Industry Updates from the Field of Stem Cell Research and Regenerative Medicine in June 2020
2020; Future Medicine; Volume: 15; Issue: 10 Linguagem: Inglês
10.2217/rme-2020-0102
ISSN1746-076X
Autores Tópico(s)Science, Research, and Medicine
ResumoRegenerative MedicineVol. 15, No. 10 Industry NewsFree AccessIndustry updates from the field of stem cell research and regenerative medicine in June 2020Dusko Ilic & Mirjana LiovicDusko Ilic *Author for correspondence: E-mail Address: dusko.ilic@kcl.ac.ukhttps://orcid.org/0000-0003-1647-0026Department of Women & Children's Health, Stem Cell Laboratories, Guy's Assisted Conception Unit, Faculty of Life Sciences & Medicine, King's College London, London, UK & Mirjana LiovicMedical Center for Molecular Biology, Faculty of Medicine, University of Ljubljana, Ljubljana, SloveniaPublished Online:19 Aug 2020https://doi.org/10.2217/rme-2020-0102AboutSectionsPDF/EPUB ToolsAdd to favoritesDownload CitationsTrack CitationsPermissionsReprints ShareShare onFacebookTwitterLinkedInRedditEmail AbstractLatest developments in the field of stem cell research and regenerative medicine compiled from publicly available information and press releases from nonacademic institutions in June 2020.Keywords: industryregenerative medicinestem cellsBusiness developmentCollaboration agreement: Magenta & Be The MatchMagenta Therapeutics (MA, USA; www.magentatx.com) and the National Marrow Donor Program® (NMDP)/Be The Match® (MN, USA; https://bethematch.org), have announced a clinical collaboration agreement to evaluate the potential utility of MGTA-145, Magenta's investigational first-line stem cell mobilization program, for mobilizing and collecting hematopoietic stem cells (HSCs) from donors in a single day and then using them for allogeneic transplants in patients [1]. Under the collaboration, Magenta and NMDP/Be The Match will run a Phase II clinical trial of MGTA-145 to mobilize and collect HSC from donors which will then be transplanted into patients with blood cancers in need of a stem cell transplant. The number of stem cells mobilized, engraftment function and benefit to disease will be measured. Magenta will retain all commercial rights to MGTA-145. MGTA-145, a CXCR2 agonist, works in combination with plerixafor, a CXCR4 antagonist, to harness the physiological mechanism of stem cell mobilization into peripheral blood. MGTA-145 achieved all of the safety and activity end points in the recently completed Phase I trials in over 100 volunteers. Results showed that MGTA-145, in combination with plerixafor, enabled safe, same-day dosing, mobilization and collection of superior functional HSC for transplant compared with the current standard of care. The NMDP/Be The Match is the leading stem cell transplant organization in the USA and facilitates more than 6500 stem cell transplants per year, through its contracted global network of 187 transplant centers. The NMDP/Be The Match is an investor in Magenta.Collaboration agreement: Magenta & BeamMagenta Therapeutics (MA, USA; www.magentatx.com) and Beam Therapeutics (MA, USA; www.beamtx.com) have announced a nonexclusive research and clinical collaboration agreement to evaluate the potential utility of MGTA-117, Magenta's novel targeted antibody–drug conjugate (ADC) for conditioning of patients with sickle cell disease (SCD) and beta-thalassemia receiving Beam's base editing therapies [2].Beam is pursuing two differentiated base editing approaches to treat hemoglobinopathies: its hereditary persistence of fetal hemoglobin program to precisely and robustly elevate fetal hemoglobin, which could be used in treatments for both SCD and beta-thalassemia, as well as a novel approach to directly correct the sickle causing point mutation. Beam is using base editors to directly convert the SCD-causing point mutation (E6V) into an asymptomatic, naturally occurring variant (E6A), also known as Hb G-Makassar. This hemoglobin variant has been observed in individuals who do not have polymerization (sickling) and are otherwise asymptomatic. Beam demonstrated that its base editor variants can reach up to 70% direct correction of the sickle hemoglobin (HbS) in SCD patient-derived fibroblasts.Conditioning is a critical component necessary to prepare a patient's body to receive the edited cells, which carry the corrected gene and must engraft in the patient's bone marrow in order to be effective. Today's conditioning regimens rely on nonspecific chemotherapy or radiation, which are associated with significant toxicities. MGTA-117 precisely targets only hematopoietic stem and progenitor cells, sparing immune cells, and has shown high selectivity, potent efficacy, wide safety margins and broad tolerability in nonhuman primate models. MGTA-117 may be capable of clearing space in bone marrow to support long-term engraftment and rapid recovery in patients. Beam has demonstrated the ability to edit individual DNA bases in HSC at high efficiency and with little impact on the viability of edited cells relative to unedited cells using its novel base editing technology. Combining MGTA-117 with Beam's hereditary persistence of fetal hemoglobin and Makassar base editors could meaningfully advance the treatment of patients with SCD or beta-thalassemia.Collaboration agreement: Regeneron & IntelliaRegeneron Pharmaceuticals (NY, USA; www.regeneron.com) and Intellia Therapeutics (MA, USA; www.intelliatx.com) announced an expansion of their existing collaboration to provide Regeneron with rights to develop products for additional in vivo CRISPR/Cas9-based therapeutic targets and for the companies to jointly develop potential products for the treatment of hemophilia A and B [3]. Regeneron also receives nonexclusive rights to independently develop and commercialize ex vivo gene edited products. Intellia will receive an upfront payment of US$70 million, and Regeneron will make an additional equity investment in Intellia of US$30 million at US$32.42 per share. Regeneron and Intellia have worked together to make significant advances with Intellia's CRISPR/Cas9 platform to enable the targeted insertion of therapeutic proteins and antibodies. This collaboration expansion allows the companies to leverage more fully their jointly developed targeted transgene insertion capabilities and potentially accelerate efforts to discover and develop new therapeutics, including products for hemophilia A and B. In preclinical studies, the companies demonstrated the first CRISPR/Cas9-mediated targeted transgene insertion in the liver of nonhuman primates, which generated normal or higher levels of circulating human Factor IX. Factor IX is a blood-clotting protein that is missing or defective in hemophilia B patients.Licensing agreement: Minerva & MSKCCMinerva Biotechnologies (MA, USA; www.minerva.bio.com) has licensed from Memorial Sloan Kettering Cancer Center (NY, USA; www.mskcc.org) '1XX' technology for use with Minerva's proprietary anti-MUC1 antibodies to increase CAR T cell persistence in patients. CAR T cell persistence, which is the amount of time that infused CAR T cells have the potential to kill tumor cells, is a recognized problem in the revolutionary field of cancer immunotherapy [4]. Since over-activation drives CAR T cell exhaustion, calibrating their activation potential through 1XX mutations in their signaling domain staves off expression of exhaustion molecules that turn CAR T cells off. With this agreement, Minerva will gain nonexclusive access to two of MSK's innovative CAR T technologies, including the 1XX CAR T cell signaling construct, whose activation potential more closely resembles that of naturally occurring T cells. Minerva expects its next-generation anti-MUC1 CAR T cell therapies to have enhanced therapeutic profiles due to their extended persistence.Manufacturing, marketing & distribution agreement: AgeX & PluristyxAgeX Therapeutics (CA, USA; www.agexinc.com), a biotechnology company developing therapeutics for human aging and regeneration, and Pluristyx (WA, USA; www.pluristyx.com), an advanced therapy tools and services company, have entered into a Manufacturing, Marketing and Distribution Agreement through which Pluristyx will undertake these activities on behalf of AgeX with respect to AgeX's research- and clinical-grade ESI brand human embryonic stem cells [5]. The agreement is a key step in AgeX's licensing and collaboration strategy to facilitate industry and academic access to its human embryonic stem cell lines, its PureStem® cell derivation and manufacturing platform, and its UniverCyte™ immunotolerance technology in order to generate near- and long-term revenues.Partnership agreement: Cell-Easy & University Hospital ToulouseCell-Easy (France; https://cell-easy.com/), a start-up specialized in stem cell therapy, signed an agreement with the University Hospital Centre in Toulouse (France; www.chu-toulouse.fr) as well as secured the authorization to open a pharmaceutical manufacturing plant dedicated to the production of stem cells - a first in France [6]. This follows the announcement made last December of raising 1M euros capital investment. The start-up will be producing mesenchymal stromal/stem cells (MSCs) sourced from qualified fat tissue in accordance with good manufacturing practices. A first clinical trial is on its way to fight against Alzheimer disease.AchievementsNantKwestNantKwest (CA, USA; www.nantkwest.com), a clinical-stage, natural killer (NK) cell-based therapeutics company, has announced the publication of two peer-reviewed manuscripts [7–9]. The studies, conducted in collaboration with the National Cancer Institute pursuant to a Cooperative Research and Development Agreement, support the mechanism and functionality of NantKwest's clinical-stage engineered NK cell lines, haNK and first-in-class PD-L1 t-haNK, as effecting anti-tumor activity in treatment-refractory cancer types even in the hypoxemic setting of the solid tumor microenvironment.Clinical trialsHematopoietic stem cellsbluebirdbluebird bio (MA, USA; www.bluebirdbio.com) announced new data from its ongoing Phase I/II HGB-206 study of investigational LentiGlobin™ gene therapy for adult and adolescent patients with SCD show a near-complete reduction of serious vaso-occlusive crises and acute chest syndrome (www.clinicaltrials.gov; ID: NCT02140554) [10]. SCD is a serious, progressive and debilitating genetic disease caused by a mutation in the β-globin gene that leads to the production of abnormal HbS. HbS causes red blood cells to become sickled and fragile, resulting in chronic hemolytic anemia, vasculopathy and unpredictable, life-threatening episodes of painful vaso-occlusive crises. If patients survive the acute complications, vasculopathy and end-organ damage, resulting complications can lead to pulmonary hypertension, renal failure and early death; in the US the median age of death for someone with SCD is 43–46 years. LentiGlobin for SCD was designed to add ex vivo functional copies of a modified form of the β-globin gene (βA-T87Q-globin gene) into a patient's own HSC, which were then transplanted back into patients that were subjected to myeloablative conditioning with busulfan. Once patients have the βA-T87Q-globin gene, their red blood cells can produce anti-sickling hemoglobin, HbAT87Q, that decreases the proportion of HbS, with the goal of reducing sickled red blood cells, hemolysis and other complications. As of 3 March 2020, a total of 37 patients have been treated with LentiGlobin for SCD to-date in the HGB-205 (n = 3) and HGB-206 (n = 34) clinical studies.In 16 patients with six or more months of follow-up, median levels of gene therapy-derived anti-sickling hemoglobin, HbAT87Q, were maintained with HbAT87Q contributing at least 40% of total hemoglobin. All patients in this cohort were able to stop regular blood transfusions and remain off transfusions at 3 months post-treatment.The company also announced that new data from ongoing Phase III studies (www.clinicaltrials.gov; ID: NCT02906202) of betibeglogene autotemcel (beti-cel; formerly LentiGlobin for β-thalassemia gene therapy) show pediatric, adolescent and adult patients with a range of genotypes of transfusion-dependent β-thalassemia (TDT) achieve and maintain transfusion independence with hemoglobin (Hb) levels that are near-normal (≥10.5 g/dl) [11]. A total of 60 pediatric, adolescent and adult patients across genotypes of TDT have been treated with beti-cel in the Phase I/II Northstar (HGB-204) and HGB-205 studies, and the Phase III Northstar-2 (HGB-207) and Northstar-3 (HGB-212) studies as of 3 March 2020. In studies of beti-cel, transfusion independence is defined as no longer needing red blood cell transfusions for at least 12 months while maintaining a weighted average Hb of at least 9 g/dl. TDT is a severe genetic disease caused by mutations in the β-globin gene that results in significantly reduced or absent adult hemoglobin (HbA). In order to survive, people with TDT maintain Hb levels through lifelong, chronic blood transfusions. These transfusions carry the risk of progressive multi-organ damage due to unavoidable iron overload. Beti-cel is a one-time gene therapy designed to address the underlying genetic cause of TDT by adding functional copies of a modified form of the β-globin gene (βT87Q-globin gene) into a patient's own HSCs. This means there is no need for donor HSCs from another person, as is required for allogeneic HSC transplantation (allo-HSCT). Once a patient has the βT87Q-globin gene, they have the potential to produce HbAT87Q, which is gene therapy-derived Hb, at levels that eliminate or significantly reduce the need for transfusions. As of 3 March 2020, all 23 patients in HGB-207 were treated and have been followed for a median of 19.4 months. These patients ranged in age from four to 34 years, including eight pediatric ( 12 years of age) patients. Only 19 patients were evaluable for transfusion independence; four additional patients do not yet have sufficient follow-up to be assessed for transfusion independence.Immune system cellsAvalonAvalon GloboCare (NJ, USA; www.avalonglobocare.com), a clinical-stage global developer of cell-based technologies and therapeutics, has completed Phase I first-in-human clinical study of its leading Chimeric Antigen Receptor (CAR) T-cell therapy candidate, AVA-001 (www.clinicaltrials.gov; ID: NCT03952923) [12]. Nine out of ten patients with relapsed/refractory B-cell acute lymphoblastic leukemia have achieved complete remission ([CR] rate of 90%) within one month after the initiation of AVA-001 CAR T-cell therapy treatment. The treatment with AVA-001 was generally well tolerated with minimal toxicities and adverse side effects. No neurotoxicity or greater than grade-1 cytokine release syndrome was observed in this cohort of patients treated with AVA-001. All patients who achieved CR successfully proceeded allogeneic bone marrow transplant with curative intent. AVA-001 is a third generation CAR T-cell therapy which involves the 4-1BB (or CD28) co-stimulation signaling pathway. Accessary laboratory testing that accompanied this pilot clinical study has demonstrated evidence of enhancement in CAR T cell persistence and protection against CAR T cell exhaustion.OthersAiVivaAiViva Biopharma (CA, USA; https://aiviva.com) has announced completion of its first Phase I/IIa study evaluating AIV001 in dermal scarring (www.clinicaltrials.gov; ID: NCT03639883) [13]. A total of 16 subjects who were scheduled to undergo abdominoplasty were enrolled and completed in one of the four double-blind dose escalation or open label cohorts. Incisional wounds on the abdomen received a focal intradermal treatment of AIV001 or vehicle on Days 1 and 21, or not treated as a control, and the wound healing was monitored for 49 days post wounding. Our results showed that AIV001 was well tolerated by intradermal treatment as compared with the vehicle. No serious local or systemic side effects were observed in the subjects at any of the doses administered. Transient local skin reactions were observed at high doses and resolved by the end of the study. A pharmacokinetics analysis showed prolonged drug residence around the treated incisional wound with very low systemic exposure detected. Histological evaluation of the incisional wounds showed reduction in fibrosis within 7–10 days post wounding following AIV001 treatment. These data were consistent with nonclinical minipig studies that show AIV001 delayed granulation and reduced fibrosis formation. However, there was no noticeable difference in the healing among untreated, vehicle treated and AIV001 treated wounds by clinical and photographic examination. AIV-001 is a novel formulation of a multi-kinase inhibitor combined with AiViva's proprietary delivery technology, designed for prolonged drug release via intradermal treatment.Regulations, approvals, acquisitions…AcquisitionsCentury & EmpiricaCentury Therapeutics (PA, USA; www.centurytx.com) has announced its acquisition of Empirica Therapeutics (ON, Canada; https://empiricatx.com) to leverage its iPSC-derived allogeneic cell therapies against glioblastoma (GBM) [14]. Century Therapeutics is harnessing the power of stem cells to develop curative cell therapy products for cancer that overcome the limitations of first-generation cell therapies. Their genetically engineered, universal iPSC-derived immune effector cell products (iNK, iT) are designed to specifically target hematologic and solid tumor cancers. Century was launched in 2019 by founding investor Versant Ventures (CA, USA: www.versantventures.com) in partnership with Fujifilm (Japan; www.fujifilm.com) and Leaps by Bayer (Germany; https://leaps.bayer.com). Empirica's science is based on a powerful integrative multi-omics platform, combined with its unique patient-derived, therapy-adapted models of recurrent GBM, that has led to the discovery and validation of novel brain tumor targets. Empirica's cutting edge preclinical models of recurrent GBM have demonstrated the potential of CAR-T cell therapy in GBM.Green lightBaylxBaylx (CA, USA; www.baylx.com), a clinical-stage biopharmaceutical company dedicated to developing novel stem cell therapeutics and products to address unmet medical needs, has announced that the US FDA has cleared Baylx's Investigational New Drug (IND) application for BX-U001, a fresh, nonfrozen human umbilical cord tissue mesenchymal stem cells product in patients with rheumatoid arthritis (RA) [15]. Prior to the clearance of this IND in the US, Baylx's strategic partner Beijing Beilai Biosciences (China) has received the IND approval for clinical testing of the same stem cell product in RA from the National Medical Products Administration (NMPA; http://english.nmpa.gov.cn), a China agency equivalent to the FDA. This was also the first IND approval in China on stem cells for the treatment of RA or autoimmune disease.CelltexCelltex (TX, USA; www.celltexbank.com) has received approval from the FDA to proceed with the Company's IND application (IND 22055) to investigate the 'prophylactic efficacy of autologous adipose tissue-derived mesenchymal stem cells' (AdMSCs) against coronavirus disease 2019 (COVID-19) [16]. This is a Phase II multi-center, randomized, double-blind, placebo-controlled study that will evaluate the safety and prophylactic efficacy of AdMSCs against COVID-19. Celltex will enroll 200 patients in the study (www.clinicaltrials.gov; ID: NCT04428801) who have never been infected by COVID-19. One hundred patients will receive intravenous infusions of autologous AdMSCs while 100 will receive placebo treatments. The primary objective of the study is to evaluate overall safety of AdMSCs, and the secondary objective is to demonstrate the efficacy of AdMSCs as a prophylactic medicine for those at a high risk of contracting the virus.ElixirgenElixirgen Therapeutics (MD, USA; https://elixirgentherapeutics.com) biotechnology company focused on the discovery, development and commercialization of therapies for genetic diseases and vaccines, received confirmation from the FDA that its IND application for its lead candidate, EXG4217, was approved [17]. EXG34217 is an autologous cell therapy for telomere biology disorders with bone marrow failure [18,19]. The FDA's approval allows Elixirgen Therapeutics to proceed with its planned Phase I/II, open label, single center clinical trial to assess the safety and tolerability of EXG34217 (https://clinicaltrials.gov; ID: NCT04211714). First, the patients' CD34+ HSCs are mobilized, and then collected using standard apheresis. Next, the CD34+ cells are treated with a human ZSCAN4 protein-expressing vector in a closed, sterile tubing system outside the patient's body for 24 h. Finally, those cells are reinfused back to the patient. ZSCAN4 is expressed specifically at the 2-cell stage embryo, where it binds sequence-dependent microsatellite DNA and protects fragile genomic regions from DNA damage.KiteKite (CA, USA; www.kitepharma.com), a Gilead Company (CA, USA; www.gilead.com), has received approval to implement a variation to the Yescarta (axicabtagene ciloleucel) Marketing Authorization from the EMA for end-to-end manufacturing [20]. With this approval, Kite's European manufacturing facility, designed and dedicated to the manufacture of individualized cell therapies, is now fully operational. Yescarta was the first CAR T cell therapy to be approved by the FDA for the treatment of adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, primary mediastinal large B-cell lymphoma, and high grade B-cell lymphoma and DLBCL arising from follicular lymphoma. Yescarta is also approved in the EU as a treatment for adult patients with relapsed or refractory DLBCL and primary mediastinal large B-cell lymphoma, after two or more lines of systemic therapy.Because of the risk of cytokine release syndrome (CRS) and neurologic toxicities, Yescarta is available only through a restricted program called the Yescarta REMS which requires that: healthcare facilities that dispense and administer Yescarta must be enrolled and comply with the REMS requirements and must have on-site, immediate access to a minimum of two doses of tocilizumab for each patient for infusion within 2 h after Yescarta infusion, if needed for treatment of CRS. Certified healthcare facilities must ensure that healthcare providers who prescribe, dispense or administer Yescarta are trained about the management of CRS and neurologic toxicities (further information is available at www.yescartarems.com).Rocket pharmaceuticalsRocket Pharmaceuticals (NY, USA; www.rocketpharma.com), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, has received clearance from the FDA for the Company's IND application for RP-L401 [21]. RP-L401 is the Company's ex vivo lentiviral vector-based gene therapy using autologous HSCs for the treatment of infantile malignant osteopetrosis, a rare, severe monogenic bone resorption disorder characterized by skeletal deformities, neurologic abnormalities and bone marrow failure. RP-L401 was in-licensed from Lund University (Sweden; http://www.lunduniversity.lu.se).Red lightAgeX TherapeuticsAgeX Therapeutics (CA, USA; www.agexinc.com), a biotechnology company developing therapeutics for human aging and regeneration, has received on 1 June 2020 a Deficiency Letter from the staff of the New York Stock Exchange American (NY, USA; www.nyse.com) indicating that AgeX does not meet certain of the NYSE's continued listing standards as set forth in Section 1003(a)(i) of the NYSE Company Guide in that AgeX has stockholders equity of less than US$2,000,000 and has incurred losses from continuing operations and/or net losses during its two most recent fiscal years [22]. Pursuant to Section 1009 of the NYSE Company Guide and as provided in the Deficiency Letter AgeX intends to provide the NYSE staff with a plan advising the NYSE staff of action AgeX has taken and will take that would bring AgeX into compliance with the NYSE's continued listing standards by 1 December 2021. There is no assurance that the NYSE staff will accept AgeX's Plan. If the NYSE staff accepts AgeX's plan, the NYSE staff will review AgeX's compliance with the plan on a quarterly basis and if AgeX does not show progress consistent with the plan or is not in compliance with the NYSE's continued listing standards by 1 December 2021, the NYSE will commence delisting procedures. If the NYSE staff does not accept AgeX's plan, the NYSE staff will promptly initiate delisting proceedings. AgeX intends to make arrangements to have its common stock quoted on the OTC Bulletin Board if its common stock is delisted from the NYSE American.Businesses selling unproven & unlicensed "stem cell treatments" for COVID-19Leigh Turner from Center for Bioethics, University of Minnesota, has reported to the FDA numerous businesses selling unlicensed and unproven stem cell interventions [23]. On a pro bono basis, he filed an expert opinion report and rebuttal report supporting plaintiffs in a lawsuit filed against a business marketing purported stem cell intervention. He was also deposed in this case and was examined and cross-examined in a court hearing. His commentary about businesses that are taking advantage of widespread fears by marketing purported stem cell treatments for COVID-19 with misleading claims have been also published.Capital market & financesMagentaMagenta Therapeutics (MA, USA; www.magentatx.com), a clinical-stage biotechnology company developing novel medicines to bring the curative power of stem cell transplant to more patients, has closed its previously announced underwritten public offering of 8,625,000 shares of its common stock, including the exercise in full by the underwriters of their option to purchase an additional 1,125,000 shares, at a price to the public of US$8.00 per share [24]. The total gross proceeds from the offering, before deducting underwriting discounts and commissions and estimated offering expenses, are expected to be US$69.0 million. Magenta intends to use the net proceeds from this offering to advance its clinical and earlier stage programs and for research and development, working capital and general corporate purposes.NantKwestNantKwest (CA, USA; www.nantkwest.com), a clinical-stage, NK cell-based therapeutics company, has closed its previously announced underwritten public offering of an aggregate of 8,521,500 shares of its common stock (4,811,500 shares at a price to the public of US$9.50 per share and 3,710,000 shares at a price of US$12.12 per share to NantKwest's chairman, chief executive officer and principal stockholder, Dr Patrick Soon-Shiong), which includes 1,111,500 shares sold upon full exercise of the underwriters' option to purchase additional shares at a public offering price of US$9.50 per share, less the underwriting discounts and commissions [25]. All of the shares were offered by NantKwest. Including the option exercise, the aggregate gross proceeds of the offering were approximately US$90.7 million, before deducting the underwriting discounts and commissions and other offering expenses.PoseidaPoseida Therapeutics (CA, USA; https://poseida.com), a clinical-stage biopharmaceutical company dedicated to utilizing proprietary gene engineering platform technologies to create next generation cell and gene therapeutics with the capacity to cure, has closed a Series D financing round, raising US$110 million [26]. Poseida's portfolio includes allogeneic and autologous CAR-T product candidates in both hematological and solid tumor oncology indications, as well as liver-directed gene therapy programs in orphan genetic diseases. They have discovered and are developing a broad portfolio of product candidates in a variety of indications based on our core proprietary platforms, including their nonviral piggyBac DNA Modification System, Cas-CLOVER site-specific gene editing system and nanoparticle- and AAV-based gene delivery technologies. Their core platform technologies have utility, either alone or in combination, across many cell and gene therapeutic modalities and enable them to engineer their wholly owned portfolio of product candidates that are designed to overcome the primary limitations of current generation cell and gene therapeutics.Financial & competing interests disclosureThe author Dusko Ilic has received an honorarium from Future Science Group for the contribution of this work. The author has 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.References1. Magenta Therapeutics. Magenta Therapeutics announces collaboration with the National Marrow Donor Program/Be The Match to advance development of MGTA-145 for first-line mobilization of stem cells from healthy donors and subsequent patient transplant (2020). https://investor.magentatx.com/node/7871/pdfGoogle Scholar2. Magenta Therapeutics. Magenta Therapeutics and Beam Therapeutics announce collaboration to evaluate targeted antibody-drug conjugate (ADC) MGTA-117 as conditioning regimen for base editing therapies (2020). https://investor.magentatx.com/node/7881/pdfGoogle Scholar3. Regeneron Pharmaceuticals. 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The author has 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.PDF download
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