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 Date17 Jul 2026

Sponsor / Collaborator

National Cancer Institute

NCT06996119

/ Not yet recruitingPhase 1IIT

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]

NCT07228624

/ RecruitingPhase 2IIT

Peri-Hematopoietic Cell Transplantation Ruxolitinib in Patients 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 Date04 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

7,392

Literatures (Medical) associated with Busulfan

01 Mar 2026·TOXICOLOGY LETTERS

Busulfan plus cyclophosphamide induced spermatogenic dysfunction and recovery: A dynamic change perspective

Article

Author: Li, Fuping ; Li, Tianyu ; Liu, Xiao ; Luo, Xiaolei ; Wang, Yanyun ; Tang, Zhichen ; Zhou, Bin ; Song, Yaping ; Wei, Xing ; Zhang, Lin ; Su, Min

Busulfan combined with cyclophosphamide (BuCy) is a common conditioning regimen before hematopoietic stem cell transplantation, but it causes severe gonadotoxicity, with nearly half of male patients suffering from irreversible infertility. Existing mouse models mostly use busulfan alone, which does not fully mimic clinical treatment. Here, we applied a BuCy treatment regimen in mice and performed a longitudinal characterization of testicular injury and recovery. BuCy treatment caused marked testicular atrophy in mice, severely disrupted seminiferous tubule architecture, and dramatically reduced sperm count and motility, with partial recovery at later time points. ScRNA-seq revealed a stepwise decline in germ cell populations, with spermatogonia disappearing earlier than spermatogonial stem cells (SSCs). In addition, we observed the highest number of differentially expressed genes (DEGs) at day 28. Functional enrichment highlighted disruptions in spermatogenesis, RNA metabolism, and chromatin regulation. This study systematically characterized the long-term, multi-time-point dynamics of BuCy-induced testicular damage and recovery in mice, with single-cell transcriptomic profiling providing complementary observations at the cellular level.

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 Mar 2026·Pharmacogenetics and Genomics

Effects of glutathione-S-transferase polymorphisms on intravenous busulfan in hematopoietic stem cell transplant patients: a meta-analysis

Review

Author: Al Riyami, Intisar ; Al Khabori, Murtadha ; Salman, Bushra ; Al Maskari, Raya

Objective:

This study examined the impact of glutathione- S -transferase polymorphisms (GSTA1, GSTM1, GSTP1, and GSTT1) on the area under the curve (AUC), clearance, veno-occlusive disease (VOD), and graft-versus-host disease (GvHD) in hematopoietic stem cell transplant (HSCT) patients treated with intravenous busulfan.

Methods:

A systematic review was performed on three electronic databases to identify relevant studies. The relative risk and the 95% confidence interval (CI) of the association of different GST polymorphisms with pharmacokinetic and clinical outcomes were reported using the random and fixed effect models. Quality of the studies was assessed using the Newcastle–Ottawa Scale for cohort studies. Heterogeneity between studies and publication bias were also carried out using R software.

Results:

Eighteen studies were included in the meta-analysis. GSTA1*A/*B was significantly associated with lower clearance (95% CI: 0.008–1.223, P = 0.048) and higher AUC (95% CI: −374.960 to –56.661, P = 0.008) than the GSTA1*A/*A genotype. GSTA1*B/*B had a higher busulfan AUC than GSTA1*A/*A (95% CI: −403.531 to −89.454, P = 0.002). None of the other genotypes was significantly associated with busulfan pharmacokinetic parameters or the risk of VOD or GvHD.

Conclusion:

GSTA1 should be considered as a guide for intravenous busulfan dosing in allogeneic HSCT patients, where patients with the GSTA1*A/*A genotype require a higher dose than GSTA1*B carriers.

136

News (Medical) associated with Busulfan

23 Feb 2026

·www.biospace.com

Sickle Cell Gene Therapies Casgevy and Lyfgenia Still Lacking Traction 2 Years In

iStock, BrianAJackson The first gene therapies approved to treat sickle cell disease in December 2023 are struggling on the market. But there are glimpses of forward momentum as Vertex and Genetix Bio provide updates. Just over two years ago, Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy and bluebird bio’s Lyfgenia won FDA approval as the first gene therapies for sickle cell disease. It was hailed as a watershed moment for patients with the debilitating blood disorder, but even then, there were concerns about access to treatment. “The numbers of people who can be treated are limited,” Eric Kmiec, founder and executive director of the ChristianaCare Gene Editing Institute, said in a statement to BioSpace the day of the approvals. “We must work with the health care industry and pharmaceutical companies who will market, produce and deliver the treatments to make sure that all people can get access.” Fast-forward two years and Kmiec’s warning has come to pass. Last year, just 64 patients with sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT)—for which Casgevy was greenlit in January 2024—received infusions of the gene therapy, according to Vertex’s full-year 2025 earnings report . An additional 147 people had their first cell collection. Meanwhile, Genetix Bio— formerly bluebird bio —has treated over 100 patients with Lyfgenia, which is only approved for SCD, a member of Genetix’s executive team told BioSpace in an email. But both companies are aiming for more, of course. In 2025, Vertex earned $116 million in revenue from Casgevy. The company expects the gene therapy—along with recently approved pain medication Journavx—will combine to bring in $500 million this year, a 185% increase over 2025. Whether Vertex will hit those numbers remains to be seen. Courtney Rice, principal at Acadia Strategy Partners, pointed to potential reasons for the seemingly lackluster launch that could continue to challenge growth. “You’re asking a lot of time for these patients to be out of the game, both from the conditioning time to the procedure to the engraftment,” Rice said of the new gene therapies. “And that somehow sort of got glossed over in the parade, in the champagne shaking and excitement.” Barriers to Uptake Approximately 100,000 Americans suffer from sickle cell disease, which is caused by a mutation in the genes involved in producing hemoglobin’s beta subunit, an iron-rich compound found in red blood cells (RBC). This results in some of the cells being shaped like “sickles,” slowing or blocking blood flow and leading to anemia and episodes of extreme pain. More than 90% of patients are Black. In a statement to BioSpace in December 2023, Tim Hunt, CEO of the Alliance for Regenerative Medicine, called the approval of Casgevy, the first-ever CRISPR-based gene editing therapy, “a seminal moment in the history of biotechnology and human health.” The Genetix executive emphasized that tens of thousands of patients with SCD could benefit from Lyfgenia. “However, despite broad reimbursement already in place, the vast majority of patients have not been treated.” For Rice, the harsh conditioning regimen is the primary impediment to uptake of the gene therapies. Both Casgevy and Lyfgenia require patients to be treated with a conditioning regimen called busulfan, a chemotherapy drug used to prepare the bone marrow for the infusion of the gene therapies. Busulfan is used prior to the infusion of Casgevy and Lyfgenia to destroy the defective blood stems in the bone marrow to make way for the edited cells. “That conditioning regimen is gnarly at best,” Rice said. For Victoria Gray, a patient advocate and the first person with SCD to be treated with Casgevy, this conditioning regimen was the most challenging part of the process. Gray told BioSpace that in vivo gene therapy could open up access to more patients because it negates the need for these chemotherapeutic agents. Genetic editing biotech Tessera Therapeutics is developing an in vivo gene writer for SCD, aiming to directly correct the mutation that causes cells to sickle. The company received a $50 million investment from the Bill & Melinda Gates Foundation for the program in December 2024. Another barrier to access for the current gene therapies, Rice noted, is that busulfan comes with a high risk of infertility. In addition to destroying the defective blood stem cells in the bone marrow to make way for the edited cells, the toxic treatment wipes out other rapidly dividing cells, including ovarian cells that develop into eggs. “Sterility is a hot button issue in the SCD community,” Cantor Fitzgerald noted in its presentation. “Younger (pre-pubescent) patients who are most appropriate for cell transplantation are not able to prevent infertility through sperm or egg cell banking.” Meanwhile, the extensive time requirement involved in treatment with Casgevy and Lyfgenia is somewhat of a catch-22, according to Rice, who noted that people who are healthy enough to be eligible “are probably in the working population and commercially insured.” Rice recalled a conversation with a colleague about another potentially curative SCD treatment: hematopoietic stem cell transplantation (HSCT), which, unlike the approved gene therapies, requires a human leukocyte antigen (HLA)–identical sibling donor. Her colleague explained that patients are reluctant to receive a transplantation even when there is an HLA match identified because of the conditioning regimen. A 2023 presentation by Cantor Fitzgerald shared with BioSpace showed that of the approximately 100,000 patients with SCD, around 15,000 have an HLA-matched sibling donor, but only between 1,000 and 2,000 are treated with HSCT. Rice recalled her colleague posing a poignant question about Casgevy and Lyfgenia: “What makes you think that because [SCD treatment is] now open to all people . . . that the uptake would be higher?” Gene therapy In Vivo Is Having a Moment as Cell and Gene Therapy Sector Gathers in San Diego A recurring theme Tuesday morning at Phacilitate’s Advanced Therapies Week was the quickly emerging potential of in vivo approaches to cell and gene therapy—a trend also reflected in recent investments by Eli Lilly and Regeneron. February 11, 2026 · 4 min read · Heather McKenzie Finally, Gray pointed to a lack of prescriber knowledge as stunting the launches of Casgevy and Lyfgenia. “When [patients] go in and ask about Casgevy, gene therapy, Lyfgenia, they’re being discouraged by medical providers, and it’s not surprising to me because I’ve been there,” she said during her presentation. “Until the doctors that see patients on a regular basis are educated, there are going to be so many gaps.” Upward Momentum These barriers and others involved with these complex treatments—such as manufacturing issues and a challenging return-on-investment proposition—made it impossible for bluebird to survive as a public company. Bluebird bio sold itself to two global investment firms, Carlyle and SK Capital Partners, in February last year for $30 million—a troubling smoke signal sent out by one of gene therapy’s pioneers. Unable to successfully market its groundbreaking products, the biotech was running out of money, and the go-private deal was a lifeboat. Then in September 2025, the company reverted to its roots, assuming its original moniker of Genetix Biotherapeutics . Genetix at that time also revealed some of its strategy to revive the company. This included plans “to substantially increase our manufacturing capacity within the next year to meet growing demand,” according to a Sept. 18 press release . Bankruptcy Bluebird’s Go-Private Deal Highlights Fraught Life Preserver for Struggling Biotechs Facing declining valuations and funding challenges, public biotechs like bluebird bio are going private to restructure, reduce regulatory burdens and refocus on long-term growth. March 12, 2025 · 6 min read · Ana Mulero Indeed, commercial demand for Lyfgenia “has surged significantly,” the Genetix executive said. “We expect rapid demand and growth for Lyfgenia in 2026 and beyond,” the company executive said. Casgevy also appears to be trending upward. Thirty of the 64 patients infused with Vertex and CRISPR’s product received those infusions during the fourth quarter of 2025, Vertex noted in its 2025 earnings report. And the company plans to approval of Casgevy in children aged 5 to 11 in the first half of this year, CEO Reshma Kewalramani said on a Feb. 12 earnings call. Vertex received a Commissioner’s National Priority Review Voucher for Casgevy and expects a quick review for the label expansion. Rice was less optimistic, predicting that the uptake of Casgevy and Lyfgenia will actually decrease. “I tracked both of those launches, and leading up to it, we had this bolus. People [were] waiting in line.” Now, she said, “We’ve probably burned through that bolus.” .responsive-container { display: flex; flex-wrap: wrap; border: 1px solid #ededed; font-family: Helvetica, Arial, Sans-Serif; padding: 20px 20px 10px 20px; } .column-left { flex: 1; max-width: 45%; padding: 20px 20px 10px 20px; } .column-right { flex: 2; padding: 20px 20px 10px 20px; } @media (max-width: 768px) { .column-left, .column-right { max-width: 100%; flex: 100%; padding: 10px 0; } } Subscribe to ClinicaSpace! Clinical trial results, research news, the latest in cancer, cell and gene therapy hbspt.forms.create({ region: "na1", portalId: "4413123", formId: "674f4dc0-7371-4190-86cc-e1afd00b08ea", onFormSubmitted: function($form, data) { window.dataLayer = window.dataLayer || []; window.dataLayer.push({ 'event': 'GTMevent', 'event_name': 'newsletter_sign_up' }); } });

Gene TherapyPriority ReviewDrug Approval

11 Feb 2026

·www.biospace.com

Is Having a Moment as Cell and Gene Therapy Sector Gathers in San Diego

iStock, quantic69 A recurring theme Tuesday morning at Phacilitate’s Advanced Therapies Week was the quickly emerging potential of in vivo approaches to cell and gene therapy—a trend also reflected in recent investments by Eli Lilly and Regeneron. In vivo cell and gene therapy is en vogue at Phacilitate’s Advanced Therapies Week in San Diego this week, a theme that reflects a recent rush of activity in the space, including Eli Lilly’s $2.4 billion acquisition of Orna Therapeutics on Monday. “In vivo is the new allogeneic,” Susan Nichols, CEO of Propel Bio, a commercialization acceleration partner, said during Tuesday’s kickoff session, which convened Phacilitate’s advisory board to discuss 2025: What Worked. What Didn’t. What Comes Next? While ex vivo cell therapy involves extracting patient cells, modifying or engineering them outside the body and then reinfusing them into the patient, in vivo approaches deliver the therapeutic payload directly into the patient’s body, bypassing this step. In vivo cell and gene therapies have “huge promise [and] heavy investment,” Nichols continued, though she added that human data is limited for now. “As one advisor put it, in vivo approaches are full of promise, but the investment momentum is outpacing the clinical data.” While in vivo cell and gene therapy (CGT) approaches might be early-stage, Big Pharma—including obesity darling Eli Lilly—is buying in. In the past, issues around accessibility had limited Lilly’s interest in the modality. “The data is amazing,” Lilly CEO David Ricks told Reuters in 2019, “but practically, it’s not reaching many people.” Lilly appears to believe that Orna’s technology, which uses circular RNA paired with novel lipid nanoparticles to enable a patient’s own body to generate cell therapies, can solve this challenge. “We look forward to working with Orna colleagues to potentially unlock an entirely new class of genetic medicines and cell therapies for patients who today have limited or no treatment options,” Francisco Ramírez-Valle, head of Immunology Research and Early Clinical Development at Lilly, said in a statement . Mergers & acquisitions Eli Lilly Enters CAR T Realm With $2.4B Orna Buy, Starting With Autoimmune The deal gets Lilly access to Orna’s in vivo CAR T technology. The biotech’s lead asset, which has yet to start clinical testing, is focused on B cell–driven autoimmune diseases. February 9, 2026 · 2 min read · Dan Samorodnitsky Negating the Need for Chemotherapy Victoria Gray, a patient advocate who was the first person with sickle cell disease (SCD) to be treated with Vertex Pharmaceuticals’ and CRISPR Therapeutics’ CRISPR-based gene editing treatment Casgevy, told BioSpace that in vivo gene therapy could be available to more people because it negates the need for immunosuppressive chemotherapy. “I would love to see the science advance for patients like myself” who are currently required to undergo a conditioning regimen with a chemotherapeutic agent, Gray said during a patient advocacy session at Advanced Therapies Week on Tuesday. That agent, busulfan, is used to destroy the defective blood stem cells in the bone marrow to make way for the edited cells. Both Casgevy and Genetix Bio’s (formerly bluebird bio) Lyfgenia come with this caveat. “That was the hardest part for me,” Gray said. Kristen Hege, former senior vice president of Early Clinical Development, Hematology/Oncology & Cell Therapy at Bristol Myers Squibb, emphasized the long-term impact of lymphodepletion treatment. “When we think about in vivo gene therapy, I think a lot of people talk about patient access, cost of goods, but there’s more than that,” she said during a session titled: We’ve solved for science. What now? The chemotherapy that’s required before the infusion of ex vivo CAR T cells can cause immune suppression that can last for weeks, she continued. This can “increase the risk of infection, and we don’t really talk about it, but [can also] cause leukemia. Eliminating the need for lymphodepleting chemotherapy, if it pans out with in vivo , could be another real advantage.” Indeed, the approach is catching on. In addition to Lilly, Regeneron also recently bought into the in vivo approach, linking up with Cambridge, Mass.–based Tessera Therapeutics late last year. The partnership is aimed at advancing the in vivo editor TSRA-196 for alpha-1 antitrypsin deficiency (AATD). Tessera is also developing a gene writer for SCD, Cell & Gene.com reported in January 2025, aiming to directly correct the mutation that causes cells to sickle through a one-time IV treatment. But the potential advantages don’t stop with access and tolerability, according to Hege. “Regulations are often built around what you can measure, and if you can measure it, then you’re required to measure it. Or if you can control it, then you’re required to control it,” she said. “I think that’s been one of the challenges for ex vivo manufacturing, because there’s a lot of things that you can measure and control.” With in vivo approaches, Hege continued, “What happens in the patient is beyond your control, and therefore you’re not held to a standard” in terms of pharmacodynamic, phenotypic and gene expression endpoints as part of the product characterization. “Belief is currently leading data, not following it,” Nichols said of in vivo treatments during her presentation. Ultragenyx Pharmaceutical has long stood behind in vivo gene therapy. The rare disease–focused biotech presented new data last week from two trials of its novel in vivo AAV9 gene therapy UX111 at the WORLDSymposium 2026. The candidate, in development for Sanfilippo A syndrome, sustained cognitive improvements over up to eight years of follow-up, according to Ultragenyx’s Feb. 3 press release . UX111 was rejected by the FDA in July 2025, but clinical data was not the issue. Rather, the agency wanted to see “improvements” in “certain aspects” of the therapy’s chemistry, manufacturing and controls,” Ultragenyx said in its announcement of the complete response letter. “Regulation is no longer the gating issue,” Nichols concluded. Editor’s note: BioSpace is a media partner of Phacilitate’s Advanced Therapies Week, held Feb. 9–12 at the San Diego Convention Center.

Gene TherapyAcquisitionCell TherapyImmunotherapy

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

100 Deals associated with Busulfan

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KEGG	Wiki	ATC	Drug Bank
D00248	Busulfan	L01AB01	DB01008

R&D Status

Approved

10 top approved records.

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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.

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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 America Pharmaceutical, Inc.	01 May 2010
Relapse multiple myeloma	Phase 2	United States	Otsuka Pharmaceutical Development & Commercialization, 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


Tandem Meetings ASTCT and CIBMTR2026	Not Applicable	-	48	Busulfan+CyclophosphamideFludarabine + Busulfan+CyclophosphamideFludarabineamide (3.2 mg/kg)	bcjzpiqnbl(ulldcigntg) = syrwgejbcd vjregdzylh (vnxrizhsxz ) View more	Positive	04 Feb 2026
Tandem Meetings ASTCT and CIBMTR2026	Not Applicable	-	48	Busulfan+Fludarabine (3.6 mg/kg)	bcjzpiqnbl(ulldcigntg) = pkgfzdjixl vjregdzylh (vnxrizhsxz ) View more	Positive	04 Feb 2026
NCT01349101 (CTgov)	Phase 2	Hematologic Neoplasms \| Chronic Myelomonocytic Leukemia \| acute leukemia	78	Hematopoietic stem cell transplantation+Cyclophosphamide+Tacrolimus+mycophenolate mofetil (Myeloablative HSCT)	ceusdqubyr = elmbzshaxu xddkygbaji (fsvboqhbuw, vosjrvnwtb - zsuilplhck) View more	-	15 Dec 2025
NCT01349101 (CTgov)	Phase 2		78	Hematopoietic stem cell transplantation+Fludarabine+Busulfan+Cyclophosphamide+Tacrolimus+mycophenolate mofetil (Reduced Intensity HSCT)	ceusdqubyr = icmovcvimi xddkygbaji (fsvboqhbuw, ietqpprgjn - drcyrfsckr) View more	-	15 Dec 2025
NCT05667402 (ASH2025)	Not Applicable	Central nervous system leukaemia	21	Mitoxantrone hydrochloride liposome injection combined with thiotepa-busulfan-fludarabine (MTBF)	gqnjkjxzor(ksmnvlbtib) = The most common adverse reactions of conditioning were nausea,vomiting,diarrhea and oral mucositis pqucfhnloq (gquevtblkb ) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Hematologic Neoplasms	150	Pharmacokinetics-guided TBF conditioning	algjqwydqc(omtvkmddnt) = icglyjffot oajcawyujf (nrtqfjvsir ) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Hematologic Neoplasms	150	Fixed-dose TBF	algjqwydqc(omtvkmddnt) = ouvhytpcqg oajcawyujf (nrtqfjvsir ) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Anemia, Sickle Cell	60	FludarabineMethotrexatebusulfanCyclosporin + FludarabineMethotrexatebusulfanCyclosporin (Sickle Cell Disease)	lvqvmibatu(xspkflxoke) = vwkvhnwaef xnjibkiitj (fdjygkcwys, 86 - 100) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Diffuse large B-cell lymphoma recurrent	30	Rituximab+Busulfan+Melphalan	yosjfchyvx(rlxnmggjnn) = ekvygkdhfc kmwzfhxqkg (vqcicjouyz ) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Hematologic Neoplasms	27	Thiotepa+busulfan+cyclophosphamide	snyozrxwkw(vsddncjlhq) = xkoxswuvst olnulmeaxy (jcuggnaxvd, 6.8) 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)	mrdbvehckc(slivkeoorn) = yypfksoeqm qhjrhbejqb (zwmzmfjezt ) 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))	lthvvwaiit = emrnmwozhk xaxyeirbrn (ltdoaxiumr, pekrgorddk - rtcckteasu) 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))	lthvvwaiit = juvbbvlwbe xaxyeirbrn (ltdoaxiumr, jrnwjlemzt - yvhgahfcmc) View more	-	02 Oct 2025
NCT03247088 (S266, EHA2025)	Phase 1/2	Acute Myeloid Leukemia FLT3 ITD	59	Sorafenib 800mg	sknfqqlgpq(aphzxdowaj) = cbsgmqwuzt egxvxsihtn (hbyzqflkpg, 61.8 - 84.8)	Positive	14 May 2025

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