Verbrinacogene Setparvovec

The dynamics of the cell and gene therapies market in rare disorders are anticipated to change as companies across the globe are thoroughly working toward developing new therapeutic options to treat a wide array of indications. LAS VEGAS, May 9, 2023 /PRNewswire/ -- DelveInsight's Cell and Gene Therapies in Rare Disorders Market Insights report includes a comprehensive understanding of current treatment practices, emerging cell and gene therapies for various rare disorders, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Key Takeaways from the Cell and Gene Therapies in Rare Disorders Market Report As per DelveInsight analysis, the cell and gene therapies in rare disorders market size in the 7MM was approximately USD 1.5 billion in 2022. According to the assessment done by DelveInsight, the estimated total prevalent cases of selected indications for cell and gene therapies in rare disorders in the 7MM were approximately 900K in 2022. Leading companies developing novel cell and gene therapies for various rare disorders, such as Hemophilia A, Hemophilia B, Fabry disease, Retinitis Pigmentosa, Amyotrophic Lateral Sclerosis, Duchenne Muscular Dystrophy, and others include Roche, Freeline Therapeutics, Spark Therapeutics, Astellas Gene Therapies, Actus Therapeutics, GenSight Biologics, Coave Therapeutics, Johnson & Johnson, MeiraGTx, Applied Genetic Technologies Corporation, GenSight Biologics, Nanoscope Therapeutics, 4D Molecular Therapeutics, Ocugen, jCyte, ReNeuron, REGENXBIO, Amicus Therapeutics, Pfizer, Sarepta Therapeutics, Capricor Therapeutics, Nippon Shinyaku, Brainstorm Cell Therapeutics, CRISPR Therapeutics, Vertex Pharmaceuticals, Editas Medicine, Sangamo Therapeutics, Krystal Biotech, Abeona Therapeutics, Castle Creek Biosciences, Holostem Terapie Avanzate S.r.l., RHEACELL, Ishin Pharma, Anterogen, Ultragenyx Pharmaceutical, and others. The promising cell and gene therapies for various rare disorders in the pipeline include Giroctocogene fitelparvovec, Dirloctocogene samoparvovec, Fidanacogene elaparvovec, Verbrinacogene setparvovec (FLT-180a), FLT190, Isaralgagene civaparvovec (ST-920), 4D-310, SPK-3006, AT845, ACTUS-101, LUMEVOQ (lenadogene nolparvovec), CTx-PDE6b, Botaretigene sparoparvovec, ATGC-501 (laruparetigene zosaparvovec), GS030, MCO-010 (sonpiretigene isteparvovec), 4D-125, OCU400, jCell, RGX-121, AT-GTX-502 (scAAV9.P546.CLN3), PF-06939926, SRP-9001, CAP-1002, NurOwn (MSC-NTF cells), Exagamglogene autotemcel, EDIT-301, BIVV003, VYJUVEK (beremagene geperpavec), EB-101, D-Fi (dabocemagene autoficel), RV-LAMB3-transduced epidermal stem cells, Allogeneic ABCB5-positive Stem Cells, ISN001, ALLO-ASC-SHEET, DTX301, UX111 (ABO-102), DTX401 (AAV8G6PC), and others. In July 2022, the Institute for Clinical and Economic Review (ICER) released a final evidence report assessing the comparative clinical effectiveness and value of ZYNTEGLO (betibeglogene autotemcel) for treating beta-thalassemia. In May 2022, Orchard Therapeutics reached an agreement with Gesetzliche Krankenversicherung Spitzenverband (GKV-SV), which will result in reimbursed access to LIBMELDY (atidarsagene autotemcel) for all metachromatic leukodystrophy (MLD) patients in Germany who fall within the scope of the European marketing authorization. Discover which therapies are expected to grab the major cell and gene therapies in rare disorders market share @ Cell and Gene Therapies in Rare Disorders Market Report Cell and Gene Therapies in Rare Disorders Overview Cell and gene therapies use genes and cells to treat disease. A gene is a unit of DNA containing genetic information passed down from generation to generation. The genome comprises all genes; genes may contain information on observable features such as height or eye color. Many genes contain instructions for RNA or protein molecules that are not visible from the outside but serve crucial tasks in the body's cells. Cells are the building blocks of plants and animals (including humans); they are small functional units that work together to generate organs and tissues. Cell and gene therapy technology is quickly evolving for many different diseases. However, cell and gene treatments are still experimental drugs, and much more study is required before many of these therapies are available to patients worldwide. Cell and Gene Therapies in Rare Disorders Epidemiology Segmentation DelveInsight estimates that there were approximately 900K prevalent cases of selected indications for cell and gene therapies in rare disorders in the 7MM in 2022. As per our analysis, the highest prevalent cases from the selected indications for cell and gene therapies in rare disorders were for Retinitis Pigmentosa in the United States, whereas the least cases were reported for Hunter Syndrome in 2022. The cell and gene therapies in rare disorders market report proffers epidemiological analysis for the study period 2019–2032 in the 7MM segmented into: Total Prevalent Cases of Shortlisted Indications for Cell and Gene Therapies in Rare Disorders Total Indication Eligible-wise Cases for Cell and Gene Therapies in Rare Disorders Indication-wise Treated Cases of Cell and Gene Therapies Cell and Gene Therapies in Rare Disorders Market Insights Numerous cell and gene therapies for rare diseases are currently approved in the 7MM, including retinitis pigmentosa ( LUXTURNA), beta-thalassemia ( ZYNTEGLO), epidermolysis bullosa ( JACE), limbal stem cell deficiency ( OCURAL), and many others. As per Delveinsight analysis, the total cell and gene therapies in rare disorders market size was around USD 1.5 billion in 2022. According to predictions, the United States will have the largest cell and gene therapies in rare disorders market. ZOLGENSMA produced the highest revenue of roughly USD 1 billion among the 7MM in 2022, while ROCTAVIAN is predicted to take the highest market share by 2032. ROCTAVIAN has received conditional approval in Europe for the treatment of severe hemophilia A. In addition to this approval, BioMarin Pharmaceutical is working to approve the drug in the United States, with a PDUFA target action date of June 30, 2023. Hemophilia A is predicted to produce the most revenue among the selected indications by 2032, owing to the precedence of existing high treatment cost and expected high cost for emerging therapies along with significant residual unmet need. Gene treatments for Hemophilia A are estimated to earn around USD 6 billion in sales revenue by 2032 in the 7MM. The field of cell and gene therapies for rare indications is expected to rapidly expand in the coming years, as an increasing number of companies submit investigational new drug applications for these treatments each year, along with rising regulatory approval in the United States and Europe. In terms of manufacturing aspects of cell and gene therapies, there will be more competition for contract manufacturing businesses and pharmaceutical/biotechnology firms. The competition for contract manufacturing organization's production capacity will intensify as more companies enter the cell and gene therapy market, possibly driving up manufacturing costs. To ensure pharmaceutical/biotechnology firms can compete or obtain an advantage over competitors, companies may need to invest in manufacturing technologies or acquire companies with manufacturing expertise. To know more about cell and gene therapies in rare disorders treatment guidelines, visit @ Cell and Gene Therapy Insights Emerging Cell and Gene Therapies for Hemophilia A and Key Companies Giroctocogene fitelparvovec: Pfizer/Sangamo Therapeutics Dirloctocogene samoparvovec: Roche Emerging Cell and Gene Therapies for Hemophilia B and Key Companies Fidanacogene elaparvovec: Pfizer/Spark Therapeutics Verbrinacogene setparvovec (FLT-180a): Freeline Therapeutics Emerging Cell and Gene Therapies for Fabry Disease and Key Companies FLT190: Freeline Therapeutics Isaralgagene civaparvovec (ST-920): Sangamo Therapeutics 4D-310: 4D Molecular Therapeutics Emerging Cell and Gene Therapies for Pompe Disease and Key Companies SPK-3006: Spark Therapeutics AT845: Astellas Gene Therapies ACTUS-101: Actus Therapeutics Emerging Cell and Gene Therapies for Leber Hereditary Optic Neuropathy and Key Companies LUMEVOQ (lenadogene nolparvovec): GenSight Biologics Emerging Cell and Gene Therapies for Retinitis Pigmentosa and Key Companies CTx-PDE6b: Coave Therapeutics Botaretigene sparoparvovec: Johnson & Johnson/ MeiraGTx ATGC-501 (laruparetigene zosaparvovec): Applied Genetic Technologies Corporation GS030: GenSight Biologics MCO-010 (sonpiretigene isteparvovec): Nanoscope Therapeutics 4D-125: 4D Molecular Therapeutics OCU400: Ocugen jCell: jCyte Emerging Cell and Gene Therapies for Hunter Syndrome and Key Companies RGX-121: REGENXBIO Emerging Cell and Gene Therapies for Batten Disease and Key Companies AT-GTX-502 (scAAV9.P546.CLN3): Amicus Therapeutics Emerging Cell and Gene Therapies for Duchenne Muscular Dystrophy (DMD) and Key Companies PF-06939926: Pfizer SRP-9001: Sarepta Therapeutics CAP-1002: Capricor Therapeutics/Nippon Shinyaku Emerging Cell and Gene Therapies for Amyotrophic Lateral Sclerosis (ALS) and Key Companies NurOwn (MSC-NTF cells): Brainstorm Cell Therapeutics Emerging Cell and Gene Therapies for Beta Thalassemia and Sickle Cell Anemia and Key Companies Exagamglogene autotemcel: CRISPR Therapeutics/Vertex Pharmaceuticals EDIT-301: Editas Medicine BIVV003: Sangamo Therapeutics Emerging Cell and Gene Therapies for Dystrophic Epidermolysis Bullosa and Key Companies VYJUVEK (beremagene geperpavec): Krystal Biotech EB-101: Abeona Therapeutics D-Fi (dabocemagene autoficel): Castle Creek Biosciences RV-LAMB3-transduced epidermal stem cells: Holostem Terapie Avanzate S.r.l. Allogeneic ABCB5-positive Stem Cells: RHEACELL ISN001: Ishin Pharma ALLO-ASC-SHEET: Anterogen Emerging Cell and Gene Therapies for Ornithine Transcarbamylase Deficiency and Key Companies DTX301: Ultragenyx Pharmaceutical Emerging Cell and Gene Therapies for Sanfilippo Syndrome Type A and Key Companies UX111 (ABO-102): Ultragenyx Pharmaceutical Emerging Cell and Gene Therapies for Glycogen Storage Disease Type IA and Key Companies DTX401 (AAV8G6PC): Ultragenyx Pharmaceutical Learn more about the FDA-approved cell and gene therapies for rare disorders @ Approved Cell and Gene Therapies in Rare Disorders Treatment Cell and Gene Therapies in Rare Disorders Market Dynamics The cell and gene therapies in rare disorders market is predicted to grow positively due to an increase in the approval of a growing number of gene therapies and their ease of adoption following approval, the ability to treat a wide range of conditions, an increase in the number of cases, an expected one-time dosing approach, and curative treatment options. The approval of LIBMELDY, SKYSONA, HOLOCLAR, UPSTAZA, ROCTAVIAN, and other medicines has successfully created regulatory channels for the development of further cell and gene therapies. Companies around the world are working hard to develop new cell and gene therapies options to treat a wide range of indications, such as hemophilia A and B, lysosomal storage disorder (Fabry, Pompe Disease, Danon Disease, MPS I, MPS II, MPS III), neurological disorders (Batten, Parkinson), musculoskeletal disorders (DMD, myotubular myopathy), eye diseases (achromatopsia, limbal stem cell deficiency, retinitis pigmentosa, retinoschisis, age-related macular degeneration, Leber's hereditary optic neuropathy), and other indications such as diabetic macular edema, inborn metabolism disorder (Wilson's disease, Phenylketonuria, OTC deficiency/urea cycle disorders), dystrophic epidermolysis bullosa, gangliosidosis, and xerostomia. Many diseases' treatment landscapes have drastically evolved in the last few years. Companies are now developing cell and gene therapies that will play an important role in the future, particularly in the treatment of rare genetic disorders. The process of defining ideal candidates for given gene therapy and cell therapy will have to wait for the enrolment and long-term follow-up of a sufficient number of study subjects to provide satisfactory clarity regarding its safety and efficacy. In conclusion, the future of cell and gene therapy looks optimistic. Several clinical trials have yielded favorable results in terms of safety and efficacy. The findings of these studies motivate additional research into many indications, and the current scenario predicts a positive shift in the cell and gene therapies in rare disorders market for the forecast period. However, several factors may impede the growth of cell and gene therapies in rare disorders market in the coming years. Despite advances since the enactment of the Orphan Drug Act, people in the United States with rare diseases continue to face challenges to diagnosis, care, and treatment. Moreover, in Europe, a possible crisis with gene treatments for rare diseases is developing, and few companies have withdrawn the drug after failing to get reimbursement in the EU. Scope of the Cell and Gene Therapies in Rare Disorders Market Report Therapeutic Assessment: Cell and Gene Therapies in Rare Disorders current marketed and emerging therapies Cell and Gene Therapies in Rare Disorders Market Dynamics: Conjoint Analysis of Emerging Cell and Gene Therapies in Rare Disorders Drugs Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Unmet Needs, KOL's Views, Analyst's Views, Cell and Gene Therapies in Rare Disorders Market Access and Reimbursement Discover more about cell and gene therapies for rare disorders in development @ Cell and Gene Therapy Clinical Trials Table of Contents Related Reports Gene and Cell Therapies in Rare Disorder Epidemiology Forecast Gene and Cell Therapies in Rare Disorder Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted gene and cell therapies in rare disorder epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Cell And Gene Therapy For Multiple Myeloma Market Cell And Gene Therapy For Multiple Myeloma Market Insights, Epidemiology, and Market Forecast – 2032 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 cell and gene therapy for multiple myeloma companies, including Pfizer, MorphoSys, Biokine Therapeutics, Arcellx, Rapa Therapeutics, among others. Gene Therapy For Ocular Rare Disease Pipeline Gene Therapy For Ocular Rare Disease Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key gene therapy for ocular rare disease companies, including Roche Holding, Novartis, Ocugen, among others. Adeno-Associated Virus Vectors in Gene Therapy Market Adeno-Associated Virus Vectors in Gene Therapy Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key adeno-associated virus vectors in gene therapy companies, including Pfizer, CSL Behring, Spark Therapeutics, Freeline Therapeutics, RegenxBio, Amicus Therapeutics, 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] +1(919)321-6187 Logo: SOURCE DelveInsight Business Research, LLP

LONDON, July 10, 2022 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) today announced the presentation of safety and initial efficacy data from the first cohort of the Phase 1/2 dose-confirmation B-LIEVE trial for FLT180a, the company’s AAVS3-based gene therapy candidate for people with hemophilia B, at the International Society on Thrombosis and Haemostasis (ISTH) Congress being held in London, July 9-13, 2022. As of the data cut-off of May 23, 2022, a one-time FLT180a dose of 7.7e11 vg/kg generated a rapid increase of coagulation factor IX (FIX), reaching levels in the normal range (93, 92 and 80 IU/dL) for the three patients in cohort 1 through days 77, 56 and 36, respectively. Patients stopped FIX prophylaxis and did not require FIX replacement or experience bleeding following treatment with FLT180a. The treatment and the prophylactic immune management regimen were well tolerated. No serious adverse events or infusion reactions were observed, and there has been no evidence of FIX inhibitors. All adverse events (AEs) were mild, and most were transient. AEs related to immune management were consistent with the known profiles of corticosteroids and tacrolimus. Dosing of cohort two was completed in June, with early results showing a similar initial response to FLT180a. Based on the data from cohort one and consistent with the advice of the independent Data Monitoring Committee of the B-LIEVE trial, patients in cohort two received the same dose of FLT180a and prophylactic immune management regimen that were used in the first cohort. As the data continue to evolve since the data cut-off for cohort one, two patients have experienced a decrease in FIX expression together with a mild and transient increase in liver enzymes. All patients continue to have expression levels above baseline, and no patient has experienced a bleed or required FIX supplementation. “The initial data show that FLT180a provides rapid and consistent elevations in FIX to normal levels, which can prevent bleeding and the need for regular FIX replacement in people with hemophilia B,” said Pamela Foulds, MD, Chief Medical Officer of Freeline. “Emerging data suggest that while FIX expression was maintained at protective levels, a further refined immune management regimen may be required to avoid mild and transient transaminitis and to sustain FIX levels in the normal range. Potential adjustments in cohort two and forthcoming results from that cohort will help us interpret this further.” “Our strategy is to advance gene therapy programs that have the potential to deliver best-in-class or first-in-class treatments,” said Michael Parini, Chief Executive Officer of Freeline. “While we continue to believe FLT180a has the potential to deliver a best-in-class gene therapy for people with hemophilia B, the availability of other treatment options and the need to prioritize our valuable resources dictate that we evaluate strategic options for FLT180a. These include, but are not limited to, seeking a partner that would enable the continuation of FLT180a through Phase 3 development.” “With two other promising programs in Fabry disease and Gaucher disease in the clinic, each with the potential to be first-in-class gene therapy treatments for these debilitating diseases, we will continue to focus our attention and resources on those programs that offer the highest value for patients and Freeline’s shareholders,” said Parini. The poster #PB0213 entitled “Results from B-LIEVE, a Phase 1/2 Dose-Confirmation Study of FLT180a AAV Gene Therapy in Patients with Hemophilia B” by Guy Young et al. will be presented from 6:30pm-7:30pm BST today and will be made available on the Investors section of Freeline’s website. About the B-LIEVE Dose-Confirmation Trial B-LIEVE is a Phase 1/2 dose confirmation trial of FLT180a using a short course of prophylactic immune management with the goal of normalizing FIX levels in patients with severe and moderately severe hemophilia B. The starting dose of 7.7e11 vg/kg was selected based on the results of the Phase 1/2 B-AMAZE dose-finding trial and modeling. The goal of the B-LIEVE trial is to finalize a dose for a pivotal Phase 3 trial that enables predictable and sustained factor IX (FIX) expression at protective levels. About FLT180a for People with Hemophilia B Freeline’s FLT180a candidate uses a potent AAVS3 capsid rationally designed for effective targeting and transduction of liver cells and containing an expression cassette encoding a gain of function Padua variant of human factor IX (FIX). FLT180a has been studied in B-AMAZE, a Phase 1/2 dose-finding trial with the goal of normalizing FIX activity in patients with moderately severe and severe hemophilia B. Patients treated in B-AMAZE are being followed in a long-term follow-up study. A Phase 1/2 dose-confirmation trial of FLT180a called B-LIEVE to finalize a dose for a Phase 3 pivotal trial is in progress. About Hemophilia B Hemophilia B is a rare, debilitating, hereditary bleeding disorder caused by a defect in the gene encoding coagulation factor IX (FIX). Hemophilia B is linked to the X chromosome and mainly affects boys and men; however, women who carry an affected copy of the coagulation factor gene may also experience symptoms. Hemophilia B is classified as mild, moderate or severe, depending on the level of FIX in the blood, and is diagnosed through blood tests. The 2020 Annual Global Survey by the World Federation of Hemophilia estimates that there are approximately 15,000 patients with hemophilia B in the United States, Europe and Japan. A meta-analysis using national registries in Australia, Canada, France, Italy, New Zealand and the UK estimated a prevalence in males of 3.8 in 100,000 or approximately 1 in 30,000.1 About Freeline Therapeutics Freeline is a clinical-stage biotechnology company developing transformative adeno-associated virus (AAV) vector-mediated systemic gene therapies. The company is dedicated to improving patient lives through innovative, one-time treatments that may provide functional cures for inherited systemic debilitating diseases. Freeline uses its proprietary, rationally designed AAV vector, along with novel promoters and transgenes, to deliver a functional copy of a therapeutic gene into human liver cells, thereby expressing a persistent functional level of the missing or dysfunctional protein into the patient’s bloodstream. The company’s integrated gene therapy platform includes in-house capabilities in research, clinical development and commercialization. The company has clinical programs in hemophilia B, Fabry disease, and Gaucher disease Type 1. Freeline is headquartered in the UK and has operations in Germany and the U.S.