conditioning agents, these have fallen short due to the absence of sufficiently selective targets. Furthermore, there are few options for salvaging unsuccessful transplants or for dealing with residual or recurring disease.

Cimeio seeks to transform HSC transplant and ACT eligibility and outcomes with its cell-shielding technology and precisely paired immunotherapies. The company’s proprietary immunotherapies deplete diseased cells, while its cell-shielding technology protects healthy transplanted cells and allows them to engraft. Because the transplanted cells are shielded, the immunotherapy can continue to be safely administered post-transplant to boost engraftment or to treat minimal residual disease.

Lineage Cell Therapeutics

Lineage Cell Therapeutics (CA, USA; https://lineagecell.com), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, has announced a new cell therapy development program: photoreceptor neural cell (PNC) transplants for the treatment of vision loss due to photoreceptor dysfunction or damage [11]. Similar to the company’s recently announced pipeline expansion into auditory neurons for the treatment of hearing loss [12], Lineage has filed for intellectual property protection covering the composition and methods for generating PNCs. Based on recent in vivo data generated using the company’s PNCs, these cells may be capable of forming reconstructed retina with high survivability and neural connectivity to surrounding functional layers. Notably, Lineage has demonstrated feasibility which could support a large-scale method for producing both types of photoreceptors, known as rods and cones.

Ncardia

Ncardia (Belgium; www.ncardia.com) has launched a new business, Cellistic™ (Belgium; www.cellistic.com) to focus Ncardia’s iPSC expertise on the cell therapy development and manufacturing sector [13]. This strategy expands on Ncardia’s existing drug discovery solutions business and capitalizes on the company’s deep expertise in iPSC differentiation and expansion to focus dedicated resources and capabilities to address the growing need for iPSC cell therapy solutions, including the development of robust cell-specific manufacturing platforms.

Pluristyx & Accelerated Biosciences

Pluristyx (WA, YSA; www.pluristyx.com), an advanced therapy tools and services biotechnology company, and Accelerated Biosciences (CA, USA; www.acceleratedbio.com), a regenerative medicine innovator in the use of proprietary human trophoblast stem cells (hTSCs), have signed an agreement for Pluristyx to manufacture clinical grade hTSC banks under GMP [14]. Accelerated Biosciences will use and make these stem cell banks available to commercial partners for further manufacturing of advanced biologic and cellular therapies. hTSCs have unique legal, ethical and intellectual property advantages, including a high expansion capacity and a unique genetic identity not associated with any person or embryo, making them the ideal starting material for manufacturing the next generation of advanced therapies.

Clinical trials

Pluripotent stem cells

Cynata

Cynata Therapeutics (Australia; www.cynata.com), a clinical-stage biotechnology company specializing in cell therapeutics, has announced enrolment of initial patients in a clinical trial of CYP-006TK as a potential treatment for diabetic foot ulcers [15]. Subjects are now being followed as planned for the 4 week treatment period out to the conclusion of the study at 24 weeks.

The Phase I trial (protocol number CYP-DFU-P1-01) aims to recruit 30 adult patients with diabetic foot ulcers who will be randomly assigned to receive CYP-006TK or standard care of treatment. CYP-006TK is a novel polymer-coated silicon wound dressing seeded with Cymerus™ iPSC-derived mesenchymal stromal/stem cells to facilitate topical application to the wound. Cynata has exclusively licensed the dressing technology from leading manufacturer of innovative biomedical coatings, TekCyte (Australia; https://tekcyte.com).

Lineage Cell Therapeutics

Lineage Cell Therapeutics (CA, USA; https://lineagecell.com), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, has announced that Cancer Research UK (UK; www.cancerresearchuk.org) recently completed patient enrollment in the ongoing Phase I clinical trial of VAC2, an allogeneic cancer vaccine product candidate, for the treatment of non-small-cell lung cancer [16,17]. Under the terms of an existing agreement, Cancer Research UK will complete the ongoing clinical trial and Lineage has now assumed responsibility for further clinical development of the VAC2 product candidate and any future development opportunities derived from the VAC platform.

VAC2 is an allogeneic, or non patient specific ‘off-the-shelf’, cancer vaccine product candidate designed to stimulate patient immune responses to an antigen commonly expressed in cancerous cells but not in normal adult cells. VAC2, which is produced from a pluripotent cell technology using a directed differentiation method, is comprised of a population of nonproliferating mature dendritic cells. As the most potent type of antigen presenting cell in the body, dendritic cells instruct the body’s immune system to attack and eliminate harmful pathogens and unwanted cells. Because the tumor antigen is loaded exogenously into the dendritic cells prior to administration, VAC2 is a platform technology that could be modified to carry selected antigens, including patient-specific tumor neo-antigens or viral antigens. VAC2 is currently being tested in a Phase I study in adult patients with non-small-cell lung cancer in the advanced and adjuvant settings, conducted by Cancer Research UK.

Immune cells

Mustang Bio

Mustang Bio (MA, USA; www.mustangbio.com), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, has announced its plan to initiate a Phase I clinical trial combining CAR T cells and oncolytic virus for the treatment of recurrent glioblastoma, supported by interim data from two ongoing investigator-sponsored Phase I clinical trials evaluating two clinical candidates, MB-108 (C134 oncolytic virus) and MB‐101 (City of Hope’s IL13Rα2‐targeted CAR T-cell therapy) [18–20]. Preclinical data also presented support the safety of administering these two therapies sequentially in a regimen designated as MB-109.

Regulations, approvals, acquisition

Acquisitions & mergers

Cytovia

Isleworth Healthcare Acquisition Corporation (FL, USA; https://isleworthhealthcare.com), a special purpose acquisition company and Cytovia (FL, USA; www.cytoviatx.com), a biopharmaceutical company empowering NK cells to fight cancer through stem cell engineering and multispecific antibodies, have entered into a definitive business combination agreement [21]. Isleworth will be renamed Cytovia Therapeutics, and its common stock and warrants are expected to remain listed on NASDAQ under the ticker symbols INKC and INKCW, respectively.

The combined company will continue Cytovia’s operations and remain focused on developing and manufacturing complementary NK cell and NK engager antibody platforms. Cytovia is developing three types of iPSC-derived (or iNK) cells: unedited iNK cells, TALEN^® gene-edited iNK cells with improved function and persistence and TALEN gene-edited iNK cells with chimeric antigen receptors (CAR-iNKs) to improve tumor-specific targeting. The second complementary cornerstone technology is a quadrivalent multifunctional antibody platform designed to engage NK cells by targeting NKp46 using Cytovia’s proprietary Flex-NK™ technology.

The transaction would value the combined company at a pro forma equity value of US$602 million, assuming a US$10 per share price, no Isleworth stockholder redemptions and no additional financing obtained prior to the closing of the transaction. As a result of the transaction, the combined company would have up to US$227 million in gross proceeds from a combination of approximately US$207 million in cash held in Isleworth’s trust account (assuming no Isleworth stockholders exercise their redemption rights at closing) and $20 million from PIPE financing of new investors, and without considering the proceeds of any additional prospective financing.

Caladrius & Cend

Caladrius Biosciences (NJ, USA; www.caladrius.com), a clinical-stage biopharmaceutical company, and Cend Therapeutics (CA, USA; https://cendrx.com), a privately-held, clinical-stage biotechnology company focused on a novel approach to enable more effective treatments for solid tumor cancers, have entered into a definitive merger agreement under which Cend will merge with a wholly owned subsidiary of Caladrius in an all-stock approximate ‘merger of equals’ transaction unanimously approved by the Boards of Directors of each company [22]. Following closing, the combined company will be renamed Lisata Therapeutics and will trade on the Nasdaq under the ticker symbol ‘LSTA’. The merger is currently expected to close in the third quarter of 2022 subject to the approval of Caladrius and Cend stockholders as well as the satisfaction of certain other customary closing conditions and applicable approvals. Caladrius current product candidates include XOWNA^®, autologous CD34⁺ cells for a treatment of Coronary microvascular dysfunction.

FUJIFILM & Atara

FUJIFILM Corporation (Japan; www.fujifilm.com) has completed the acquisition of a dedicated cell therapy manufacturing facility from Atara Biotherapeutics (CA, USA; www.atarabio.com) for US$100 million [23]. The facility located in Thousand Oaks, CA, USA will be operated as part of FUJIFILM Diosynth Biotechnologies’ global network (Japan; https://fujifilmdiosynth.com), which is a subsidiary of FUJIFILM Corporation, and a world-leading contract manufacturing organization.

The 8400 m² site is readily expandable to add capacity and has the flexibility to produce both clinical and commercial cell therapies including allogeneic T-cell and CAR T immunotherapies. FUJIFILM Diosynth Biotechnologies will retain 134 employees in manufacturing and quality positions at the site.

As part of a long-term manufacturing and services agreement, FUJIFILM Diosynth Biotechnologies will support the production of Atara’s promising clinical and commercial-stage allogeneic cell therapies at the site, including tabelecleucel (tab-cel^®), currently in Phase III clinical development and under review to support registration in Europe, for the treatment of Epstein–Barr virus positive post-transplant lymphoproliferative disease.

Green light

Gamida Cell

Gamida Cell (Israel; www.gamida-cell.com), the leader in the development of nicotinamide (NAM)-enabled cell therapies for patients with hematologic and solid cancers and other serious diseases, today announced that the US FDA cleared its investigational new drug application and removed the clinical hold for a cryopreserved formulation of GDA-201 [24]. GDA-201 is an off-the-shelf cell therapy candidate for the treatment of patients with follicular and diffuse large B cell lymphomas. GDA-201 leverages Gamida Cell’s proprietary NAM technology platform to expand the number and functionality of NK cells to direct tumor cell killing properties and antibody-dependent cellular cytotoxicity. Gamida Cell expects to initiate a company-sponsored Phase I/II clinical study in patients with follicular and diffuse large B-cell lymphomas in 2022 [25].

Kite

Kite (CA, USA; www.kitepharma.com), a Gilead Company (CA, USA; www.gilead.com), has announced the FDA has approved Yescarta^® (axicabtagene ciloleucel) CAR T-cell therapy for adult patients with large B-cell lymphoma that is refractory to first line chemoimmunotherapy or that relapses within 12 months of first line chemoimmunotherapy [26]. Yescarta demonstrated a clinically meaningful and statistically significant improvement in event-free survival over the current standard of care that has been in place for decades [27]. The FDA approval of Yescarta CAR T-cell therapy for adult patients with large B-cell lymphoma that is refractory to first-line chemoimmunotherapy or that relapses within 12 months of first line chemoimmunotherapy is based on results from the ZUMA-7 study. ZUMA-7 is a randomized, open-label, global, multicenter, phase III study evaluating the safety and efficacy of Yescarta versus current standard of care on a long run.

In an unrelated press release, Kite announced that the FDA has also approved commercial production at the company’s new CAR T-cell therapy manufacturing facility in Frederick, MD, USA [28]. The site will produce Kite’s FDA approved CAR T-cell therapy used to treat blood cancer.

Precigen

Orecigen (MD, USA; www.precigen.com) a biopharmaceutical company specializing in the development of innovative gene and cell therapies to improve the lives of patients, has announced that the FDA has granted Fast Track designation for PRGN-3006 UltraCAR-T^® in patients with relapsed or refractory acute myeloid leukemia [29,30]. PRGN-3006 was previously granted FDA Orphan Drug designation.

PRGN-3006 UltraCAR-T is a multigenic autologous CAR-T cell treatment utilizing Precigen’s non viral sleeping beauty system to simultaneously express a CAR specifically targeting CD33, which is over expressed on acute myeloid leukemia blasts; membrane bound IL-15 for enhanced in vivo expansion and persistence; and a kill switch to conditionally eliminate CAR-T cells for an improved safety profile.

Precigen’s UltraCAR-T platform is designed to overcome limitations of currently available CAR-T therapies by utilizing an advanced overnight non-viral gene delivery manufacturing process at a medical center’s cGMP facility without the need for ex vivo expansion. Current CAR-T cell therapies are limited due to, inter alia, the prolonged interval between apheresis to product infusion and an exhausted phenotype of T cells resulting from lengthy ex vivo expansion. UltraCAR-T cells for the PRGN-3006 study are manufactured overnight using Precigen’s proprietary UltraPorator™ system.

Red light

US Stemology

US Stemology (WA, USA; https://usstemology.com) is being sued for deceptively marketing and selling stem cell therapies for a cure-all for dozens of serious medical conditions including the company including dozens of other serious medical conditions, including asthma, lupus, Parkinson’s, congestive heart failure, multiple sclerosis and COVID-19 [31]. The state of Washington claims that US Stemology’s Seattle Stem Cell Center violated the state’s consumer protection legislation by deceptively marketing stem cell therapies for severe conditions without scientific basis or concrete evidence. US Stemology charged 107 of its patients US $748,250. The patients were also billed as much as US $10,000 for being involved in the clinic’s supposed patient-funded research. The state is asking the court to order US Stemology and company owner to compensate its patients.

The company has claimed it was treating patients as part and parcel of clinical trials. However, these so-called trials did not follow generally accepted standards of scientific research. Typically, the organizers of legitimate clinical trials pay patients and do not charge them thousands of dollars for participating in an experiment.

The trial was also tarnished by a conflict of interest. Per FDA requirements, all clinical trials must be approved and monitored by an Institutional Review Board (IRB). Universities and large research institutes run most of these IRBs. The independence of the IRB members is critical. However, US Stemology’s clinical trial was supervised by the International Cell Surgical Society (ISCC; CA, USA; https://internationalcellsurgicalsociety.org) and two of the chief investigators involved in the US Stemology trials were serving as principal officers of the ICSS.

Capital market & finances

Axol

Axol Bioscience (UK; www.axolbio.com), a provider of iPSC-derived cells, media and characterization services for life science discovery, has raised GBP3.2 (circa US$4.2) million in the extension of its previous funding round, bringing the total raised to GBP7 (circa US$9.2) million [32]. The funds will be used to bring new human iPSC products and service solutions for cardiac, neuroscience and immune cell modeling to the drug discovery and screening markets, including Axol’s recently developed comprehensive in vitro proarrhythmia assay (CiPA)-validated human iPSC-derived cardiomyocytes. Funding will enable Axol to further optimize cell culture quality and iPSC manufacturing capabilities at the Roslin Institute (UK; www.ed.ac.uk/roslin), as well as support recruitment efforts to expand its commercial team. It will also facilitate the continued development of the Company’s digital platform.

Be Bio

Be Biopharma (MA, USA; www.be.bio), a pioneer in the discovery and development of Engineered B Cell Medicines (BeCM), has closed a US$130 million Series B financing, bringing the total investment in the Company to over US $180 million [33]. The proceeds will advance Be Bio’s proprietary autologous and allogeneic BeCM platforms across multiple therapeutic areas and progress pipeline candidates toward the clinic.

BioEclipse Therapeutics

BioEclipse Therapeutics (CA, USA; www.bioeclipse.com), a privately held, clinical-stage biopharmaceutical company with a proprietary platform for developing next-generation cancer immunotherapies, has been awarded a $7,999,689.00 grant by the California Institute for Regenerative Medicine (CIRM; CA, USA; www.cirm.ca.gov) to support its ongoing clinical study: a Phase I, open-label, dose-escalation study of CRX100 in patients with advanced solid tumors [34,35].

BioEclipse is performing a Phase I clinical study evaluating the safety and tolerability of CRX100, a first-in-class immunotherapy that pairs the power of an oncolytic virus with the tumor-locating ability of cytokine-induced killer cells. These immune cells naturally locate and destroy cancer cells and when combined with our oncolytic virus create a powerful cancer immunotherapy to treat patients who are resistant to standard of care treatments and have recurring disease.

bluebird

Following a review of its strategic priorities, bluebird bio (MA, USA; www.bluebirdbio.com) is initiating a comprehensive restructuring intended to deliver up to US$160 million in cost savings over the next 2 years [36].

bluebird intends to sharpen its focus on near-term catalysts, including anticipated FDA approvals for its gene therapies for beta-thalassemia and cerebral adrenoleukodystrophy in 2022, and the potential submission of a biologics license application for lovotibeglogene autotemcel (lovo-cel) gene therapy for sickle cell disease planned in the first quarter of 2023. The Company expects to maintain targeted research efforts focused on in vivo lentiviral vector gene therapy and will deprioritize direct investments in reduced toxicity conditioning and cryopreserved apheresis.

The initiative is expected to reduce the Company’s cash burn in 2022 to less than US $340 million, with a 35–40% reduction in operating costs anticipated by the year end 2022, which is expected to be reflected in bluebird’s operating budget for 2023. As part of the changes, bluebird plans to reduce its workforce by approximately 30%. The restructuring is expected to extend the Company’s cash runway into the first half of 2023.

Financial & competing interests disclosure

The author Dusko Ilic has received an honorarium from Future Science Group for the contribution of this work. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

No writing assistance was utilized in the production of this manuscript.