Pilot Study of Emapalumab With Post-Transplant Cyclophosphamide as Graft-Versus-Host Disease Prophylaxis for Reduced-Intensity Allogeneic Hematopoietic Cell Transplantation

This phase I trial tests the safety, side effects and effectiveness of emapalumab with post-transplant cyclophosphamide, tacrolimus, and mycophenolate mofetil in preventing graft-versus-host disease (GVHD) in patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) after reduced-intensity donor (allogeneic) hematopoietic cell transplant (HCT). Giving chemotherapy, such as fludarabine, melphalan, or busulfan, before a donor [peripheral blood stem cell] transplant helps kill cancer cells in the body and helps make room in the patient's bone marrow for new blood-forming cells (stem cells) to grow. When healthy stem cells for a donor are infused into a patient (allogeneic HCT), they may help the patient's bone marrow make more healthy cells and platelets. Allogeneic HCT is an established treatment, however, GVHD continues to be a major problem of allogeneic HCT that can complicate therapy. GVHD is a disease caused when cells from a donated stem cell graft attack the normal tissue of the transplant patient. Emapalumab binds to an immune system protein called interferon gamma. This may help lower the body's immune response and reduce inflammation. Cyclophosphamide is in a class of medications called alkylating agents. It works by damaging the cell's deoxyribonucleic acid and may kill cancer cells. It may also lower the body's immune response. Tacrolimus is a drug used to help reduce the risk of rejection by the body of organ and bone marrow transplants. Mycophenolate mofetil is a drug used to prevent GVHD after organ transplants. It is also being studied in the prevention of GVHD after stem cell transplants for cancer, and in the treatment of some autoimmune disorders. Mycophenolate mofetil is a type of immunosuppressive agent. Giving emapalumab with post-transplant cyclophosphamide, tacrolimus and mycophenolate mofetil may be safe, tolerable and/or effective in preventing GVHD in patients with AML or MDS after a reduced-intensity allogeneic HCT.

Start Date25 May 2026

Sponsor / Collaborator

City of Hope National Medical Center

[+1]

NCT06954987

/ Not yet recruitingPhase 2IIT

A Two Cohort, Randomized, Double-Blind, Placebo-Controlled, Phase II Multi-Center Signal-Finding Study of Venetoclax Combined With Reduced-Intensity Conditioning Followed by Allogeneic Hematopoietic Cell Transplantation Then Venetoclax Maintenance in Adult Patients With Acute Myeloid Leukemia in First Complete Remission: A MyeloMATCH Sub-Study

This phase II MyeloMATCH treatment trial compares the effect of adding venetoclax or placebo to reduced intensity conditioning chemotherapy with fludarabine and busulfan or melphalan, with or without total body irradiation, followed by hematopoietic stem cell transplant and either venetoclax or placebo maintenance therapy after transplant, for the treatment of patients with acute myeloid leukemia (AML). Venetoclax is in a class of medications called B-cell lymphoma-2 (BCL-2) inhibitors. It may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. Giving chemotherapy and total body irradiation before a donor stem cell transplant helps kill cancer cells in the body and helps make room in the patient's bone marrow for new blood-forming cells (stem cells) to grow. When the healthy stem cells from a donor are infused into a patient, they may help the patient's bone marrow make more healthy cells and platelets and may help destroy any remaining cancer cells. Adding venetoclax to conditioning therapy before, and giving it as maintenance therapy after, a hematopoietic stem cell transplant may be a more effective treatment option than the usual approach in patients with AML.

Start Date18 Mar 2026

Sponsor / Collaborator

National Cancer Institute

NCT07228624

/ RecruitingPhase 2IIT

Expanding Use of Peri-Hematopoietic Cell Transplantation Ruxolitinib to Patients of Advanced Age With Myelofibrosis and Myelodysplastic Syndrome/Myeloproliferative Neoplasm Overlap Syndromes

This phase II trial tests the effect of adding ruxolitinib to standard graft versus host disease (GVHD) prevention in treating older patients with myelofibrosis (MF) or myelodysplastic syndrome/myeloproliferative neoplasm (MDS/MPN) overlap syndromes before, during, and after a donor (allogeneic) hematopoietic cell transplant (HCT). Allogeneic HCT is a procedure in which a person receives blood-forming stem cells (cells from which all blood cells develop) from a genetically similar, but not identical donor. Giving chemotherapy, such as cytoxan and busulfan or fludarabine and melphalan, before a donor transplant helps kill cancer cells in the body and helps make room in the patient's bone marrow for new blood-forming cells (stem cells) to grow. However, sometimes the transplanted cells from a donor can attack the body's normal cells (called GVHD). Giving standard prevention (prophylaxis) therapies, such as tacrolimus and methotrexate, after the transplant may stop this from happening. Methotrexate, a type of antifolate, is in a class of medications called antimetabolites. Methotrexate stops cells from using folic acid to make deoxyribonucleic acid and may kill cancer cells. Tacrolimus is used to help reduce the risk of rejection by the body of organ and bone marrow transplants. Ruxolitinib, a type of Janus-associated kinase (JAK) inhibitor, blocks a protein called JAK, which may help keep abnormal blood cells or cancer cells from growing. It may also lower the body's immune response and prevent the development of GVHD. Giving ruxolitinib before, during and after allogeneic HCT in addition to standard GVHD prophylaxis may be safe, tolerable and effective in preventing GVHD and improving outcomes in older patients with MF or MDS/MPN overlap syndrome.

Start Date21 Feb 2026

Sponsor / Collaborator

Fred Hutchinson Cancer Research Center

[+1]

100 Clinical Results associated with Busulfan

100 Translational Medicine associated with Busulfan

100 Patents (Medical) associated with Busulfan

7,247

Literatures (Medical) associated with Busulfan

01 Mar 2026·REPRODUCTIVE TOXICOLOGY

A mouse model of busulfan-induced ovarian toxicity and evidence for attenuation with mTOR inhibition

Article

Author: Inatomi, Ayako ; Nakamura, Akiko ; Tanaka, Yuji ; Takahashi, Akimasa ; Yamanaka, Hiroyuki ; Tsuji, Shunichiro ; Hanada, Tetsuro ; Deguchi, Mari ; Amano, Tsukuru ; Yoneoka, Yutaka

Busulfan, an alkylating agent used in hematopoietic stem cell transplantation conditioning, is associated with a high risk of ovarian toxicity. We established a time-resolved in vivo model of busulfan-induced ovarian injury and tested whether sapanisertib, an mTOR inhibitor, mitigates damage. Mice received busulfan (30 mg/kg, intraperitoneally) or saline; ovaries were assessed at 12, 24, and 72 h and 7 days by hematoxylin and eosin histology, follicle counting, TUNEL, and Ki-67 immunohistochemistry. A separate cohort received daily oral sapanisertib (0.3 mg/kg) from 7 days before busulfan through euthanasia; proliferation was assessed at 24 h, and follicle counts at 7 days. In the busulfan-only group, Ki-67 staining showed an early proliferative surge at 24 h in growing follicles, including primary follicles, whereas loss of primordial follicles and an increased primary-to-primordial follicle ratio were evident by 7 days. Primordial follicles showed no TUNEL-positive cells through 72 h, whereas growing follicles were frequently TUNEL-positive. Relative to busulfan alone, the mTOR inhibitor preserved primordial follicles, shifted the primary-to-primordial ratio toward control values, and reduced the proportion of primary follicles with extensive proliferation at 24 h. These findings are consistent with premature activation of primordial follicles as a proximate contributor to busulfan-induced loss of ovarian reserve and suggest that mTOR inhibition may mitigate this process. A two-time-point assay (24 h for proliferation, 7 days for morphology) provides a practical framework for future studies and translation.

01 Feb 2026·MOLECULAR IMMUNOLOGY

Acupuncture alleviates premature ovarian insufficiency via Rictor/mTOR pathway in mice by stimulating the Guanyuan acupoint

Article

Author: Zhu, Haimin ; Luo, Yu ; Huang, Suning ; Ma, Fei ; Chen, Yan ; Wu, Jiaman ; Zhao, Tianqi ; Ning, Yan ; Feng, Shiyu

Acupuncture is a popular therapeutic therapy for premature ovarian insufficiency (POI). The specific effect and the underlying mechanism of Guanyuan acupoint on the ovarian function of POI model mice remain unclear. The female C57BL6 mice were injected peritoneally with 12 mg/kg busulfan and 120 mg/kg cyclophosphamide to induce POI. The acupuncture intervention at Guanyuan acupoint was performed on the second day after the modeling. Vaginal smears were used to monitor the estrous cycle. The hematoxylin and eosin (H&E) staining was used to observe the morphological changes of ovarian tissue, and the TUNEL fluorescence staining assay was carried out to detect the apoptotic level of granulosa cells. The sex hormone levels were monitored as well. RNA sequencing was performed to select candidate gene which was regulated by acupuncture. Finally, the upstream lactating modification mechanism of Rictor was further investigated. The results showed that acupuncture at Guanyuan acupoint increased the number of vaginal exfoliated cells and inhibited the apoptosis of granulosa cell in POI model mice. Sex hormone levels indicated a marked decrease in AMH and E₂ in POI group, with a concurrent significant rise in FSH levels. Furthermore, the Rictor/mTOR pathway was inactivated in POI model group, while was prominently activated by acupuncture at Guanyuan acupoint. Acupuncture intervened H3K18la to increase Rictor promoter activity in POI. In conclusion, acupuncture at Guanyuan acupoint increased body and ovarian weight, improved ovarian tissue morphology and structure, improved follicle development, and regulated ovarian function in POI model mice. This might be related to the activation of the Rictor/mTOR pathway.

27 Jan 2026·Blood Advances

Comparative analysis of CRISPR-Cas9, lentiviral transduction, and base editing for sickle cell disease in a murine model

Article

Author: Le, Anh ; Yates, Bradley P. ; Liu, David R. ; Butt, Henna ; Eaton, William A. ; Gudmundsdottir, Bjorg ; Burke, Emma V. ; Newby, Gregory A. ; Pierciey, Francis J. ; Uchida, Naoya ; Sathish, Shruti ; Tisdale, John F. ; Hsieh, Matthew ; Lee, Duck-Yeon ; London, Evan ; Li, Quan ; Leonard, Alexis ; Demirci, Selami

Abstract:

Sickle cell disease (SCD) is a red blood cell disorder caused by a mutation in the β-globin gene, leading to sickle hemoglobin polymerization under low oxygen conditions. Both CRISPR-Cas9 editing and lentiviral transduction have shown promising clinical outcomes, but it remains unclear which approach is superior. Alternatively, new editing tools such as base editing may also be promising and reduce risks of genotoxicity. To compare these approaches, we studied them in an immunocompromised mouse model. We optimized ex vivo conditions in CD34+ hematopoietic stem and progenitor cells (HSPC) and infused edited SCD HSPC into busulfan-conditioned nonirradiated NOD,B6.SCID Il2rγ−/−KitW41/W41 (NBSGW) mice. Ex vivo analysis confirmed successful editing and transduction. At 16 weeks, bone marrow analysis showed similar human CD45+ cell engraftment across all groups (75%-90%). In the competitive transplantation group, there was a lower amount of B-cell lymphoma/leukemia 11A enhancer editing than base editing and lentiviral transduction. A secondary transplantation model yielded similar results. An antisickling assay showed significantly higher red blood cell sickling reduction in the base editing, transduction, and competitive transplantation groups compared to CRISPR-Cas9. In conclusion, although all methods showed therapeutic potential, base editing and lentiviral transduction provided superior outcomes over CRISPR-Cas9–mediated editing in a competitive murine transplantation model.

134

News (Medical) associated with Busulfan

02 Jan 2026

·www.prnewswire.com

Gene Therapy: 5 Biotech Stocks Chasing the $36B Prize

Issued on behalf of Avant Technologies Inc. VANCOUVER, BC, Jan. 2, 2026 /PRNewswire/ -- Equity Insider News Commentary – The gene therapy market is surging toward $36.55 billion by 2032, powered by rising demand for curative treatments that target genetic diseases at their source[1]. The FDA approved three transformative cell therapies this December alone, marking an inflection point where living medicine moves from experimental concept to commercial reality[2]. This revolution in restorative biology defines the pipelines of Avant Technologies, Inc. (OTCQB: AVAI), Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX), CRISPR Therapeutics (NASDAQ: CRSP), Prime Medicine, Inc. (NASDAQ: PRME), and Madrigal Pharmaceuticals, Inc. (NASDAQ: MDGL). The cell and gene therapy sector is projected to reach $39.61 billion by 2034, growing at a 17.98% compound annual rate as precision-based therapies address previously untreatable genetic disorders[3]. Manufacturing breakthroughs in viral vector delivery are now enabling scalable production of these therapies, driving capital inflows from investors betting on functional cures rather than lifelong symptom management[4]. Avant Technologies, Inc. (OTCQB: AVAI) is developing cell-based therapies for diabetes, aging, and chronic diseases using a proprietary encapsulation technology that protects genetically modified therapeutic cells from immune rejection. The company operates through two strategic joint ventures focused on addressing massive global health markets. The first, Insulinova, Inc., is a partnership with SGAustria Pte. Ltd., through which Avant is developing treatments for type 1 and insulin-dependent type 2 diabetes. The approach centers on a cell encapsulation technology that allows genetically modified insulin-producing cells to survive long-term in the body without being susceptible to any immune system attacks. This solves a fundamental problem that historically required patients to take lifelong immunosuppressive drugs with serious side effects. "Cell encapsulation is a game-changer in the field of regenerative medicine," said Chris Winter, CEO of Avant Technologies. "By partnering with SGAustria, we're ensuring that any genetically modified insulin-producing cells that we develop together can thrive in the body long-term and offer the potential of restoring natural glucose control and dramatically improving patients' quality of life. This technology not only minimizes risks like immune rejection but also prevents potential complications such as cell escape or tumor formation, making it a cornerstone for safe and scalable diabetes therapies." The diabetes market represents a substantial opportunity. According to the International Diabetes Federation, 589 million people globally live with type 1 and insulin-dependent type 2 diabetes, projected to reach 853 million by 2050. SGAustria's Cell-in-a-Box® technology creates a protective barrier around genetically modified cells, shielding them from any immune response while allowing nutrients, oxygen, and insulin to pass through freely. Avant's second joint venture, Klothonova, partners with Singapore-based Austrianova to develop anti-aging therapies and treatments for age-related diseases that restore circulating α-Klotho levels using genetically modified human cells that overexpress the Klotho protein. Research from the Mayo Clinic shows declining α-Klotho levels are associated with arterial stiffness, endothelial dysfunction, and vascular calcification. Both joint venture projects will utilize cell-encapsulation platforms backed by over 50 publications representing decades of development. The potential market spans multiple therapeutic areas: the global Alzheimer's market is projected to reach $32.8 billion by 2033, cardiovascular disease remains the world's leading cause of death, and kidney disease affects 850 million people worldwide. CONTINUED... Read this and more news for Avant Technologies at: Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) presented first-ever data on CASGEVY in children ages 5-11 years with severe sickle cell disease or transfusion-dependent beta thalassemia at the American Society of Hematology Annual Meeting. In the Phase 3 CLIMB-151 study for SCD, all four patients with sufficient follow-up achieved freedom from vaso-occlusive crises for at least 12 consecutive months, with the longest VOC-free duration approaching two years. In the Phase 3 CLIMB-141 study for TDT, all six evaluable patients achieved transfusion independence for at least 12 months. "These results — the first clinical data ever presented on any genetic therapy for children ages 5-11 years with SCD — again demonstrate the transformative potential of CASGEVY," said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. "With dosing completed in the 5-11 age group and the Commissioner's National Priority Voucher for CASGEVY in this population in hand, we are excited to begin global regulatory filings in the first half of next year." Vertex expects to initiate global regulatory submissions for the 5-11 age group in 1H 2026, with the Priority Voucher potentially accelerating review to 1-2 months. CRISPR Therapeutics (NASDAQ: CRSP) announced positive Phase 1 data from its ongoing clinical trial evaluating CTX310, an investigational in vivo CRISPR/Cas9 gene-editing therapy targeting ANGPTL3, demonstrating dose-dependent, durable reductions in circulating ANGPTL3 and lipids following a single-course intravenous infusion. At the highest dose, patients achieved mean reductions of 73% in ANGPTL3 (maximum 89%), 55% in triglycerides (maximum 84%), and 49% in LDL cholesterol (maximum 87%). Among patients with elevated baseline triglycerides exceeding 150 mg/dL, mean reductions of 60% were observed at therapeutic doses. "For the first time, we've shown that a single-course in vivo CRISPR treatment can safely and durably lower ANGPTL3, leading to clinically meaningful reductions in triglycerides and LDL," said Naimish Patel, M.D., Chief Medical Officer of CRISPR Therapeutics. "These data provide strong support for continued advancement of CTX310 and our broader cardiovascular gene-editing portfolio." CTX310 was well tolerated with no treatment-related serious adverse events and no Grade 3 or higher changes in liver transaminases, and CRISPR Therapeutics is advancing the therapy into Phase 1b clinical trials prioritizing severe hypertriglyceridemia and mixed dyslipidemia. Madrigal Pharmaceuticals, Inc. (NASDAQ: MDGL) presented new two-year data demonstrating Rezdiffra (resmetirom) significantly improved liver stiffness, fibrosis biomarkers, and markers of clinically significant portal hypertension risk in patients with compensated MASH cirrhosis at the American Association for the Study of Liver Diseases meeting. In a new analysis examining patients with more advanced compensated MASH cirrhosis (platelet count <100,000/µL), Rezdiffra demonstrated improvements across multiple imaging tests and biomarkers. Patients experienced mean reductions in liver stiffness of -7.9 kPa and -6.4 kPa respectively, with two thirds shifting to lower Baveno CSPH risk scores by year two. "MASH cirrhosis carries a 42 times higher risk of liver-related mortality, so there is an urgent need for an approved therapy that can protect patients from progressing to adverse outcomes," said David Soergel, M.D., Chief Medical Officer of Madrigal Pharmaceuticals. "Madrigal is determined to pioneer treatment in compensated MASH cirrhosis, and we are currently executing a fully enrolled Phase 3 outcomes study in this population." The company's Phase 3 MAESTRO-NASH OUTCOMES trial is evaluating Rezdiffra in compensated MASH cirrhosis, with Rezdiffra currently approved in the U.S. and Europe for MASH with moderate to advanced fibrosis. Prime Medicine (NASDAQ: PRME) announced the New England Journal of Medicine publication of Phase 1/2 clinical data for PM359, its investigational autologous hematopoietic stem cell product for p47phox chronic granulomatous disease, demonstrating the first-in-human safety and efficacy of Prime Editing technology. Both patients achieved rapid neutrophil engraftment with 69% and 83% dihydrorhodamine-positive neutrophils by Day 30, far exceeding the 20% threshold for clinical benefit, with stable activity suggesting correction in long-term repopulating hematopoietic stem cells. Both patients remained free of new CGD-related complications, with one stopping mesalamine treatment without disease flare. "Publication of these first-in-human data highlights Prime Editing's promise as a next-generation therapeutic platform, which is capable of delivering meaningful benefits to patients and which can be manufactured and delivered at clinical scale," said Mohammed Asmal, M.D., Ph.D., Chief Medical Officer of Prime Medicine. "Beyond demonstrating early clinical efficacy, these results offer important insights into Prime Editing's safety profile and potential advantages over other gene editing technologies." No clinically significant adverse events were attributable to PM359, with all observed toxicities consistent with busulfan-based conditioning. The results support Prime Editing's mechanism, which does not induce double-strand breaks, as potentially better tolerated by hematopoietic stem cells than other gene editing approaches. Madrigal Pharmaceuticals, Inc. (NASDAQ: MDGL) presented new two-year data demonstrating Rezdiffra (resmetirom) significantly improved liver stiffness, fibrosis biomarkers, and markers of clinically significant portal hypertension risk in patients with compensated MASH cirrhosis at the American Association for the Study of Liver Diseases meeting. In a new analysis examining patients with more advanced compensated MASH cirrhosis (platelet count <100,000/µL), Rezdiffra demonstrated improvements across multiple imaging tests and biomarkers. Patients experienced mean reductions in liver stiffness of -7.9 kPa and -6.4 kPa respectively, with two thirds shifting to lower Baveno CSPH risk scores by year two. "MASH cirrhosis carries a 42 times higher risk of liver-related mortality, so there is an urgent need for an approved therapy that can protect patients from progressing to adverse outcomes," said David Soergel, M.D., Chief Medical Officer of Madrigal Pharmaceuticals. "Madrigal is determined to pioneer treatment in compensated MASH cirrhosis, and we are currently executing a fully enrolled Phase 3 outcomes study in this population." The company's Phase 3 MAESTRO-NASH OUTCOMES trial is evaluating Rezdiffra in compensated MASH cirrhosis, with Rezdiffra currently approved in the U.S. and Europe for MASH with moderate to advanced fibrosis. Source: CONTACT: Equity Insider [email protected] (604) 265-2873 DISCLAIMER: Nothing in this publication should be considered as personalized financial advice. We are not licensed under securities laws to address your particular financial situation. No communication by our employees to you should be deemed as personalized financial advice. Please consult a licensed financial advisor before making any investment decision. This is a paid advertisement and is neither an offer nor recommendation to buy or sell any security. We hold no investment licenses and are thus neither licensed nor qualified to provide investment advice. The content in this report or email is not provided to any individual with a view toward their individual circumstances. Equity Insider is a wholly-owned subsidiary of Market IQ Media Group, Inc. ("MIQ"). MIQ has been paid a fee for Avant Technologies Inc. advertising and digital media from the company directly. There may be 3rd parties who may have shares Avant Technologies Inc., and may liquidate their shares which could have a negative effect on the price of the stock. This compensation constitutes a conflict of interest as to our ability to remain objective in our communication regarding the profiled company. Because of this conflict, individuals are strongly encouraged to not use this publication as the basis for any investment decision. The owner/operator of MIQ own shares of Avant Technologies Inc. which were purchased in the open market. MIQ reserves the right to buy and sell, and will buy and sell shares of Avant Technologies Inc. at any time thereafter without any further notice. We also expect further compensation as an ongoing digital media effort to increase visibility for the company, no further notice will be given, but let this disclaimer serve as notice that all material disseminated by MIQ has been approved by the above mentioned company; this is a paid advertisement, and we own shares of the mentioned company that we will sell, and we also reserve the right to buy shares of the company in the open market, or through other investment vehicles. While all information is believed to be reliable, it is not guaranteed by us to be accurate. Individuals should assume that all information contained in our newsletter is not trustworthy unless verified by their own independent research. Also, because events and circumstances frequently do not occur as expected, there will likely be differences between any predictions and actual results. Always consult a licensed investment professional before making any investment decision. Be extremely careful, investing in securities carries a high degree of risk; you may likely lose some or all of the investment. SOURCES CITED: Logo - 21% more press release views with Request a Demo

Clinical ResultPhase 3Drug Approval

08 Dec 2025

·www.globenewswire.com

Prime Medicine Announces The New England Journal of Medicine Publication of PM359 Clinical Data for the Treatment of Chronic Granulomatous Disease

Dec. 07, 2025 --- Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company committed to delivering a new class of differentiated one-time curative genetic therapies, today announced the publication of Phase 1/2 clinical data with PM359, the Company’s investigational autologous hematopoietic stem cell product for p47phox chronic granulomatous disease (CGD) in the New England Journal of Medicine (NEJM). The data will also be presented in a poster session at the 67th American Society of Hematology (ASH) Annual Meeting, December 6-9, 2025 in Orlando, Florida. The publication, titled “Prime Editing for p47-phox Chronic Granulomatous Disease,” reports initial data for two patients treated in the Phase 1/2 trial of PM359, which was designed to assess safety, biological activity and preliminary efficacy in adult and pediatric study participants. Both patients experienced rapid neutrophil and platelet engraftment, as well as durable restoration of NADPH oxidase activity and early clinical benefit, without any safety concerns. Together, these results provide the first-in-human demonstration of the safety and efficacy of Prime Editing, and support the potential for PM359 as a precise therapeutic strategy for CGD: Both patients enrolled in the study had a history of prior CGD-defining complications, including CGD-associated colitis (CAC), and skin and soft tissue infections, and both were maintained on long-term prophylactic therapy. Both patients experienced rapid neutrophil engraftment, achieving 69% and 83% dihydrorhodamine-positive (DHR+) neutrophils by Day 30, respectively, far in excess of the 20% projected minimum threshold for clinical benefit. DHR activity remained stable over time in both patients, suggesting that gene correction occurred in the long-term repopulating hematopoietic stem cells (HSCs) of the bone marrow. Both patients remain free of new CGD-related complications or significant intercurrent illnesses post-infusion; additionally, Patient 1 stopped his mesalamine treatment and has not experienced a flare of CAC, and Patient 2’s levels of fecal calprotectin have decreased substantially, and his chronic CAC symptoms have abated. No clinically significant adverse events attributable to PM359 occurred in either patient, and all observed toxicities were consistent with busulfan-based conditioning. “Publication of these first-in-human data highlights Prime Editing’s promise as a next-generation therapeutic platform, which is capable of delivering meaningful benefits to patients and which can be manufactured and delivered at clinical scale,” said Mohammed Asmal, M.D., Ph.D., Chief Medical Officer of Prime Medicine. “Beyond demonstrating early clinical efficacy, these results offer important insights into Prime Editing’s safety profile and potential advantages over other gene editing technologies. As described in the NEJM publication, we observed high recovery rates of viable corrected cells after a single mobilization cycle, as well as the rapid reconstitution of the hematopoietic system after infusion. Both support our belief that the mechanism of Prime Editing, which does not induce double-strand breaks, may be better tolerated by HSCs and other cell types – and therefore safer for patients – than other approaches.” Prime Medicine is a leading biotechnology company dedicated to creating and delivering the next generation of gene editing therapies to patients. The Company is deploying its proprietary Prime Editing platform, a versatile, precise and efficient gene editing technology, to develop a new class of differentiated one-time curative genetic therapies. Designed to make only the right edit at the right position within a gene while minimizing unwanted DNA modifications, Prime Editors have the potential to repair almost all types of genetic mutations and work in many different tissues, organs and cell types. Taken together, Prime Editing’s versatile gene editing capabilities could unlock opportunities across thousands of potential indications. Prime Medicine is currently progressing a diversified portfolio of investigational therapeutic programs organized around our core areas of focus: liver, lung, and immunology and oncology. Across each core area, Prime Medicine is focused initially on a set of high value programs, each targeting a disease with well-understood biology and a clearly defined clinical development and regulatory path, and each expected to provide the foundation for expansion into additional opportunities. Over time, the Company intends to maximize Prime Editing’s broad and versatile therapeutic potential, as well as the modularity of the Prime Editing platform, to rapidly and efficiently expand beyond the diseases in its current pipeline, potentially including additional genetic diseases, immunological diseases, cancers, infectious diseases, and targeting genetic risk factors in common diseases, which collectively impact millions of people. The content above comes from the network. if any infringement, please contact us to modify.

Clinical ResultASHGene Therapy

07 Dec 2025

·pharma.economictimes.indiatimes.com

Vertex's gene therapy shows promise in younger children with blood disorders

Bengaluru: Vertex Pharmaceuticals said on Saturday its gene therapy helped children aged between 5 and 11 years with sickle cell disease to be free of painful events and allowed those with another blood disorder that requires frequent blood transfusions to be transfusion-free for at least 12 consecutive months. This supports the potential for the therapy to treat the blood disorders in a younger patient population and will help Vertex expand the use of Casgevy , which is currently approved for patients 12 years and older with sickle cell disease or transfusion-dependent beta thalassemia (TDT). "These results - the first clinical data ever presented on any genetic therapy for children ages 5-11 years with SCD - again demonstrate the transformative potential of Casgevy ," said Vertex Chief Medical Officer Carmen Bozic. Casgevy is based on the Nobel Prize-winning CRISPR gene editing technology, which uses molecular "scissors" to trim faulty parts of genes that can then be disabled or replaced with new strands of normal DNA. The company will begin filing to global regulators in the first half of next year. It has received a Commissioner's National Priority Voucher for Casgevy in the 5-11 year age group, which will accelerate the health regulator's review. In a late-stage trial, four children with SCD who were dosed with Casgevy with sufficient follow-up were free from vaso-occlusive crises for at least 12 consecutive months. The longest period without these events reached about two years, the company said. Vaso-occlusive crises, a key hallmark of the inherited blood disorder, are caused by blockages of blood vessels by sickled red blood cells and result in severe and debilitating pain. After infusion with Casgevy, 12 patients with TDT were transfusion free, with the longest duration being just under two years. A patient with TDT died from pneumonia and multi-organ failure due to severe veno-occlusive disease, a liver condition, related to a pre-transplant treatment that uses the chemotherapy drug busulfan. The results were presented at the American Society of Hematology Annual Meeting. (Reporting by Sriparna Roy in Bengaluru; Editing by Alan Barona)

Clinical ResultGene TherapyASH

100 Deals associated with Busulfan

External Link

KEGG	Wiki	ATC	Drug Bank
D00248	Busulfan	L01AB01	DB01008

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Hematopoietic stem cell transplantation	European Union	Pierre Fabre Médicament SAS	09 Jul 2003
Hematopoietic stem cell transplantation	Iceland	Pierre Fabre Médicament SAS	09 Jul 2003
Hematopoietic stem cell transplantation	Liechtenstein	Pierre Fabre Médicament SAS	09 Jul 2003
Hematopoietic stem cell transplantation	Norway	Pierre Fabre Médicament SAS	09 Jul 2003
Bone marrow transplant rejection	Israel	Pierre Fabre SA	22 Feb 2002
Philadelphia chromosome positive chronic myelogenous leukemia	United States	Otsuka Pharmaceutical Co., Ltd.	04 Feb 1999
Polycythemia Vera	Japan	Takeda Pharmaceutical Co., Ltd.	01 Jan 1981
Myeloid Leukemia	Japan	Takeda Pharmaceutical Co., Ltd.	07 Oct 1957
Acute Myeloid Leukemia	United States	Waylis Therapeutics LLC	26 Jun 1954

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Multiple Myeloma	Phase 3	United States	Otsuka Pharmaceutical Development & Commercialization, Inc.	30 Sep 2011
Extranodal NK-T-Cell Lymphoma	Phase 2	South Korea	Otsuka Pharmaceutical Co., Ltd.	01 Dec 2016
Diffuse Large B-Cell Lymphoma	Phase 2	United States	Celgene Corp.	22 Jun 2016
Adenosine deaminase deficiency	Phase 2	United States	Orchard Therapeutics Plc	02 Aug 2013
Relapse multiple myeloma	Phase 2	United States	Otsuka Pharmaceutical Development & Commercialization, Inc.	01 May 2010
Relapse multiple myeloma	Phase 2	United States	Otsuka America Pharmaceutical, Inc.	01 May 2010
Relapse multiple myeloma	Phase 2	Canada	Otsuka Pharmaceutical Development & Commercialization, Inc.	01 May 2010
Hodgkin's Lymphoma	Phase 2	United States	Otsuka Pharmaceutical Development & Commercialization, Inc.	01 Jan 2010
Hodgkin's Lymphoma	Phase 2	Canada	Otsuka Pharmaceutical Development & Commercialization, Inc.	01 Jan 2010
Non-Hodgkin Lymphoma	Phase 2	United States	Otsuka Pharmaceutical Development & Commercialization, Inc.	01 Jan 2010

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT01349101 (CTgov)	Phase 2	Hematologic Neoplasms \| Chronic Myelomonocytic Leukemia \| acute leukemia	78	Hematopoietic stem cell transplantation+Cyclophosphamide+Tacrolimus+mycophenolate mofetil (Myeloablative HSCT)	izkhcttdkj = wpfgqvaayc akfcrdqimw (qhthgdquwf, cwxmsawtup - pbliaukpxo) View more	-	15 Dec 2025
NCT01349101 (CTgov)	Phase 2		78	Hematopoietic stem cell transplantation+Fludarabine+Busulfan+Cyclophosphamide+Tacrolimus+mycophenolate mofetil (Reduced Intensity HSCT)	izkhcttdkj = hnfrmgeptd akfcrdqimw (qhthgdquwf, rbhvaiqkrl - eobchgzzmc) View more	-	15 Dec 2025
ASH2025	Not Applicable	Hematologic Neoplasms	150	Pharmacokinetics-guided TBF conditioning	bppkupfmrj(sjonpxboyj) = gnjqahtmtl lsddtqoybn (qmwvzmgswk ) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Hematologic Neoplasms	150	Fixed-dose TBF	bppkupfmrj(sjonpxboyj) = axtcaoxgmr lsddtqoybn (qmwvzmgswk ) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Diffuse large B-cell lymphoma recurrent	30	Rituximab+Busulfan+Melphalan	wcmzcfzwzl(xwmytxobeb) = jlujvrtozy qbdnwniuhh (egwpzyoctq ) View more	Positive	06 Dec 2025
NCT05667402 (ASH2025)	Not Applicable	Central nervous system leukaemia	21	Mitoxantrone hydrochloride liposome injection combined with thiotepa-busulfan-fludarabine (MTBF)	nzbzjybjrk(aquutjlfhs) = The most common adverse reactions of conditioning were nausea,vomiting,diarrhea and oral mucositis bagfgplppp (qrewnkvkuo ) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Anemia, Sickle Cell	60	FludarabineMethotrexatebusulfanCyclosporin + FludarabineMethotrexatebusulfanCyclosporin (Sickle Cell Disease)	smtmcfnchz(mrzitarcoj) = fagmmmhbds opuysgvrbv (zfdzygatta, 86 - 100) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Beta-Thalassemia \| Anemia, Sickle Cell	113	Fludarabine + Dexamethasone + Busulfan + Cyclophosphamide + Cyclosporine + Methotrexate (Thalassemia major or Sickle cell disease)	hbmydyaisr(bxsfsoercc) = nxdmuvbksv mkwqkujvrv (emuwproegi ) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Hematologic Neoplasms	27	Thiotepa+busulfan+cyclophosphamide	stizpbzwkb(kgtzopdahl) = yjlnxllvfr kyncmgbemf (myszaqjwhh, 6.8) View more	Positive	06 Dec 2025
NCT05579769 (CTgov)	Phase 2	Myeloid Tumor \| Graft vs Host Disease	3	Cyclophosphamide (Cy) (Total Body Irradiation (TBI)/Cyclophosphamide (Cy))	mvylkasksj = yanhbdqpnv crevzvzzak (lciawrprfy, jlpjpgmfdu - jbzzhhxtdb) View more	-	02 Oct 2025
NCT05579769 (CTgov)	Phase 2	Myeloid Tumor \| Graft vs Host Disease	3	Fludarabine (TBF)+Busulfan+Thiotepa (Thiotepa, Busulfan, and Fludarabine (TBF))	mvylkasksj = ixnxfeyjpe crevzvzzak (lciawrprfy, ntidllajqy - gxbclgovcj) View more	-	02 Oct 2025
NCT03247088 (S266, EHA2025)	Phase 1/2	Acute Myeloid Leukemia FLT3 ITD	59	Sorafenib 800mg	pcisbivmpu(txhdsgctdq) = dwqsoqyetu jorfrlekmw (mnrrgmapis, 61.8 - 84.8)	Positive	14 May 2025
PB3380 (EHA2025)	Not Applicable	Hematologic Diseases	27	Thiotepa 5 mg/kg/day	wsptenxhqh(htgnxniyzj) = Mostly grade 1-2, well tolerated wxdbpidumd (mcxwkudnui )	Positive	14 May 2025
PB3380 (EHA2025)	Not Applicable	Hematologic Diseases	27	Busulfan 3.2mg/kg/d		Positive	14 May 2025

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