RegulationPriority Review (United States), Fast Track (United States), Orphan Drug (United States), Orphan Drug (European Union), PRIME (European Union), Regenerative Medicine Advanced Therapy (United States), Orphan Drug (United Kingdom), Priority Review (Canada), Conditional marketing approval (United Kingdom), Rare Pediatric Disease (United States)

Clinical Trials associated with Exagamglogene Autotemcel

NCT05951205

/ Not yet recruitingPhase 3

A Phase 3 Study to Evaluate Efficacy and Safety of a Single Dose of Exa-cel in Subjects With Severe Sickle Cell Disease, βS/βC Genotype

The purpose of the study is to evaluate the efficacy and safety of CTX001 (exa-cel) in adolescent and adult participants with severe sickle cell disease (SCD), βS/βC genotype (HbSC).

Start Date31 Jul 2027

Sponsor / Collaborator

Vertex Pharmaceuticals, Inc.

ChiCTR2300073795

/ RecruitingEarly Phase 1IIT

An International, Multicenter, Phase I Open Label Study to Evaluate Safety and Efficacy of Administration of Autologous CD34+ Cells Gene Edited Ex Vivo Using a CRISPR/AAV6 Platform to Introduce a Codon Optimized cDNA Version of the Red-Cell Type Pyruvate Kinase Genein Adult and Pediatric Patients with Severe Pyruvate Kinase

Start Date09 Nov 2023

Sponsor / Collaborator

Shanghai Children's Medical Center

NCT05477563

/ RecruitingPhase 3

A Phase 3b Study to Evaluate Efficacy and Safety of a Single Dose of Autologous CRISPR Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Transfusion-Dependent β-Thalassemia or Severe Sickle Cell Disease

This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) using CTX001.

Start Date02 Aug 2022

Sponsor / Collaborator

Vertex Pharmaceuticals, Inc.

100 Clinical Results associated with Exagamglogene Autotemcel

100 Translational Medicine associated with Exagamglogene Autotemcel

100 Patents (Medical) associated with Exagamglogene Autotemcel

Literatures (Medical) associated with Exagamglogene Autotemcel

01 May 2025·PHARMACOECONOMICS

Innovative Payment Models for Sickle-Cell Disease Gene Therapies in Medicaid: Leveraging Real-World Data and Insights from CMMI’s Gene Therapy Access Model

Article

Author: Nair, Kavita ; Leonard, Jim ; DiStefano, Michael J ; Wright, Garth C ; Zemplenyi, Antal ; McQueen, R Brett ; Anderson, Kelly E

OBJECTIVE:

This study aims to evaluate the financial implications of implementing various payment models, including outcome-based agreements (OBAs), volume-based rebates, and guaranteed rebates, for the newly approved gene therapies, exagamglogene autotemcel (exa-cel) and lovotibeglogene autotemcel (lovo-cel), in the treatment of sickle-cell disease (SCD) from the perspective of Colorado Medicaid. The analysis specifically examines the cost of standard of care (SoC) for severe SCD, the impact of different eligibility criteria based on vaso-occlusive events (VOEs), and the potential financial impacts associated with rebate structures.

METHODS:

Data from the Colorado Department of Health Care Policy & Financing (HCPF) database was used to estimate the annual costs for Medicaid-enrolled patients with severe SCD from 2018 to 2023. Patients were selected based on various eligibility criteria, including the number of VOEs, acute chest syndrome events, and stroke diagnoses. Three-state Markov models (SCD, stable, and dead) were constructed to compare the costs of SoC and gene therapies. The durability of gene therapy effectiveness and the financial impact of OBAs, volume-based rebates, and guaranteed rebates were evaluated over a 6-year contract period, with scenarios reflecting different VOE criteria and treatment durability.

RESULTS:

The average annual SoC cost for severe SCD patients (N = 138) was US$45,941 (SD US$59,653), with higher costs associated with more frequent VOEs. Gene therapies exa-cel and lovo-cel, with one-off list prices of US$2.2 million and US$3.1 million, respectively, exhibited high upfront costs, resulting in a negative cumulative balance averaging - US$2.11 million for exa-cel and - US$3.00 million for lovo-cel per patient over 6 years compared with SoC. Outcome-based rebates could potentially save Medicaid approximately US$260K (uncertainty interval 88K-772K) per patient on average for exa-cel and US$367K (uncertainty interval 122K-1111K) for lovo-cel after they pay the full up-front cost. Volume-based and guaranteed rebates also offered potential savings but varied in impact based on contract duration and effectiveness of gene therapy.

CONCLUSIONS:

The study highlights critical considerations for Medicaid in negotiating OBAs for SCD gene therapies. Achieving budget neutrality over 6 years is unlikely due to low SoC costs. However, payment models can enhance value-based spending by linking high therapy costs and potential rebates to the health gains these treatments may offer. OBAs offer offsets contingent on therapy effectiveness durability and contract terms (such as length and price), while varying eligibility criteria impact budgets and outcomes. Medicaid real-world data is crucial for navigating complexities in defining eligible populations and structuring OBAs.

01 May 2025·CYTOTHERAPY

Sickle cell disease gene therapy drug expenses and reimbursement: a litmus test for commercial pricing strategy and patient access for curative therapies

Article

Author: Galipeau, Jacques ; Hume, Ashley ; Grilley, Bambi J

Durable gene therapies newly approved by the Food and Drug Administration for sickle cell disease offer the promise of cure with subsequent improvement in quality of life through avoidance of repeated hospitalizations and worsening of disabilities in these patients. In the United States, following Food and Drug Administration approval, lovotibeglogene autotemcel (Lyfgenia, bluebird bio, Inc, Sommerville, MA, USA) and exagamglogene autotemcel (Casgevy, Vertex Pharmaceuticals Inc, Boston, MA, USA) were launched in 2024 for treatment of patients 12 years and older with frequent vaso-occlusive crises. Here we will discuss the first treatment with lovotibeglogene as a test case serving to highlight the multiple drug reimbursement pathways in the United States for cell and gene products for orphan disorders and their impact on patient adoption and access.

01 May 2025·ADVANCES IN THERAPY

Impact of Different Definitions of Vaso-Occlusion on Efficacy Assessments in Sickle Cell Disease Clinical Trials

Review

Author: Xuan, Fengjuan ; Imren, Suzan ; Eckrich, Michael J ; Grupp, Stephan A ; Li, Nanxin ; Locatelli, Franco ; Frangoul, Haydar

INTRODUCTION:

Patients with sickle cell disease (SCD) experience recurrent, severe pain events due to vaso-occlusion. Eliminating these acute pain events is a key outcome in SCD clinical trials; however, the definition of a vaso-occlusive crisis (VOC) or a vaso-occlusive event (VOE) has not been consistently applied, hampering comparisons of treatment efficacy between different therapeutic approaches. We have examined the degree to which differing definitions of vaso-occlusion in clinical trial endpoints impact efficacy outcomes.

METHODS:

Descriptions of clinical endpoints related to vaso-occlusion and pain events were reviewed from trials of exagamglogene autotemcel (exa-cel), lovotibeglogene autotemcel (lovo-cel), renizgamglogene autogedtemcel (reni-cel), hydroxyurea, L-glutamine, voxelotor, and crizanlizumab. Patient-level data from the published exa-cel Phase 3 pivotal trial (CLIMB SCD-121; data cut 14 Jun 2023) was used to evaluate efficacy outcomes based on differing endpoint definitions of vaso-occlusion.

RESULTS:

In the seven clinical trials reviewed, definitions of vaso-occlusion and/or pain events varied by care setting, duration of care, treatments used, and associated complications, with the frequency and duration of medical facility visits for acute pain events being most dissimilar between trials. Definitions of severe VOCs (exa-cel), VOC (voxelotor), and sickle cell-related pain crises (SCPCs; crizanlizumab and L-glutamine) included pain events requiring a medical facility visit of any duration, whereas the definition of painful crises (hydroxyurea) required a medical facility visit of > 4 h and the definition of severe vaso-occlusive events (VOEs; lovo-cel and reni-cel) required a hospital or emergency room (ER) observation unit visit lasting ≥ 24 h or ≥ 2 visits to a day unit or ER over a 72-h period. Based on the definition of severe VOCs, 29/30 patients [96.7%; 95% confidence interval (CI): 82.8, 99.9] in the CLIMB SCD-121 trial were considered free from severe VOCs for ≥ 12 consecutive months, whereas when the severe VOEs definition was applied to the same data, all patients (30/30; 100.0%; 95% CI: 88.4, 100.0) were considered free from severe VOEs for ≥ 12 consecutive months.

CONCLUSION:

Differences exist in definitions of vaso-occlusion and pain events used in SCD clinical trials. Severe VOCs (exa-cel), VOC (voxelotor), and SCPCs (crizanlizumab and L-glutamine) were more broadly inclusive than severe VOEs (lovo-cel and reni-cel) or painful crisis (hydroxyurea). Clinically, these differences resulted in differing numbers of patients being considered free from vaso-occlusion pain events, underscoring the challenge in comparing frequencies of pain events across SCD clinical trials.

450

News (Medical) associated with Exagamglogene Autotemcel

29 Jun 2025

·www.globenewswire.com

CRISPR Therapeutics Reports Positive Additional Phase 1 Data for CTX310™ Targeting ANGPTL3 and Provides Update on In Vivo Cardiovascular Pipeline

-New Phase 1 clinical data for CTX310™ continues to demonstrate dose-dependent reductions in triglycerides (TG) and low-density lipoprotein (LDL), with peak reduction of up to 82% in TG and up to 86% in LDL, with a well-tolerated safety profile- -Complete Phase 1 data presentation for CTX310 anticipated at a medical meeting in the second half of 2025- -Data update for CTX320™, targeting the LPA gene, now expected in the first half of 2026- -Preclinical in vivo cardiovascular program CTX340™ advancing toward IND / CTA filings targeting refractory hypertension- June 26, 2025 -- CRISPR Therapeutics (NASDAQ: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases today announced updates across its in vivo cardiovascular disease programs. These include new data for CTX310™, targeting ANGPTL3, as well as continued progress on CTX320™, targeting the LPA gene, and CTX340™, targeting the AGT gene. “CRISPR Therapeutics remains focused on executing against our strategic priorities and advancing our portfolio of innovative therapies,” said Samarth Kulkarni, Ph.D., Chairman and Chief Executive Officer of CRISPR Therapeutics. “The additional data from our ongoing Phase 1 clinical trial for CTX310 reinforces the potential of our platform to transform the treatment of serious cardiovascular diseases. We are progressing with our dose-finding study and expect to share complete data at a medical meeting in the second half of this year. For CTX320, we are continuing our dose-finding study and anticipate sharing data in the first half of 2026, reflecting a strategic decision to incorporate emerging insights from the evolving Lp(a) treatment landscape.” CTX310 targets ANGPTL3, a gene that encodes for key protein involved in the regulation of low-density lipoprotein (LDL) and triglyceride (TG) levels – both of which are recognized risk factors for atherosclerotic heart disease (ASCVD). Loss-of-function mutations in ANGPTL3 are associated with significantly reduced levels of LDL and TGs, as well as reduced risk of ASCVD, without known adverse health effects. More than 40 million patients in the U.S. alone are affected by elevated LDL, severely elevated TG or both – representing a significant unmet need and a large addressable population. CTX310 is initially focused on high-risk patients with the greatest unmet medical need and limited effective treatment options. CTX310 is in an ongoing Phase 1 first-in-human clinical trial targeting ANGPTL3 in four patient groups: homozygous familial hypercholesterolemia (HoFH), severe hypertriglyceridemia (sHTG), heterozygous familial hypercholesterolemia (HeFH), or mixed dyslipidemias (MDL). Eligible participants have levels of TG >300 mg/dL and/or LDL-C >100 mg/dL (or >70 mg/dL for subjects with ASCVD). Both LDL and TG are validated surrogate endpoints accepted by regulatory agencies. These new results build upon previously disclosed results from the first 10 patients across the first four cohorts (lean body weight-based doses of DL1 [0.1 mg/kg], DL2 [0.3 mg/kg], DL3 [0.6 mg/kg] and DL4 [0.8 mg/kg]) with at least 30 days of follow-up for each participant. As dose-range finding continues, data to date demonstrate peak reductions of up to 82% in TG and LDL reductions of up to 86% at DL4 without any clinically significant changes in liver enzymes and a safety and tolerability profile consistent with previous findings. The Company anticipates presenting the complete Phase 1 data for CTX310 at a medical meeting in the second half of 2025. CTX320 is in an ongoing Phase 1 clinical trial targeting the LPA gene in patients with elevated lipoprotein(a) [Lp(a)], a genetically determined risk factor associated with increased incidence of major adverse cardiovascular events (MACE). Elevated Lp(a) levels affect up to 20% of the global population and remains unaddressed by current therapies. The Phase 1 trial is enrolling patients and dose-finding is ongoing. An update is now expected in the first half of 2026, reflecting a strategic decision to incorporate emerging insights from the evolving Lp(a) landscape. CRISPR Therapeutics is also advancing its preclinical in vivo cardiovascular program CTX340, targeting angiotensinogen (AGT) for the treatment of refractory hypertension. CTX340 is currently progressing through IND/CTA-enabling studies. CRISPR Therapeutics has established a proprietary lipid nanoparticle (LNP) platform for the delivery of CRISPR/Cas9 to the liver. The Company’s in vivo portfolio includes its lead investigational programs, CTX310 (directed towards angiopoietin-related protein 3 (ANGPTL3)) and CTX320 (directed towards LPA, the gene encoding apolipoprotein(a) (apo(a)), a major component of lipoprotein(a) [Lp(a)]). Both are validated therapeutic targets for cardiovascular disease. CTX310 and CTX320 are in ongoing clinical trials in patients with heterozygous familial hypercholesterolemia, homozygous familial hypercholesterolemia, mixed dyslipidemias, or severe hypertriglyceridemia, and in patients with elevated lipoprotein(a), respectively. In addition, the Company’s research and preclinical development candidates include CTX340 and CTX450™, targeting angiotensinogen (AGT) for refractory hypertension and 5’-aminolevulinate synthase 1 (ALAS1) for acute hepatic porphyria (AHP), respectively. Since its inception over a decade ago, CRISPR Therapeutics has evolved from a research-stage company advancing gene editing programs into a leader that celebrated the historic approval of the first-ever CRISPR-based therapy. The Company has a diverse portfolio of product candidates across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine, cardiovascular, autoimmune, and rare diseases. In 2018, CRISPR Therapeutics advanced the first-ever CRISPR/Cas9 gene-edited therapy into the clinic to investigate the treatment of sickle cell disease and transfusion-dependent beta thalassemia. Beginning in late 2023, CASGEVY® (exagamglogene autotemcel [exa-cel]) was approved in several countries to treat eligible patients with either of these conditions. The Nobel Prize-winning CRISPR technology has revolutionized biomedical research and represents a powerful, clinically validated approach with the potential to create a new class of potentially transformative medicines. To accelerate and expand its efforts, CRISPR Therapeutics has formed strategic partnerships with leading companies including Vertex Pharmaceuticals. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Boston, Massachusetts and San Francisco, California. CRISPR THERAPEUTICS® standard character mark and design logo CTX310™, CTX320™, CTX340™ and CTX450™ are trademarks and registered trademarks of CRISPR Therapeutics AG. CASGEVY® and the CASGEVY logo are registered trademarks of Vertex Pharmaceuticals Incorporated. All other trademarks and registered trademarks are the property of their respective owners. The content above comes from the network. if any infringement, please contact us to modify.

Drug ApprovalGene Therapy

23 Jun 2025

·www.fiercebiotech.com

Vertex\'s remaining islet cell therapy reduces dangerously low blood sugar levels in phase 1/2 test

At the American Diabetes Association conference in Chicago last week, Vertex Pharmaceuticals shared what it claimed were “unprecedented” data from 12 patients in the phase 1/2 portion of the study who received the full infusion of zimislecel and were followed up for a year.\n Vertex Pharmaceuticals may have narrowed its focus this year, but the biopharma’s decision to stick with its remaining islet cell therapy appears to be paying off so far.The allogeneic human stem cell–derived islet cell therapy, called zimislecel, is being evaluated in an ongoing phase 1/2/3 study in patients with Type 1 diabetes with impaired hypoglycemic awareness and who suffer from severe hypoglycemic events, a term for dangerously low blood sugar levels. At the American Diabetes Association conference in Chicago last week, Vertex shared what it claimed were “unprecedented” data from 12 patients in the phase 1/2 portion of the study who received the full infusion of zimislecel and were followed up for a year.The stem-cell-derived islets were engrafted successfully to all 12 patients, producing glucose-responsive endogenous C-peptide up to the one-year follow-up, Vertex said.None of the patients experienced any severe hypoglycemic events from day 90 onward, with glycated hemoglobin (HbA1c) levels—a measure of diabetes control—at the recommended level of less than 7%, hitting the primary endpoint of the phase 1/2 portion of the study.Meanwhile, 10 of the 12 patients no longer required insulin replacement therapy by the 12-month checkup, Vertex noted.While two patients involved in the trial died, Vertex stressed that these fatalities were “both unrelated to treatment with zimislecel.” The treatment was “generally well tolerated,” the company added, with no serious adverse events linked to the therapy itself.“These data on the first fully differentiated, stem cell-derived, off-the-shelf islet cell therapy continue to be unprecedented,” Vertex Chief Medical Officer Carmen Bozic, M.D., said in a June 20 release.“The magnitude, consistency and durability of the results from all 12 patients with more than one year of follow-up reinforce the transformative potential of zimislecel for people living with T1D complicated by severe hypoglycemia,” Bozic added. “We are excited to complete enrollment and dosing in the phase 1/2/3 program and look forward to potential regulatory submissions next year.” Zimislecel is Vertex’s only remaining clinical-stage islet cell treatment in development after the company ended work on VX-264 last month. A 90-day analysis of a phase 1/2 study of VX-264—which involved creating insulin-producing cells and surgically implanting them into people with Type 1 diabetes—had not produced the levels of a key biomarker for insulin production that had been hoped for.At the time, William Blair analysts described the discontinuation of VX-264 as “disappointing given that this would have expanded Vertex’s type 1 diabetes market significantly from that being addressed from zimislecel.”Unlike VX-264, zimislecel is administered along with immunosuppressants to help the cells graft. The phase 3 portion of the zimislecel study is running in the U.S., Canada, the U.K. and the EU, with approval applications to regulators penciled in for next year.Scrapping VX-264 was only part of Vertex’s wider efforts this year to streamline its pipeline. The biopharma—which is in the process of launching its approved sickle cell disease and beta thalassemia gene therapy Casgevy—has also recently shifted away from adeno-associated virus vectors as a delivery mechanism for its genetic medicines.

$Vertex\'s remaining islet cell therapy reduces dangerously low blood sugar levels in phase 1/2 test$

Phase 3Cell TherapyGene TherapyClinical ResultClinical Study

12 Jun 2025

·investors.vrtx.com

Vertex Presents Longer-Term Data at the 2025 European Hematology Association (EHA) Congress Demonstrating Durability of CASGEVY® and Provides Update on Expanding Global Access to CASGEVY

- Data from longer-term follow-up of patients in clinical trials further demonstrate durability of the transformative benefits of CASGEVY® - - Multiple reimbursement agreements secured, expanding access to CASGEVY to more patients around the world - LONDON - June 12, 2025 - Vertex Pharmaceuticals (Nasdaq: VRTX) today announced positive longer-term data for CASGEVY® (exagamglogene autotemcel) from global clinical trials in people with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT). The results, presented at the European Hematology Association (EHA) Congress, continue to demonstrate the transformative, durable clinical benefits of CASGEVY. The longest follow up in SCD patients now extends more than 5.5 years and in TDT patients more than 6 years, with a mean of 39.4 months and 43.5 months, respectively. CASGEVY is the first and only approved CRISPR/Cas9 gene-edited therapy. “These longer-term data further confirm that CASGEVY can provide significant and durable clinical benefits to eligible people living with sickle cell disease or transfusion-dependent beta thalassemia,” said Franco Locatelli, M.D., Ph.D., Professor of Pediatrics at the Catholic University of the Sacred Heart of Rome, Director of the Department of Pediatric Hematology and Oncology at Bambino Gesù Children’s Hospital, Chair of Vertex’s TDT Program Steering Committee, and Presenting Author of the CASGEVY TDT clinical data at EHA. “Given the urgent and unmet need for new transformative treatments for these diseases, I am delighted by the strong progress that has been made to bring CASGEVY to eligible patients in the real world.” New longer-term follow-up data presented from the CASGEVY trials In SCD, 43/45 (95.6%) evaluable patients (those with at least 16 months of follow-up) were free from vaso-occlusive crises (VOCs) for at least 12 consecutive months (VF12) in CLIMB-121 and CLIMB-131 combined (95% CI: 84.9%, 99.5%). The mean duration of VOC-free was 35.0 months (range 14.4 to 66.2 months). All evaluable patients (45/45 [100%]) achieved freedom from in-patient hospitalization for severe VOCs for at least 12 consecutive months (HF12) in CLIMB-121 and CLIMB-131 combined (95% CI: 92.1%, 100%), with a mean hospitalization-free of 36.1 months (range 14.5 to 66.2 months). In TDT, 54/55 (98.2%) evaluable patients (those with at least 16 months of follow-up) achieved transfusion-independence for at least 12 consecutive months with a weighted average hemoglobin (Hb) of at least 9 g/dL (TI12) in CLIMB-111 and CLIMB-131 combined (95% CI: 90.3%, 100%). The mean duration of transfusion independence was 40.5 months (range 13.6 to 70.8 months). The one evaluable patient who did not achieve TI12 has been transfusion free for 14.8 months. Iron removal therapy has been stopped for more than 6 months in 39/56 (69.6%) treated patients following infusion with CASGEVY, with sustained improvement in ferritin and liver iron content, suggesting that CASGEVY has the potential to correct ineffective erythropoiesis. Patients continue to demonstrate stable levels of fetal hemoglobin (HbF) and allelic editing. The safety profile of CASGEVY continues to be generally consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant. All evaluable patients (45/45 [100%]) achieved freedom from in-patient hospitalization for severe VOCs for at least 12 consecutive months (HF12) in CLIMB-121 and CLIMB-131 combined (95% CI: 92.1%, 100%), with a mean hospitalization-free of 36.1 months (range 14.5 to 66.2 months). The one evaluable patient who did not achieve TI12 has been transfusion free for 14.8 months. Iron removal therapy has been stopped for more than 6 months in 39/56 (69.6%) treated patients following infusion with CASGEVY, with sustained improvement in ferritin and liver iron content, suggesting that CASGEVY has the potential to correct ineffective erythropoiesis. Progress in bringing CASGEVY to patients Through reimbursement agreements, Vertex has secured access for eligible SCD or TDT patients in multiple countries including Austria, Bahrain, England, the Kingdom of Saudi Arabia, Northern Ireland, Scotland, the United Arab Emirates, the United States and Wales. Vertex is continuing to work with government and reimbursement authorities globally to secure sustainable access for additional eligible patients. About Sickle Cell Disease (SCD) SCD is a debilitating, progressive, life-shortening genetic disease. SCD patients report health-related quality of life scores well below the general population and significant health care resource utilization. SCD affects the red blood cells, which are essential for carrying oxygen to all organs and tissues of the body. SCD causes severe pain, organ damage and shortened life span due to misshapen or “sickled” red blood cells. The clinical hallmark of SCD is vaso-occlusive crises (VOCs), which are caused by blockages of blood vessels by sickled red blood cells and result in severe and debilitating pain that can happen anywhere in the body at any time. SCD requires lifelong treatment and significant use of health care resources, and ultimately results in reduced life expectancy, decreased quality of life and reduced lifetime earnings and productivity. In Europe, the mean age of death for patients living with SCD is around 40 years. About Transfusion-Dependent Beta Thalassemia (TDT) TDT is a serious, life-threatening genetic disease. TDT patients report health-related quality of life scores below the general population and significant health care resource utilization. TDT requires frequent blood transfusions and iron chelation therapy throughout a person’s life. Due to anemia, patients living with TDT may experience fatigue and shortness of breath, and infants may develop failure to thrive, jaundice and feeding problems. Complications of TDT can also include an enlarged spleen, liver and/or heart, misshapen bones and delayed puberty. TDT requires lifelong treatment and significant use of health care resources, and ultimately results in reduced life expectancy, decreased quality of life and reduced lifetime earnings and productivity. In Europe, the mean age of death for patients living with TDT is 50-55 years. About CASGEVY® (exagamglogene autotemcel) CASGEVY® is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene through a precise double-strand break. This edit results in the production of high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. CASGEVY has been shown to reduce or eliminate VOCs for patients with SCD and transfusion requirements for patients with TDT. CASGEVY is approved for eligible SCD and TDT patients 12 years and older by multiple regulatory bodies around the world. About the CLIMB Trials The ongoing Phase 1/2/3 open-label trials, CLIMB-111 and CLIMB-121, are designed to assess the safety and efficacy of a single dose of CASGEVY in patients ages 12 to 35 years with TDT or with SCD and recurrent VOCs. The trials are closed for enrollment. Patients will be followed for approximately two years after CASGEVY infusion in these trials. Each patient will be asked to participate in the ongoing long-term, open-label trial, CLIMB-131. CLIMB-131 is designed to evaluate the long-term safety and efficacy of CASGEVY in patients who received CASGEVY, including those in other CLIMB trials. The trial is designed to follow patients for up to 15 years after CASGEVY infusion. About Vertex Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and conditions. The company has approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia and acute pain, and it continues to advance clinical and research programs in these areas. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes and myotonic dystrophy type 1. Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 15 consecutive years on Science magazine's Top Employers list and one of Fortune’s 100 Best Companies to Work For. For company updates and to learn more about Vertex’s history of innovation, visit www.vrtx.com/en-global. Vertex Special Note Regarding Forward-Looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, the statements by Franco Locatelli, M.D., Ph.D., in this press release, and statements regarding expectations for the anticipated transformative, durable clinical benefits of CASGEVY, plans to continue working with government and reimbursement authorities globally to secure sustainable access for patients, and our plans for and design of the CLIMB studies. While we believe the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that eligible patient access to CASGEVY may not be achieved on the anticipated timeline, or at all, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy, and other reasons, and other risks listed under the heading “Risk Factors” in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at www.sec.gov and available through the company's website at www.vrtx.com. You should not place undue reliance on these statements, or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available. Vertex Pharmaceuticals IncorporatedInvestors:InvestorInfo@vrtx.com Media:mediainfo@vrtx.comorInternational: +44 20 3204 5275orU.S.: 617-341-6992

Clinical ResultClinical StudyDrug ApprovalCell Therapy

100 Deals associated with Exagamglogene Autotemcel

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R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Anemia, Sickle Cell	United Kingdom	Vertex Pharmaceuticals (Europe) Ltd. (Ireland)	15 Nov 2023
Transfusion-dependent Beta Thalassemia	United Kingdom	Vertex Pharmaceuticals (Europe) Ltd. (Ireland)	15 Nov 2023

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Transfusion-dependent Thalassemia	Phase 3	United States	Vertex Pharmaceuticals, Inc.	03 May 2022
Transfusion-dependent Thalassemia	Phase 3	United States	CRISPR Therapeutics AG	03 May 2022
Transfusion-dependent Thalassemia	Phase 3	Canada	CRISPR Therapeutics AG	03 May 2022
Transfusion-dependent Thalassemia	Phase 3	Canada	Vertex Pharmaceuticals, Inc.	03 May 2022
Transfusion-dependent Thalassemia	Phase 3	Germany	Vertex Pharmaceuticals, Inc.	03 May 2022
Transfusion-dependent Thalassemia	Phase 3	Germany	CRISPR Therapeutics AG	03 May 2022
Transfusion-dependent Thalassemia	Phase 3	Italy	CRISPR Therapeutics AG	03 May 2022
Transfusion-dependent Thalassemia	Phase 3	Italy	Vertex Pharmaceuticals, Inc.	03 May 2022
Transfusion-dependent Thalassemia	Phase 3	United Kingdom	Vertex Pharmaceuticals, Inc.	03 May 2022
Transfusion-dependent Thalassemia	Phase 3	United Kingdom	CRISPR Therapeutics AG	03 May 2022

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


CLIMB THAL-111 and CLIMB-131 (EHA2025)	Phase 3	Transfusion-dependent Thalassemia	56	Exagamglogene autotemcel	vzwqvljlxi(kozsnsrxsk) = gmkxnyuivk jvynnffycc (rjnozdgqed ) View more	Positive	14 May 2025
CLIMB SCD-121 (EHA2025)	Phase 3	Vaso-occlusive crisis	58	Exagamglogene autotemcel	dqqbqdtgqq(clotmxgejf) = vrgwvvbqkd evgvizatwk (usagoeztvx ) View more	Positive	14 May 2025
ASH2024	Not Applicable	Anemia, Sickle Cell	-	Exagamglogene autotemcel	xoybyizyqo(mozuogctvr) = 52.2% kewrydoyfy (hgtpqscfci ) View more	-	09 Dec 2024
ASH2024	Not Applicable	Transfusion-dependent Thalassemia	-	Exagamglogene autotemcel	zaqpwevdjf(gjjrbktaqj) = 60.7% xevopclikz (mghhitkekt ) View more	-	08 Dec 2024
NEWS Manual	Not Applicable	Transfusion-dependent Beta Thalassemia \| Anemia, Sickle Cell	-	Casgevy (SCD)	glerhyezay(ntwrssdzeu) = tkcipbxogv vguoussesp (bvaxmscbui ) View more	Positive	15 Jun 2024
NEWS Manual	Not Applicable		-	Casgevy (TDT)	eyezkrlxcx(zohoxawzbv) = uvcyxompal tbymbghajj (yvkvuicsdm ) View more	Positive	15 Jun 2024
NCT03655678 (CTX001-111 \| CLIMB THAL-111, EHA2024) Manual	Phase 3	Transfusion-dependent Thalassemia	24	Exagamglogene autotemcel	apvaycdtcb(bgsjfblhrx) = eceasoeryd jihoyfbdde (sqcireruco, 0.21) View more	Positive	14 May 2024
NCT03745287 (CLIMB SCD-121, EHA 12024 \| EHA 22024) Manual	Phase 3	Anemia, Sickle Cell	24	exa-cel	djypfigurp(nfaujjewel) = By month 6, EQ-5D-5L health utility US index score and EQ VAS score showed substantial improvements,which were maintained through month 24 (change at month 24 [n=17]: 0.13 [0.19]; minimal clinically important difference [MCID] 0.078 and 26.9 [22.6]; MCID 7 to 10, respectively). uzgwfzevuy (panypkwzgg ) View more	Positive	14 May 2024
NCT03745287 (CLIMB SCD-121, EHA 12024 \| EHA 22024) Manual	Phase 3	Anemia, Sickle Cell	46	Exagamglogene autotemcel	hsbmsoafpz(rhyjrjmcxq) = mdaputiwnc zmvyuoxexa (wkicvrytel, 79 - 99) View more	Positive	14 May 2024
NCT03655678 (CTX001-111, FDA) Manual	Phase 2/3	Transfusion-dependent Beta Thalassemia	52	CASGEVY	layafrtrom(yxnixosttg) = xflsyjtikt tlcvvhcozt (anxkxgfrkv, 75.7 - 100) View more	Positive	07 Feb 2024
NCT03655678 (CLIMB THAL-111, Tandem Meetings ASTCT and CIBMTR2024) Manual	Phase 3	Transfusion-dependent Beta Thalassemia	44	Exagamglogene autotemcel	hfvpuhquqa(vulrtaprpj) = eurecedaan zurjtsdeeh (ybvporwfci )	Positive	01 Feb 2024

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