A Phase 3, Randomized, Double-Blind, Placebo-Controlled Trial Evaluating the Efficacy and Safety of Human Allogeneic Cardiosphere-Derived Cells for the Treatment of Duchenne Muscular Dystrophy

HOPE-3 is a two cohort, Phase 3, multi-center, randomized, double-blind, placebo-controlled clinical trial evaluating the efficacy and safety of a cell therapy called CAP-1002 in study participants with Duchenne muscular dystrophy (DMD) and impaired skeletal muscle function. Non-ambulatory and ambulatory boys and young men who meet eligibility criteria will be randomly assigned to receive either CAP-1002 or placebo every 3 months for a total of 4 doses during the first 12-months of the study. All participants will be eligible to receive 4 doses of CAP-1002 for an additional 12 months as part of an open-label extended assessment period.

开始日期2022-06-22

申办/合作机构

Capricor, Inc.

NCT04623671 / Completed临床2期

A Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety and Efficacy of Intravenous Infusion of CAP-1002 in Patients With COVID-19 (INSPIRE)

This is a randomized, double-blind, placebo-controlled, Pilot, Phase 2 Exploratory study that will enroll subjects with a clinical diagnosis of COVID-19 confirmed by laboratory testing and who are in severe or critical condition as indicated by life-support measures.

开始日期2020-11-15

申办/合作机构

Capricor, Inc.

NCT04428476 / Active, not recruiting临床2期

Open-Label Extension of the HOPE-2 Duchenne Muscular Dystrophy Trial

This Phase 2, multi-center, open-label extension trial will provide deramiocel (CAP-1002) to subjects that were enrolled in the HOPE-2 trial and completed 12 months of follow-up. The trial will explore the safety and efficacy of twenty intravenous administrations of deramiocel, each separated by three months. Subjects will undergo a targeted screening during a 30-day screening period, eligible subjects will then undergo baseline safety and efficacy assessments on Day 1 prior to their first infusion of deramiocel.
Subjects will complete trial assessments at Screening; Day 1; Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48, 51, 54, 57, and 60. Safety and efficacy assessments will be conducted prior to deramiocel administration at the Day 1, Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48, 51, 54, and 57 trial visits, unless otherwise indicated.
All deramiocel infusions will be conducted in an outpatient setting at the investigative site on Day 1 and Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48, 51, 54, and 57. Subjects will be observed in the outpatient setting for at least two hours post infusion and then discharged the same day, if medically cleared by the site Investigator.

开始日期2020-07-20

申办/合作机构

Capricor, Inc.

100 项与 Deramiocel 相关的临床结果

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100 项与 Deramiocel 相关的转化医学

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100 项与 Deramiocel 相关的专利（医药）

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项与 Deramiocel 相关的文献（医药）

2023-01-02·Expert opinion on biological therapy

Biological and genetic therapies for the treatment of Duchenne muscular dystrophy

Review

作者: Wilton-Clark, Harry ; Yokota, Toshifumi

INTRODUCTION:

Duchenne muscular dystrophy is a lethal genetic disease which currently has no cure, and poor standard treatment options largely focused on symptom relief. The development of multiple biological and genetic therapies is underway across various stages of clinical progress which could markedly affect how DMD patients are treated in the future.

AREAS COVERED:

The purpose of this review is to provide an introduction to the different therapeutic modalities currently being studied, as well as a brief description of their progress to date and relative advantages and disadvantages for the treatment of DMD. This review discusses exon skipping therapy, microdystrophin therapy, stop codon readthrough therapy, CRISPR-based gene editing, cell-based therapy, and utrophin upregulation. Secondary therapies addressing nonspecific symptoms of DMD were excluded.

EXPERT OPINION:

Despite the vast potential held by gene replacement therapy options such as microdystrophin production and utrophin upregulation, safety risks inherent to the adeno-associated virus delivery vector might hamper the clinical viability of these approaches until further improvements can be made. Of the mutation-specific therapies, exon skipping therapy remains the most extensively validated and explored option, and the cell-based CAP-1002 therapy may prove to be a suitable adjunct therapy filling the urgent need for cardiac-specific therapies.

2022-03-01·Lancet (London, England)1区 · 医学

Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial

1区 · 医学

Article

作者: Johnson, Hannah ; Collado, Jorge ; Harmelink, Matthew M ; Connolly, Anne ; Muntoni, Francesco ; Furlong, Pat ; Anderson, Kristan ; Abedi, Mehrdad ; Newton, Andrew ; Dayan, Jonathan G. ; Pyzik, Erika ; Ruckdeschel Smith, Rachel ; Marbán, Linda ; Williams, Paula ; Hor, Kan N ; Varadhachary, Arun S ; Ascheim, Deborah D ; Jhawar, Sanjay ; Prasad, Poonam ; Chouteau, Wendy ; Finkel, Richard S ; Hendrix, Suzanne ; Kusmik, Kyle ; Janas, Joanne ; Wells, Julie ; Sarwary, Omaid ; Eagle, Michelle ; Brown, Michelle ; Surampudi, Prasanth ; Van Eyk, Jennifer ; Joyce, Nanette C. ; Giordano, Anthony F. ; Filar, Lauri ; Goude, Erica ; Rybalsky, Irina ; Butterfield, Russell ; McDonald, Craig M ; Anthonisen, Colleen ; Taylor, Michael D ; Rehborg, Rebecca ; Civitello, Matthew ; Wezel, Germaine ; Tian, Cuixia ; Baek, Jayoon ; Duke, Julie ; Marbán, Eduardo ; Henricson, Erik K ; Rogers, Russell G. ; Edmondson, Angie ; Vega, Melisa ; Nicorici, Alina ; Rodriguez, Aixa ; Mayer, Oscar H ; Evans, Maya ; Rogy, Siegfried ; Hogan, Nathaniel

BACKGROUND:

Cardiosphere-derived cells (CDCs) ameliorate skeletal and cardiac muscle deterioration in experimental models of Duchenne muscular dystrophy. The HOPE-2 trial examined the safety and efficacy of sequential intravenous infusions of human allogeneic CDCs in late-stage Duchenne muscular dystrophy.

METHODS:

In this multicentre, randomised, double-blind, placebo-controlled, phase 2 trial, patients with Duchenne muscular dystrophy, aged 10 years or older with moderate upper limb impairment, were enrolled at seven centres in the USA. Patients were randomly assigned (1:1) using stratified permuted blocks to receive CAP-1002 (1·5 × 10⁸ CDCs) or placebo intravenously every 3 months for a total of four infusions. Clinicians, caregivers, patients, and clinical operations personnel were fully masked to treatment groups. The primary outcome was the change in mid-level elbow Performance of Upper Limb version 1.2 (PUL 1.2) score at 12 months, assessed in the intention-to-treat population. Safety was assessed in all individuals who received an investigational product. This trial is registered with ClinicalTrials.gov, NCT03406780.

FINDINGS:

Between March 1, 2018, and March 31, 2020, 26 male patients with Duchenne muscular dystrophy were enrolled, of whom eight were randomly assigned to the CAP-1002 group and 12 to the placebo group (six were not randomised due to screening failure). In patients who had a post-treatment PUL 1.2 assessment (eight in the CAP-1002 group and 11 in the placebo group), the mean 12-month change from baseline in mid-level elbow PUL1.2 favoured CAP-1002 over placebo (percentile difference 36·2, 95% CI 12·7-59·7; difference of 2·6 points; p=0·014). Infusion-related hypersensitivity reactions without long-term sequelae were observed in three patients, with one patient discontinuing therapy due to a severe allergic reaction. No other major adverse reactions were noted, and no deaths occurred.

INTERPRETATION:

CAP-1002 cell therapy appears to be safe and effective in reducing deterioration of upper limb function in patients with late-stage Duchenne muscular dystrophy. Various measures of cardiac function and structure were also improved in the CAP-1002 group compared with the placebo group. Longer-term extension studies are needed to confirm the therapeutic durability and safety of CAP-1002 beyond 12 months for the treatment of skeletal myopathy and cardiomyopathy in Duchenne muscular dystrophy.

FUNDING:

Capricor Therapeutics.

2020-07-01·Basic research in cardiology

Allogeneic cardiosphere-derived cells (CAP-1002) in critically ill COVID-19 patients: compassionate-use case series

Article

作者: Friedman, Oren ; Chen, Peter ; Marbán, Linda ; Singh, Siddharth ; Akhmerov, Akbarshakh ; Zaman, Tanzira ; Ebinger, Joseph E ; Falk, Jeremy ; Gheorghiu, Mitch ; Marbán, Eduardo ; Makkar, Raj R ; Chakravarty, Tarun

Abstract:

There are no definitive therapies for patients with severe acute respiratory syndrome coronavirus-2 (SARS-CoV-2) infection. Therefore, new therapeutic strategies are needed to improve clinical outcomes, particularly in patients with severe disease. This case series explores the safety and effectiveness of intravenous allogeneic cardiosphere-derived cells (CDCs), formulated as CAP-1002, in critically ill patients with confirmed coronavirus disease 2019 (COVID-19). Adverse reactions to CAP-1002, clinical status on the World Health Organization (WHO) ordinal scale, and changes in pro-inflammatory biomarkers and leukocyte counts were analyzed. All patients (n = 6; age range 19–75 years, 1 female) required ventilatory support (invasive mechanical ventilation, n = 5) with PaO2/FiO2 ranging from 69 to 198. No adverse events related to CAP-1002 administration were observed. Four patients (67%) were weaned from respiratory support and discharged from the hospital. One patient remains mechanically ventilated as of April 28th, 2020; all survive. A contemporaneous control group of critically ill COVID-19 patients (n = 34) at our institution showed 18% overall mortality at a similar stage of hospitalization. Ferritin was elevated in all patients at baseline (range of all patients 605.43–2991.52 ng/ml) and decreased in 5/6 patients (range of all patients 252.89–1029.90 ng/ml). Absolute lymphocyte counts were low in 5/6 patients at baseline (range 0.26–0.82 × 103/µl) but had increased in three of these five patients at last follow-up (range 0.23–1.02 × 103/µl). In this series of six critically ill COVID-19 patients, intravenous infusion of CAP-1002 was well tolerated and associated with resolution of critical illness in 4 patients. This series demonstrates the apparent safety of CAP-1002 in COVID-19. While this initial experience is promising, efficacy will need to be further assessed in a randomized controlled trial.

109

项与 Deramiocel 相关的新闻（医药）

2024-11-12

·GlobeNewswire-Health Care

Capricor Therapeutics to Present Exosome Platform Updates at 2024 American Association of Extracellular Vesicles Annual Meeting

-Abstract Highlights New Preclinical Data on Exosome-Based Delivery of PMOs for the Treatment of DMD-SAN DIEGO, Nov. 12, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced that an abstract featuring new preclinical data highlighting the therapeutic potential of its StealthX™ exosome platform technology is being presented at the 2024 American Association of Extracellular Vesicles Annual Meeting taking place in Houston, Texas from November 10-13, 2024. The findings highlight a potential exosome-based approach for the treatment of Duchenne muscular dystrophy (DMD) by delivering phosphorodiamidate morpholino oligomers (PMOs). “We continue to build and characterize our StealthX™ exosome platform and this study further supports that endeavor as we provide a novel approach for exosome-based PMO delivery for the treatment of DMD,” said Linda Marbán, Ph.D., Capricor’s chief executive officer. “This study further demonstrates the potential for an exosome-based therapeutic approach to exhibit better cellular uptake and promote higher exon skipping efficiency. Furthermore, our platform continues to support the concept of exosomes as a potential suitable delivery vehicle for a variety of payloads and we continue to support our goal to leverage partnership opportunities to further advance this technology.” The abstract will be made available on the publications section of the Capricor website. About Capricor Therapeutics Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a biotechnology company dedicated to advancing transformative cell and exosome-based therapeutics to redefine the treatment landscape for rare diseases. At the forefront of our innovation is our lead product candidate, deramiocel (CAP-1002), an allogeneic cardiac-derived cell therapy. Extensive preclinical and clinical studies have shown deramiocel to demonstrate immunomodulatory, antifibrotic, and regenerative actions specifically tailored for dystrophinopathies and heart disease. Deramiocel is currently advancing through Phase 3 clinical development for the treatment of Duchenne muscular dystrophy. Capricor is also harnessing the power of its exosome technology, using its proprietary StealthX™ platform in preclinical development focused on the areas of vaccinology, targeted delivery of oligonucleotides, proteins and small molecule therapeutics to potentially treat and prevent a diverse array of diseases. At Capricor, we stand committed to pushing the boundaries of possibility and forging a path toward transformative treatments for those in need. For more information, visit capricor.com, and follow Capricor on Facebook, Instagram and Twitter. Cautionary Note Regarding Forward-Looking Statements Statements in this press release regarding the efficacy, safety, and intended utilization of Capricor’s product candidates; the initiation, conduct, size, timing and results of discovery efforts and clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and clinical trials; regulatory developments involving products, including the ability to obtain regulatory approvals or otherwise bring products to market; manufacturing capabilities; dates for regulatory meetings; statements about our financial outlook; the ability to achieve product milestones and to receive milestone payments from commercial partners; plans regarding current and future collaborative activities and the ownership of commercial rights; potential future agreements; scope, duration, validity and enforceability of intellectual property rights; future revenue streams and projections; expectations with respect to the expected use of proceeds from the recently completed offerings and the anticipated effects of the offerings; and any other statements about Capricor’s management team’s future expectations, beliefs, goals, plans or prospects constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words “believes,” “plans,” “could,” “anticipates,” “expects,” “estimates,” “should,” “target,” “will,” “would” and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements. More information about these and other risks that may impact Capricor’s business is set forth in Capricor’s Annual Report on Form 10-K for the year ended December 31, 2023, as filed with the Securities and Exchange Commission on March 11, 2024, and in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2024, as filed with the Securities and Exchange Commission on August 8, 2024. All forward-looking statements in this press release are based on information available to Capricor as of the date hereof, and Capricor assumes no obligation to update these forward-looking statements. Capricor has entered into an agreement for the exclusive commercialization and distribution of deramiocel (CAP-1002) for DMD in the United States and Japan with Nippon Shinyaku Co., Ltd. (U.S. subsidiary: NS Pharma, Inc.), subject to regulatory approval. Deramiocel is an Investigational New Drug and is not approved for any indications. None of Capricor’s exosome-based candidates have been approved for clinical investigation. For more information, please contact: Capricor Media Contact:Raquel ConaKCSA Strategic Communications rcona@kcsa.com212.896.1204 Capricor Company Contact:AJ Bergmann, Chief Financial Officerabergmann@capricor.com858.727.1755

细胞疗法临床3期

2024-11-04

·PRNewswire

Exosome Therapies on the Rise as 70+ Major Companies Driving Development in the Field | DelveInsight

Exosomes are emerging as key tools in personalized and regenerative medicine, offering precise, targeted therapy with low immune risk. Their potential spans cancer, chronic disease, and regenerative applications, driven by biopharma's investment in biologics and gene therapies. Stem cell-derived exosomes further enhance tissue repair and immune modulation, positioning exosomes as transformative in next-gen therapeutics. LAS VEGAS, Nov. 4, 2024 /PRNewswire/ -- DelveInsight's ' Exosomes Pipeline Insight 2024 ' report provides comprehensive global coverage of available, marketed, and pipeline exosomes-based therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the exosomes-based therapies pipeline domain. Key Takeaways from the Exosomes-based Therapies Pipeline Report DelveInsight's exosomes pipeline report depicts a robust space with 70+ active players working to develop 80+ pipeline therapies for various indications. Key exosomes-based therapies companies such as Capricor Therapeutics, Regeneus, Aegle Therapeutics, Innovex Therapeutics, Coya Therapeutics, EV Therapeutics, StemXO Therapeutics, Brexogen, ILIAS Biologics, Organicell Regenerative Medicine, Codiak BioSciences, Direct Biologics, Exostemtech, Brexogen, OmniSpirant, ShiftBio Inc., and others are evaluating new exosomes-based therapies to improve the treatment landscape. Promising exosomes-based therapies in the pipeline in various stages of development include CAP-1002, Progenza, AGLE 102, ExoCoVac, COYA 201, EV-101, StemXO Endosome Therapy, BRE AD01, ILB 202, Zofin, exoSTING, ExoFlo, CARTISOME, BRE MI01, OS006, SBI-407, and others. In July 2024, Aruna Bio announced the issuance of a US Patent for the composition of matter for neural exosomes used to deliver therapeutics to the brain. The company is on a rack to initiate Phase Ib/IIa clinical trial of AB126 in acute ischemic stroke in the second half of 2024. In July 2024, ILIAS Biologics announced that it had received the final clinical study report (CSR) for ILB-202, a Phase I clinical trial completed in Australia in November last year. In April 2024, Biological Dynamics joined the growing list of customers adopting the ExoVerita Pro instrument, a cost-efficient, automated exosomal enrichment platform. In April 2024, EXO Biologics secures up to EUR 16 million in Series A funding for exosome therapeutic development and clinical supply. In April 2024, ExoXpert entered a strategic partnership with Neucore Bio to leverage ExoXpert's platform for the evaluation of advanced exosome loading. In April 2024, INOVIQ and Promega signed a global supply and distribution agreement for EXO-NET. Last year's company entered into a Co-Marketing agreement. In March 2024, VivaZome Therapeutics and La Trobe University collaborated to explore the therapeutic potential of extracellular vesicles in models of stroke in the laboratory of Professor Chris Sobey. In January 2024, Aegle Therapeutics announced positive 12-week data for the first patient enrolled in Phase I/IIa clinical trial of AGLE-102 for the treatment of severe second-degree burns. Request a sample and discover the recent advances in exosomes-based therapies @ Exosomes Pipeline Outlook The exosomes-based therapies pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage exosomes-based therapies, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the exosomes-based therapies pipeline landscape. Exosomes Overview Exosomes are small extracellular vesicles, typically ranging from 30 to 150 nanometers in diameter, that are secreted by various cell types. They play a crucial role in intercellular communication by facilitating the transfer of proteins, lipids, and RNA between cells. This process can influence various physiological and pathological conditions, including immune responses, cancer progression, and neurodegenerative diseases. By carrying bioactive molecules, exosomes can modulate the behavior of recipient cells, contributing to processes such as tissue repair, immune regulation, and the spread of cancer cells. Their ability to transport molecular cargo makes them a subject of extensive research, particularly in the context of disease mechanisms and therapeutic applications. In recent years, exosomes have garnered significant interest in the field of biomedicine as potential biomarkers for disease diagnosis and as vehicles for targeted drug delivery. Researchers are exploring their use in therapeutic strategies, such as the delivery of RNA-based therapies and proteins, due to their natural ability to evade the immune system and penetrate cellular membranes. Additionally, exosomes derived from stem cells or engineered for specific therapeutic purposes hold promise for regenerative medicine, offering a novel approach to enhance tissue regeneration and repair. The ongoing investigation into the biology of exosomes continues to reveal their multifaceted roles in health and disease, paving the way for innovative applications in diagnostics and therapeutics. Find out more about exosomes-based therapies @ Exosomes-based Therapies in Development A snapshot of the Exosomes-based Therapies Pipeline Drugs mentioned in the report: Learn more about the emerging Exosomes-based therapies @ Exosomes-based Therapies Clinical Trials Exosomes-based Therapies Therapeutics Assessment The exosome-based therapies pipeline report proffers an integral view of the emerging exosome-based therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Exosomes-based Therapies Pipeline Report Coverage: Global Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Therapeutics Assessment By Route of Administration: Oral, Intravenous, Subcutaneous, Parenteral, Topical Therapeutics Assessment By Molecule Type: Recombinant fusion proteins, Small molecule, Monoclonal antibody, Peptide, Polymer, Gene therapy Therapeutics Assessment By Mechanism of Action: Cell replacements, Inflammation mediator modulators, Collagen type VII replacements, Immunosuppressants, NF-kappa B inhibitor Key Exosomes-based Therapies Companies: Capricor Therapeutics, Regeneus, Aegle Therapeutics, Innovex Therapeutics, Coya Therapeutics, EV Therapeutics, StemXO Therapeutics, Brexogen, ILIAS Biologics, Organicell Regenerative Medicine, Codiak BioSciences, Direct Biologics, Exostemtech, Brexogen, OmniSpirant, ShiftBio Inc., and others. Key Exosomes-based Therapies in Pipeline: CAP-1002, Progenza, AGLE 102, ExoCoVac, COYA 201, EV-101, StemXO Endosome Therapy, BRE AD01, ILB 202, Zofin, exoSTING, ExoFlo, CARTISOME, BRE MI01, OS006, SBI-407, and others. Dive deep into rich insights for exosomes-based therapies assessment, visit @ Exosomes-based Therapies Therapeutics Assessment Table of Contents For further information on the exosomes-based therapies pipeline therapeutics, reach out @ Exosomes-based Therapies Pipeline Landscape Related Reports Exosome Therapies Market Exosome Therapies Market Size, Target Population, Competitive Landscape & Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key exosome therapies companies, including Direct Biologics, Capricor Therapeutics, Aegle Therapeutics, Vitti Labs, Rion, Aruna Bio, EXO Biologics, EV Therapeutics, ReNeuron, Coya Therapeutics, Evox Therapeutics , among others. Duchenne Muscular Dystrophy Market Duchenne Muscular Dystrophy Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key DMD companies, including FibroGen, Santhera Pharmaceutical, Italfarmaco, ReveraGen BioPharma, Cumberland Pharmaceuticals, Sarepta Therapeutics, Antisense Therapeutics, Capricor Therapeutics , among others. Duchenne Muscular Dystrophy Pipeline Duchenne Muscular Dystrophy Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Duchenne muscular dystrophy companies, including Santhera Pharmaceuticals, Sarepta Therapeutics, Italfarmaco, Wave Life Sciences Ltd, FibroGen, Edgewise Therapeutics, Pfizer, Daiichi Sankyo, Sarepta Therapeutics, Inc., ENCell, Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, Hansa Biopharma, Ultragenyx Pharmaceutical, Dyne Therapeutics, Entrada Therapeutics, AAVogen, PepGen, Antisense Therapeutics, BioMarin Pharmaceutical, Avidity Biosciences, Sarepta Therapeutics, Dyne Therapeutics, Solid Biosciences Inc, Regenxbio, Stealth BioTherapeutics, among others. Atopic Dermatitis Market Atopic Dermatitis Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key atopic dermatitis companies, including Arcutis Biotherapeutics, Amgen, Kyowa Kirin, Dermavant Sciences, Cara Therapeutics, Pfizer, Arena Pharmaceuticals, BioMimetix, Eli Lilly and Company, Aldeyra Therapeutics, Inc., Hangzhou Yirui Pharmaceutical Technology Co., Ltd, LEO Pharma, Corvus Pharmaceuticals, Inc., Brexogen Inc., Sanofi, Shaperon, UCB Pharma, Q32 Bio Inc., Akeso, Apogee Therapeutics, Inc., Allakos Inc., Biosion, Inc. , among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

临床1期

2024-10-19

·药明康德

使94%患儿病情稳定的基因疗法；缓解持续三年的细胞疗法…… | CGT周报

▎药明康德内容团队编辑近期，全球细胞和基因疗法（CGT）领域迎来系列进展。美国FDA批准的首款脑性肾上腺脑白质营养不良（CALD）基因疗法Skysona的长期研究结果积极，中位随访时间超过6年，94%患者的神经功能没有下降。用于治疗杜氏肌营养不良（DMD）的细胞疗法deramiocel在一项临床试验中使患者的心脏和骨骼肌功能在三年期间趋于稳定，其上市申请有望在今年年底前完成递交。首个进入人体临床试验的工程化B细胞疗法ISP-001的初步结果积极，首位接受治疗的1型黏多糖贮积症（MPS I）患者已停止使用标准疗法。本文将节选其中部分重要进展做简单介绍，仅供读者参阅。图片来源：123RF ———✦研发进展✦——— ◇ 近日，《新英格兰医学杂志》（NEJM）上刊登了两篇论文，公布了美国FDA批准的首款脑性肾上腺脑白质营养不良基因疗法Skysona（elivaldogene autotemcel，也称为eli-cel）的长期随访结果。首批在临床试验中接受eli-cel治疗的CALD患儿的中位随访时间已超过6年。长期疗效数据显示，eli-cel能够控制这种神经系统疾病的进展，94%患者的神经功能没有下降，超过80%的患者没有出现严重残疾。 CALD是一种罕见的X染色体连锁遗传性脑部疾病，主要发生于男孩。该病患儿因ABCD1基因突变导致过氧化物酶体功能缺失，大量异常的极长链脂肪酸（VLCFAs）会沉积于大脑白质和肾上腺，从而导致患儿进行性、不可逆的神经功能丧失，最终过早死亡。Eli-cel利用Lenti-D慢病毒载体，在体外将ABCD1基因的功能性拷贝导入到患者自身的造血干细胞中，再输回到患者体内，从而促进VLCFAs分解、避免毒性积累。 ◇ Capricor Therapeutics公司公布了其主打细胞疗法deramiocel（CAP-1002）治疗不能行动的晚期DMD患者的HOPE-2开放标签扩展（OLE）试验的三年积极结果。HOPE-2试验达成其主要终点，即在测试上肢功能的指标PUL 1.2上，接受CAP-1002治疗的病患有显著疗效（2.6分差异，P=0.014）。OLE试验的分析显示，接受deramiocel治疗患者的心脏和骨骼肌功能在三年期间趋于稳定。Capricor预计在今年年底前完成递交该疗法用于治疗DMD相关心肌病患者的生物制品许可申请（BLA）。 CAP-1002是一种同种异体的细胞疗法，它们由心脏来源细胞（CDCs）组成，CDCs是一种含有心脏祖细胞的特殊细胞群。它们通过释放包含微RNA、非编码RNA和蛋白的外泌体，改善DMD患者肌肉瘢痕或纤维化以及心脏功能。CAP-1002还显示出有效的免疫调节活性，可能促进细胞再生。目前美国FDA已授予CAP-1002再生医学先进疗法认定与孤儿药资格。 ◇ MeiraGTx公司公布了在研帕金森病基因疗法AAV-GAD在随机双盲、安慰剂对照临床试验MGT-GAD-025中的顶线数据。在这项试验中，帕金森病患者接受双侧丘脑底核AAV-GAD输注或安慰剂。初步数据显示，AAV-GAD达到了安全性和耐受性的主要终点。此外，接受AAV-GAD治疗26周后，患者在运动能力和生活质量方面均获得统计显著并具有临床意义的改善。 AAV-GAD是一款在研基因疗法，能将编码谷氨酸脱羧酶（GAD）的转基因递送至丘脑底核，以增加人脑中的主要抑制性神经递质GABA的产生。GAD是GABA合成中的限速酶，因此通过基因疗法增加丘脑底核GAD的表达，将导致运动回路正常化，并改善帕金森病的症状，同时不影响其他可能造成现有疗法并发症的大脑区域。 ◇ Beacon Therapeutics公司公布了其在研基因疗法AGTC-501在治疗X连锁视网膜色素变性（XLRP）的2期临床试验SKYLINE中获得的积极结果。在接受治疗24个月后，接受高剂量AGTC-501治疗的眼睛的缓解率达到57%。接受低剂量AGTC-501治疗的眼睛的缓解率为25%。AGTC-501表现出良好的安全性和耐受性，治疗伴发不良事件的大多为轻度或中度。 XLRP是一种遗传性单基因隐性疾病，会导致男孩和年轻男性视力逐渐丧失。XLRP主要由视网膜色素变性GTPase调节剂（RPGR）基因突变引起。AGTC-501表达全长RPGR蛋白，从而解决XLRP引起的感光细胞损伤，包括视杆细胞和视锥细胞的丧失。图片来源：123RF ◇ Immusoft公司公布了首位接受其工程化B细胞疗法ISP-001治疗的1型黏多糖贮积症患者的积极结果。根据新闻稿，ISP-001是首个进入人体临床试验的工程化B细胞疗法。该疗法使用非病毒载体将表达α-L-艾杜糖苷酶的转基因引入B细胞中，能够让经过改造的B细胞长期大量生产治疗性蛋白，从而改善患者症状。初步数据显示，该患者的疾病生物标志物指标获得改善，在接受一次ISP-001治疗8个月后，已停止使用标准酶替代疗法（ERT）。 ◇ 中国国家药监局药品审评中心（CDE）官网最新公示，合源生物纳基奥仑赛注射液获批一项临床试验默示许可，针对适应症为难治性系统性红斑狼疮相关的免疫性血小板减少症（SLE-ITP）。纳基奥仑赛为靶向CD19嵌合抗原受体自体T（CD19 CAR-T）细胞注射液。根据合源生物新闻稿，这也是该产品在自身免疫性疾病治疗领域获得的第一张IND批件。今年7月，《新英格兰医学杂志》在线发表了纳基奥仑赛治疗SLE-ITP的案例报道。1名此前曾接受过大量治疗的SLE-ITP女性患者在接受纳基奥仑赛注射液回输后的第28天，血液中的CD19阳性B细胞被清除，第三个月时B细胞获得重建。患者的血小板计数在治疗后的六个月内持续上升，并达到临床完全缓解。此外，患者停用了糖皮质激素和所有免疫抑制剂。 ◇ 原天生物1类生物制品新药YT001注射液在中国获批临床，拟开发治疗膝骨关节炎（KOA）。根据原天生物新闻稿介绍，YT001是其研发的一款间充质干细胞治疗产品。间充质干细胞具有免疫调节作用，能够缓释炎症反应，促进损伤修复，其免疫调节和损伤修复功能可帮助调节关节腔内紊乱微环境，缓解软骨的降解。 ◇ 因诺惟康宣布，其自主研发的可玻璃体内给药的基因疗法IVB103注射液获得了CDE的临床许可，旨在治疗新生血管性年龄相关性黄斑变性（nAMD）。IVB103通过该公司的EASI-FIND腺相关病毒（AAV）发现平台开发，具有“best-in-class”的潜力。今年7月，IVB103注射液获得了FDA的IND许可，用于同样的适应症。 ◇ 瑞风生物申报的RM-101注射液在中国获批临床，拟开发用于治疗USH2A基因外显子13突变所致的Usher综合征2型相关视网膜色素变性（RP）或非综合征型RP。Usher综合征是与视网膜色素变性有关的最常见综合征，USH2A基因突变导致视网膜色素变性，患者通常在青少年时期逐渐出现夜盲和周边视野缺失，最终可能导致全盲。根据瑞风生物公开资料介绍，RM-101是基于AAV的基因编辑类药物，能特异靶向USH2A基因转录的RNA，调控可变剪接生物学过程，诱导功能正常蛋白的表达恢复。该产品通过视网膜下注射给药，有望实现一次性给药并长期有效。 ◇ 恒瑞医药子公司瑞宏迪医药申报的1类新药RGL-193注射液在中国获批临床，拟开发治疗帕金森病。公开资料显示，RGL-193是瑞宏迪医药针对帕金森病自主研发的一款AAV双基因药物，采用立体定向技术注入患者脑内。它可以提高左旋多巴的转化效率，同时还起到保护和修复受损多巴胺能神经元的作用，具有潜在的延缓病情进展、减少口服抗帕金森病药量的效果。 ◇ 艺妙医疗申报的CAR-T细胞注射液IM96在中国获批临床，拟开发用于治疗结直肠癌。根据艺妙医疗新闻稿介绍，IM96是其与北京大学肿瘤医院合作开发用于治疗转移性结直肠癌的全新一代抗肿瘤药物，是一款靶向鸟苷酸环化酶2C（GUCY2C）的CAR-T疗法。该产品的临床前研究数据于今年6月入选美国临床肿瘤学会（ASCO）年会，研究显示IM96针对结直肠癌的最佳客观缓解率（ORR）达到50%，且仅有5.0%的患者出现神经毒性和≥3级的细胞因子释放综合征（CRS）。 ———✦融资、合作、M&A✦——— 图片来源：123RF ◇ Tolerance公司宣布完成了1720万美元的种子轮融资，本轮融资由Columbus Venture Partners领投，Criteria Bio Ventures、Sessa Capital、BioAdvance、Ben Franklin Technology Partners等投资者参投。该公司正在开发一种以同种异体或“现货型”胸腺诱导多能干细胞（iPSC）为基础的细胞疗法平台和胸腺疗法，旨在延缓和预防人体免疫系统中的重要器官胸腺的衰退，并在胸腺功能丧失时恢复其功能，以治疗由免疫耐受异常引起的免疫介导疾病，包括癌症、自身免疫疾病、移植排斥、感染、免疫缺陷和过敏。 ◇ 睿健医药宣布完成超亿元B轮融资，该资金将用于加速其帕金森病治疗产品NouvNeu001/NouvNeu003，以及眼科治疗产品NouvSight001的中美临床推进，加强公司多款创新管线的研发推进和临床能力的搭建，以及加大公司产业化建设的力度。 NouvNeu001是一款源于iPSC，化学诱导分化的多巴胺能神经元前体细胞。新闻稿指出，NouvNeu001是帕金森领域中，全球首个进入临床阶段的基于化学诱导的通用型细胞治疗产品，其IND申请已在中美两地获批。其早发型帕金森产品NouvNeu003的1期临床试验也即将完成剂量拓展阶段，实现睿健医药覆盖帕金森病全病程的重要一步。NouvSight001是睿健医药团队依托公司独特的“AI+化学诱导”平台开发的首款iPSC来源眼科通用型细胞治疗产品，旨在针对视网膜色素变性系列适应症。该产品于今年3月获得了美国FDA授予的孤儿药资格。 ◇ Eterna Therapeutics公司与Factor Bioscience公司宣布达成一项独家许可与合作协议，旨在加速开发治疗肿瘤、罕见病和自身免疫疾病的候选细胞疗法。根据协议条款，Eterna公司获得了一项全球独家许可，利用Factor公司的细胞重编程和基因编辑技术，开发某些基于iPSC的细胞疗法产品，尤其是iPSC衍生的间充质干细胞（iMSC），该细胞经过工程化处理可表达某些细胞因子。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放参考资料（可上下滑动查看） [1] Florian Eichler, et al., (2024). Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy. The N Engl J Med, DOI: 10.1056/NEJMoa2400442 [2] Christine N. Duncan, et al., (2024). Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy. The N Engl J Med, DOI: 10.1056/NEJMoa2405541 [3] Capricor Therapeutics Announces Positive Long-Term Data from HOPE-2 OLE Study in Duchenne Muscular Dystrophy at 2024 World Muscle Society Congress. Retrieved October 11, 2024 from https://www.globenewswire.com/news-release/2024/10/11/2962050/0/en/Capricor-Therapeutics-Announces-Positive-Long-Term-Data-from-HOPE-2-OLE-Study-in-Duchenne-Muscular-Dystrophy-at-2024-World-Muscle-Society-Congress.html [4] Beacon Therapeutics Announces Positive 24-Month Data from Phase 2 SKYLINE Trial of AGTC-501 in Patients with X-Linked Retinitis Pigmentosa. Retrieved October 15, 2024, from https://www.prnewswire.com/news-releases/beacon-therapeutics-announces-positive-24-month-data-from-phase-2-skyline-trial-of-agtc-501-in-patients-with-x-linked-retinitis-pigmentosa-302275688.html [5] MeiraGTx Announces Positive Data from Randomized, Sham-controlled Clinical Bridging Study of AAV-GAD for the Treatment of Parkinson’s Disease. Retrieved October 15, 2024, from https://investors.meiragtx.com/news-releases/news-release-details/meiragtx-announces-positive-data-randomized-sham-controlled [6] Immusoft to Announce Positive Phase 1 Data for First Engineered B Cell Therapy in a Clinical Trial. Retrieved October 15, 2024, from https://www.prnewswire.com/news-releases/immusoft-to-announce-positive-phase-1-data-for-first-engineered-b-cell-therapy-in-a-clinical-trial-302255551.html [7] 合源生物纳源瑞达®新增自身免疫性疾病适应症新药临床试验（IND）申请获默示许可. Retrieved October 11, 2024, from https://mp.weixin.qq.com/s/9-w9DlWNPKFMdZ8KjxGz7g [8]中国国家药监局药品审评中心（CDE）官网. Retrieved October 13, 2024, from https://www.cde.org.cn/main/xxgk/listpage/4b5255eb0a84820cef4ca3e8b6bbe20c [9] Tolerance Bio, Inc. Launches With $17.2 Million in Seed Financing to Advance Thymus-Based Therapies for Immune-Mediated Diseases. Retrieved October 18, 2024, from https://www.businesswire.com/news/home/20241008134130/en/Tolerance-Bio-Inc.-Launches-With-17.2-Million-in-Seed-Financing-to-Advance-Thymus-Based-Therapies-for-Immune-Mediated-Diseases#:~:text=Tolerance%20Bio%2C%20Inc.%20Launches%20With%20%2417.2%20Million%20in,manipulation%20platforms%20to%20prevent%20and%20treat%20immune%20diseases [10] 睿健医药完成超亿元B轮融资，加速国际化进程. Retrieved October 18, 2024, from https://mp.weixin.qq.com/s/gIxbOkl79mn2_OSK0bAs1w [11] Eterna Therapeutics and Factor Bioscience Announce Exclusive License and Collaboration Agreement to Accelerate Cell Therapy Development for Oncology, Autoimmune, and Rare Diseases. Retrieved October 18, 2024, from https://www.globenewswire.com/news-release/2024/10/17/2964799/0/en/Eterna-Therapeutics-and-Factor-Bioscience-Announce-Exclusive-License-and-Collaboration-Agreement-to-Accelerate-Cell-Therapy-Development-for-Oncology-Autoimmune-and-Rare-Diseases.html [12] 因诺惟康IVB103注射液获得CDE临床许可. Retrieved October 18, 2024, from https://mp.weixin.qq.com/s/OU_bxfT9u4a_-Cs6WreoDQ 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

孤儿药基因疗法细胞疗法临床研究

100 项与 Deramiocel 相关的药物交易

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研发状态

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适应症	最高研发状态	国家/地区	公司	日期
杜氏肌营养不良症	申请上市	美国	Capricor Therapeutics, Inc.	2024-10-09
新型冠状病毒感染	临床2期	美国	Capricor, Inc.	2020-11-15
心脏衰竭	临床2期	美国	Medical University of South Carolina	2016-09-11
心肌疾病	临床2期	美国	Capricor, Inc.	2015-07-19
心肌梗塞	临床2期	美国	Capricor, Inc.	2012-11-13
左心室功能不全	临床2期	美国	Capricor, Inc.	2012-11-13
肌营养不良	药物发现	美国	Lonza Houston, Inc.	-

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04428476 (HOPE-2 OLE, GlobeNewswire) 人工标引	临床2期	杜氏肌营养不良症	-	Deramiocel	鹹襯網窪壓廠蓋鑰簾繭(鏇憲觸網蓋淵襯蓋顧鹽) = 襯淵觸廠廠獵範壓鏇簾觸蓋醖艱鑰夢糧膚鑰簾 (繭範糧憲鏇壓蓋願構齋 )	积极	2024-10-11
NCT02941705 (RegressHFpEF, CTgov)	临床2期	射血分数保留型心力衰竭 \| 纤维化 \| 舒张期心力衰竭	27	Allogeneic Derived Cells (RECIEVED CELLS)	鑰鹹廠顧壓遞鏇積範簾(網蓋廠醖願糧繭鏇醖壓) = 繭壓鏇鏇鏇鹽網蓋觸製觸築繭壓鏇艱築遞遞餘 (選醖範範願鬱壓蓋衊構, 鏇壓糧艱獵窪醖憲選觸 ~ 鹹廠膚遞鹽壓繭簾廠淵) 更多	-	2024-07-25
				Placebo/Control Arm (CONTROL ARM)	鑰鹹廠顧壓遞鏇積範簾(網蓋廠醖願糧繭鏇醖壓) = 蓋鬱構壓蓋鏇膚鹽範網觸築繭壓鏇艱築遞遞餘 (選醖範範願鬱壓蓋衊構, 鏇鹽遞夢齋鏇網製夢製 ~ 獵積顧繭製構淵鑰觸夢) 更多
NCT04428476 (HOPE-2-OLE, Biospace) 人工标引	临床2期	杜氏肌营养不良症	20	CAP-1002	願願製壓鹽顧齋築餘選(糧鑰憲範淵鏇餘艱簾夢) = 衊顧艱構觸醖選蓋淵鹽遞網衊齋糧窪窪廠膚繭 (構淵窪顧壓餘簾鬱鬱窪 )	积极	2024-06-04
				External Comparator	願願製壓鹽顧齋築餘選(糧鑰憲範淵鏇餘艱簾夢) = 蓋積膚繭顧鏇憲壓獵餘遞網衊齋糧窪窪廠膚繭 (構淵窪顧壓餘簾鬱鬱窪 )
NCT01458405 (ALLSTAR, CTgov)	临床1/2期	左心室功能不全 \| 心肌梗塞	135	placebo (Randomized Treatment Cohort: Placebo)	鹹膚襯願願襯築蓋糧齋(淵憲夢廠顧鏇積網窪淵) = 鹽製願餘艱窪積鹹鏇網鹽積願糧廠鹽顧衊餘構 (蓋築範膚醖齋醖遞製簾, 構簾淵選願獵糧網鹹壓 ~ 齋範繭糧夢衊鹹構蓋窪) 更多	-	2024-04-09
				CAP-1002 (Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells)	鹹膚襯願願襯築蓋糧齋(淵憲夢廠顧鏇積網窪淵) = 簾艱膚選網網鏇糧襯選鹽積願糧廠鹽顧衊餘構 (蓋築範膚醖齋醖遞製簾, 顧鏇觸築夢餘蓋製遞夢 ~ 醖齋獵網遞簾蓋鬱醖醖) 更多
NCT05126758 (HOPE-2-OLE, MDA2024) 人工标引	临床3期	杜氏肌营养不良症	12	CAP-1002	夢遞餘淵獵膚網製鑰夢(顧顧網鹽獵觸製遞製鬱) = 廠選鏇壓獵夢壓憲鏇製鬱選窪膚鏇鹽鏇窪餘憲 (築獵網積艱網鹹蓋鑰繭 ) 更多	积极	2024-03-03
NCT04428476 (Corporate Publications) 人工标引	临床2期	杜氏肌营养不良症	13	CAP-1002 (continuous CAP-1002)	網遞遞蓋襯獵鑰簾顧鬱(繭餘壓憲齋襯衊夢顧繭) = CAP-1002, when used over time, is slowing DMD’s devastating effects and may be disease modifying. 鏇餘積鹹鹹鹹鹹衊積鹹 (衊遞製膚艱積遞顧憲夢 ) 更多	积极	2022-06-27
				CAP-1002 (from placebo to CAP-1002)
NCT03406780 (HOPE-2, Pubmed \| Lancet) 人工标引	临床2期	杜氏肌营养不良症	19	CAP-1002	膚簾觸獵膚廠壓顧壓夢(簾積蓋選鏇範鹹廠獵鏇) = No other major adverse reactions were noted 醖顧壓選製遞衊願顧廠 (齋鏇網網餘鏇餘鑰廠築 ) 更多	积极	2022-03-12
				Placebo
NCT02485938 (HOPE-Duchenne, Pubmed \| Neurology) 人工标引	临床1/2期	杜氏肌营养不良症	25	CAP-1002	糧築簾糧鏇糧襯製壓築(廠觸願鑰鑰壓鹹繭衊艱) = Mid-distal PUL improved at 12 months in 8 of 9 lower functioning CAP-1002 patients, and no controls 廠選獵鹹鹽膚簾淵鏇獵 (憲鏇願壓繭壓窪觸艱衊 )	积极	2019-02-19
				usual care
NCT02293603 (Biospace) 人工标引	临床1期	扩张型心肌病 \| 心脏衰竭	-	CAP-1002	獵構觸鏇齋築艱積觸膚(齋積觸鏇選顧艱構壓淵) = 遞繭構廠網膚築淵衊餘憲範艱艱簾憲範窪壓鹽 (艱廠襯餘窪齋窪構簾憲 ) 更多	积极	2015-11-09