RegulationPriority Review (US), Fast Track (US), Rare Pediatric Disease (US), Orphan Drug (EU), PRIME (EU), Regenerative Medicine Advanced Therapy (US), Orphan Drug (GB), Priority Review (CA), Conditional marketing approval (GB), Orphan Drug (US)

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 Date01 Apr 2024

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

CTIS2024-514641-12-00 / Recruiting

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 ß-Thalassemia or Severe Sickle Cell Disease - VX21-CTX001-161

Start Date14 Nov 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 Dec 2024·CLINICAL PHARMACOLOGY & THERAPEUTICS

Infrequent Resolution of Vaso‐Occlusive Crises in Routine Clinical Care Among Patients Mimicking the Exa‐Cel Trial Population: A Cohort Study of Medicaid Enrollees

Article

Author: Desai, Rishi J. ; Beukelman, Timothy ; Titievsky, Lina ; Mahesri, Mufaddal ; Zhang, Lanju ; Lee, Su Been ; Imren, Suzan ; Levin, Raisa

The CRISPR‐based gene editing therapy exagamglogene autotemcel (exa‐cel) recently received FDA approval for patients with severe sickle cell disease (SCD). The approval was based on a phase III trial (CLIMB SCD 121), which showed 97% efficacy of this treatment in eliminating vaso occlusive crises (VOCs) for 12 consecutive months. To help contextualize results from this trial, we aimed to investigate the proportion of patients with severe SCD who remain VOC‐free for a 1‐year period in routine clinical care. Using Medicaid claims data (2000–2018), we identified a cohort of patients, 12–35 years old with severe SCD, defined by ≥ 2 VOCs per year for 2 consecutive years, who met other exa‐cel trial inclusion criteria to mimic a trial‐like population. A VOC was identified using ICD diagnosis codes during hospitalization and ER visits. The primary outcome was the proportion of patients with no VOCs during a 1‐year follow‐up. A total of 7,425 patients with severe SCD [mean (SD) age: 20.5 (6.0) years, 54.6% females, 84% African Americans], had a mean of 5.2 VOCs, 5.1 ER visits and 3.5 hospitalizations per year during the baseline period. The proportion of patients with no VOCs during the 1‐year follow‐up was 7.7% (95% confidence interval: 7.1%–8.3%). In conclusion, less than one in 12 patients with severe SCD achieved VOC‐free status within 1 year in routine clinical care. These findings suggest that the high efficacy observed for exa‐cel in the trial, if replicated in routine clinical care, could translate into a significant public health impact.

01 Nov 2024·Journal of Genetics and Genomics

Gene therapy and gene editing strategies in inherited blood disorders

Review

Author: Song, Xuemei ; Zheng, Tingfeng ; Guo, Xiang ; Wang, Xiaolong ; Liu, JinLei ; Chen, Tangcong

Gene therapy has shown significant potential in treating various diseases, particularly inherited blood disorders such as hemophilia, sickle cell disease, and thalassemia. Advances in understanding the regulatory network of disease-associated genes have led to the identification of additional therapeutic targets for treatment, especially for β-hemoglobinopathies. Erythroid regulatory factor BCL11A offers the most promising therapeutic target for β-hemoglobinopathies, and reduction of its expression using the commercialized gene therapy product Casgevy has been approved for use in the UK and USA in 2023. Notably, the emergence of innovative gene editing technologies has further broadened the gene therapy landscape, presenting possibilities for treatment. Intensive studies indicate that base editing and prime editing, built upon CRISPR technology, enable precise single-base modification in hematopoietic stem cells for addressing inherited blood disorders ex vivo and in vivo. In this review, we present an overview of the current landscape of gene therapies, focusing on clinical research and gene therapy products for inherited blood disorders, evaluation of potential gene targets, and the gene editing tools employed in current gene therapy practices, which provides an insight for the establishment of safer and more effective gene therapy methods for a wider range of diseases in the future.

01 Oct 2024·CRISPR Journal

Five Years of Progress in CRISPR Clinical Trials (2019–2024)

Article

Author: Davies, Kevin ; Barrangou, Rodolphe ; Philippidis, Alex

In July 2019, Victoria Gray became the first patient with sickle cell disease to receive a CRISPR-based cell therapy as a volunteer in the exa-cel clinical trial, sponsored by Vertex Pharmaceuticals and CRISPR Therapeutics. Barely four years later, the ensuing therapy, branded as Casgevy, received approval from regulatory agencies in Europe, the United States, and the Middle East, ushering in a new era of CRISPR-based medicines. During this period, scores of other clinical trials have been launched, including many actively recruiting patients across phase 1, phase 2, and phase 3 clinical trials around the world. In this brief Perspective, we collate the latest information on therapeutic clinical trials featuring CRISPR, base and prime editing, across a range of both in vivo and ex vivo gene and cell therapies.

318

News (Medical) associated with Exagamglogene Autotemcel

20 Nov 2024

·www.prnewswire.com

The Gene Revolution: Transforming Lives through Gene Editing Therapeutics

"The Gene Editing Therapeutics Market is all about developing advanced treatments that can directly change genes to treat or cure diseases. It's like fixing the root cause of a health issue at the DNA level, offering hope for conditions that were once thought to be untreatable. This market is rapidly growing as these innovative therapies become more widely researched and used." BOSTON, Nov. 20, 2024 /PRNewswire/ -- "According to the latest study from BCC Research, the demand for Gene Editing Therapeutics is estimated at $11 million in 2024 and is forecast to grow at a CAGR of 147% to reach $1.0 billion by the end of 2029. This report takes a close look at the current and future possibilities of gene editing treatments and explores the factors that will help or hinder their use. It also examines the competitive landscape, including what therapeutics companies are working on and how they plan to bring new treatments to market. Additionally, it predicts how the market will evolve by 2029 and provides in-depth profiles of key players, including their business focus, financials, and products. Interesting Facts about the Gene Editing Therapeutics Market The gene editing therapeutics market is just getting started, with the first approved treatment, Casgevy, coming to market in the U.K. and U.S. at the end of 2023. This groundbreaking treatment, developed by CRISPR Therapeutics and Vertex, uses CRISPR/Cas9 technology to edit genes. Casgevy's reach is expected to increase as it is set to become available for children between the ages of 5-12. While most new treatments are still in early development, two promising ones are close to a 2029 market launch: NTLA-2001 and cema-cel. There are challenges. Gene therapies are expensive and difficult to manufacture. To address this, some companies are outsourcing production and some are trying to secure government reimbursement for these life-changing treatments for rare diseases. To obtain more insights and information about the gene editing therapeutics market, click here. Factors contributing to this market's growth include: Increasing Prevalence of Chronic Diseases. More and more people are living with long-term health conditions, such as heart disease, diabetes and cancer. This trend is partly due to the world's aging population. In addition, these diseases often require ongoing treatment. Increasing Prevalence of Rare Genetic Disorders. More people are being diagnosed with uncommon, inherited health conditions caused by changes in their genes. As medical knowledge and genetic testing improve, researchers will become better at identifying these rare disorders, leading to more cases being recognized and treated. Precision Medicine.: This is a tailored approach to healthcare that focuses on treating individuals based on their unique genetic makeup, lifestyle, and environment. Instead of a one-size-fits-all method. Precision medicine provides the most effective treatment for each person, ensuring better outcomes by considering what makes each patient unique. Request a Sample Copy of the Gene Editing Therapeutics Market Report Synopsis This report addresses the following questions: What are the projections for the market? The gene editing therapeutics market is estimated at $11 million in 2024 and is forecast to grow at a CAGR of 147% to reach $1.0 billion by the end of 2029. What factors are driving the growth of the market? - Increasing prevalence of chronic diseases and rare genetic disorders. - Need for precision medicine What segments are covered in the market? Because only one product has been approved to date, with other products still under development, the market is segmented only by region. Which region has the highest market share? North America holds the highest share in the market and will continue to do so through the forecast period. Market leaders include: Allogene Therapeutics Beam Therapeutics Caribou Biosciences Inc. Crispr Therapeutics Editas Medicine Intellia Therapeutics Inc. Precision Biosciences Sangamo Therapeutics Vertex Pharmaceuticals Inc. Verve Therapeutics Inc. Vor Bio More related reports: Global Market for Cell and Gene Therapy: The worldwide industry that develops and sells treatments that use cells or genes to repair or replace damaged or diseased cells can be thought of like a repair shop for the body, where cells or genes are used to fix or replace faulty ones, helping to cure diseases and improve health. This market includes companies working on these innovative treatments. Directly Purchase a copy of the report from BCC Research. For further information or to make a purchase, please get in touch with [email protected]. About BCC Research BCC Research market reports provide objective, unbiased measurement and assessment of market opportunities. Our experienced industry analysts' goal is to help readers make informed business decisions, free of noise and hype. Contact Us Corporate HQ: 50 Milk St. Ste 16, Boston, MA 02109, USA Email: [email protected], Phone: +1 781-489-7301 For media inquiries, email [email protected] or visit our media page for access to our market research library. Any data and analysis extracted from this press release must be accompanied by a statement identifying BCC Research LLC as the source and publisher. Logo - SOURCE BCC Research LLC WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Drug ApprovalGene Therapy

18 Nov 2024

·www.biopharmadive.com

CRISPR therapy from Intellia may ameliorate rare heart disorder, data suggest

Dive Brief:A single infusion of a CRISPR therapy developed by Intellia Therapeutics showed promising signs of stabilizing a heart disorder caused by the rare disease transthyretin amyloidosis, buoying the companys hopes of finding success in late-stage clinical trials.Phase 1 study data from 36 people with the cardiomyopathy form of transthyretin, or ATTR, amyloidosis showed Intellias gene editing treatment sharply and durably lowered levels of the ATTR protein that misfolds and gathers in the toxic clumps that characterize the disease.Prior trial results, in fewer people and across shorter periods of time, had already shown Intellias therapy capable of reducing ATTR protein. The new findings, which were published Friday in The New England Journal of Medicine, show those reductions appeared to translate to stability or improvement on several markers of cardiac disease progression, too.Dive Insight:Intellia Therapeutics has, along with several of its peers, been at the forefront of the biotechnology industrys efforts to translate breakthrough CRISPR science into real medicines. CRISPR Therapeutics reached that milestone first with Casgevy, a sickle cell disease therapy thats built from gene edited stem cells.But Intellia has been the standard bearer for whats known as in vivo CRISPR editing. Rather than use CRISPR to create a medicine from, say, a patients own cells, in vivo editing means the CRISPR components are themselves the therapy, delivered directly into the body where they do their DNA-modifying work.Intellias amyloidosis medicine, now dubbed nex-z, works by using CRISPR to knock out a gene thats responsible for making the TTR protein in the liver. Its meant to be a one-time treatment that permanently turns off TTR protein production, thereby preventing further damage to the heart and nerves.The data published in NEJM Friday are the best look yet at nex-zs potential and offer encouraging signs. Nex-z reduced TTR protein levels by 90%, compared to baseline, at one year. Among the 11 patients who have been followed for two years, TTR levels remained low and virtually unchanged, the study authors wrote.Importantly, that reduction led to stabilization or improvement on three measures of heart disease progression and heart failure classification. On the last measure, for instance, 47% of participants improved by at least one class on a New York Heart Association rating scale. Only 8% of participants experienced a worsening of their NYHA class.Five patients had infusion-related reactions to nex-z, while two experienced increases in liver enzyme counts that resolved without medical intervention, according to the NEJM paper. One of the infusion reactions was judged to be severe by investigators and led to a overnight stay in the hospital for observation.While this current study didnt involve a placebo, Intellia is running a Phase 3 study in about 750 people with cardiomyopathy due to ATTR amyloidosis that will test nex-z against placebo on cardiovascular mortality and heart events.Success in that trial could position Intellia to seek regulatory approval. But even then, nex-z would be entering a market with potentially three different drugs already cleared. Pfizers Vyndamax has been on the market for several years, while BridgeBio Pharma and Alnylam Pharmaceuticals aim to win Food and Drug Administration OKs for their rival medicines this month and next year, respectively.All of those drugs need to be taken chronically, which could favor nex-z and its one-time infusion. But their comparative efficacy is already a matter of serious debate, as doctors weigh the drugs respective data. Nex-z would be no different.I think people will need to know what is the most efficacious, which actually prevents not only heart failure, but any other adverse cardiovascular events associated with amyloid deposition in the heart and, in fact, which is the most durable over time, said Jane Leopold, NEJMs deputy editor, in an audio interview on Intellias data.Shares in Intellia oscillated in Monday morning trading. '

Clinical ResultAHAPhase 1Phase 3Gene Therapy

14 Nov 2024

·endpts.com

As bluebird projects more commercial demand, the biotech continues to seek cash

Bluebird reported that 17 people with sickle cell disease have started the process of receiving its gene therapy Lyfgenia. Bluebird said in its third-quarter earnings Thursday that it recognized revenue from Lyfgenia, though it did not specify how much. In total, the gene therapy biotech reported $10.6 million in revenue, but it said it expects at least double that — $25 million — next quarter. The company dosed its first sickle cell patient with Lyfgenia, the New York Times reported in September. Vertex Pharmaceuticals, which markets the gene-edited sickle cell and thalassemia therapy Casgevy, reported that it dosed its first patient in the third quarter and recorded $2 million in revenue. That patient was a 13-year-old thalassemia patient from Saudi Arabia , Vertex confirmed to Endpoints News via email. On Thursday, bluebird also said that 35 people have started the process of receiving Zynteglo, a similar therapy for thalassemia. It reported five patient starts for Skysona, a gene therapy for cerebral adrenoleukodystrophy. The biotech added it has 17 additional starts across its gene therapy portfolio scheduled for the rest of the year, along with 30 scheduled in 2025. “Patient starts more than doubled from our second to third quarter update, providing clear evidence that our commercial launches continued to accelerate,” bluebird CEO Andrew Obenshain said in a press statement. “We remain focused on securing additional cash resources to extend our runway, which we believe would enable us to achieve this vision and reach cash flow break-even in the second half of 2025.” Bluebird announced in September that it was laying off 25% of its workforce and cutting expenses by 20%. Earlier in the year, the biotech had to rework the terms of a loan with Hercules capital, and it currently has cash to fund operations into the first quarter of 2025. When asked in an analyst call about how the company was going to get more cash, CFO James Sterling said the “strategy to extend the runway includes renegotiating key contracts and other cost initiatives, and our partnership with Hercules is key in helping move those forward.” Bluebird is also at risk of being delisted from Nasdaq and is trying to implement a reverse stock split to avoid that. It’s convening a stockholder meeting on Dec. 4 to vote on the reverse stock split.

Gene Therapy

100 Deals associated with Exagamglogene Autotemcel

External Link

KEGG	Wiki	ATC	Drug Bank
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R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Anemia, Sickle Cell	GB	Vertex Pharmaceuticals (Europe) Ltd. (Ireland)	15 Nov 2023
Transfusion-dependent Beta Thalassemia	GB	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	US	Vertex Pharmaceuticals, Inc.	03 May 2022
Transfusion-dependent Thalassemia	Phase 3	US	CRISPR Therapeutics AG	03 May 2022
Transfusion-dependent Thalassemia	Phase 3	CA	Vertex Pharmaceuticals, Inc.	03 May 2022
Transfusion-dependent Thalassemia	Phase 3	CA	CRISPR Therapeutics AG	03 May 2022
Transfusion-dependent Thalassemia	Phase 3	DE	Vertex Pharmaceuticals, Inc.	03 May 2022
Transfusion-dependent Thalassemia	Phase 3	DE	CRISPR Therapeutics AG	03 May 2022
Transfusion-dependent Thalassemia	Phase 3	IT	CRISPR Therapeutics AG	03 May 2022
Transfusion-dependent Thalassemia	Phase 3	IT	Vertex Pharmaceuticals, Inc.	03 May 2022
Transfusion-dependent Thalassemia	Phase 3	GB	Vertex Pharmaceuticals, Inc.	03 May 2022
Transfusion-dependent Thalassemia	Phase 3	GB	CRISPR Therapeutics AG	03 May 2022

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ASH2024	Not Applicable	Anemia, Sickle Cell	-	Exagamglogene autotemcel	fnolynqlmk(yqigdihvus) = 52.2% hbjabdlino (lgasrxevfu ) View more	-	09 Dec 2024
ASH2024	Not Applicable	Transfusion-dependent Thalassemia	-	Exagamglogene autotemcel	ufeacvtlqb(ehhaxuhmig) = 60.7% csmifjouws (fegmwkbgib ) View more	-	08 Dec 2024
NEWS Manual	Not Applicable	Transfusion-dependent Beta Thalassemia \| Anemia, Sickle Cell	-	Casgevy (SCD)	dafzgtgaip(lfwmmqtlrv) = ricrdhaklm fsgdklmusy (hjcidbwpai ) View more	Positive	15 Jun 2024
NEWS Manual	Not Applicable		-	Casgevy (TDT)	hgnyixjqji(isdcgzspkg) = nvaqfcpgdq licgmwruyh (zwzxtrvzck ) View more	Positive	15 Jun 2024
NCT03745287 (CLIMB SCD-121, EHA2024) Manual	Phase 3	Anemia, Sickle Cell	46	Exagamglogene autotemcel	mnlzsctpuu(nkezlufpki) = hqrbinvrzl wogrxphrms (mocosbribe, 79 - 99) View more	Positive	14 May 2024
NCT03745287 (CLIMB SCD-121, EHA2024) Manual	Phase 3	Anemia, Sickle Cell	24	exa-cel	uwlmcnujpj(hjvozjstkx) = 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). nqrjtvsrlh (hxbuyxoqxn ) View more	Positive	14 May 2024
NCT03655678 (CTX001-111 \| CLIMB THAL-111, EHA2024) Manual	Phase 3	Transfusion-dependent Thalassemia	24	Exagamglogene autotemcel	mqyadafagg(tixveotyce) = hwrgdjlhom rwwgglenqx (ribsbooorm, 0.21) View more	Positive	14 May 2024
NCT03655678 (CTX001-111, FDA) Manual	Phase 2/3	Transfusion-dependent Beta Thalassemia	52	CASGEVY	uadxwgakyj(cpwfkngnrt) = mmoiyifban nicvmlhhpc (phjhlqafrx, 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	dkubglctpz(zvzzwiurmj) = zbgzphahsr jqtrhroshz (tbebijtojv )	Positive	01 Feb 2024
CLIMB SCD-121 (ASH2023)	Phase 3	Anemia, Sickle Cell	42	Exagamglogene autotemcel	ewzsxcrefg(zuokdyihls) = oncwnhlopa ttlfgicoub (ifrjtgnqib, 75.1% - 99.9%) View more	Positive	11 Dec 2023
CLIMB THAL-111 (ASH2023)	Phase 3	Transfusion-dependent Beta Thalassemia	52	Exagamglogene autotemcel	nrtkifrrkr(zsnmrywyzb) = krqxnzvvuw yrotahnrow (zrvolcmazc ) View more	Positive	11 Dec 2023

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Exagamglogene Autotemcel

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