25. Individuals 14 years of age and older with low testosterone or estrogen may be eligible. This decline in transgene expression occurred in the absence of transaminitis, with the greatest decrease in FVIII activity occurring in those patients with the highest level of FVIII expression. Regeneron is leading development for both hemophilia A and hemophilia B development programs. Recursion Pharmaceuticals Inc. is sponsoring a phase 2, multi-center, randomized, double-blind, placebo-controlled study to evaluate the safety, efficacy, and pharmacokinetics of REC-994 compared to placebo for the treatment of patients with symptomatic CCM. For more information, visit:ClinicalTrials.gov NCT02722304. A nod from the FDA would give Roctavian the same title in the U.S., cementing its leading position against rival treatments being developed by Roche and Sangamo Therapeutics. For more information visitClinicalTrials.gov:NCT03559699. With little data to go on, experts question how useful Eisai, Biogens new Alzheimers drug will be, String of gene therapy deals spurs cautious optimism on Wall Street, Building a biotech in a downturn: 3 lessons from VCs and startup CEOs, Rubius replaces CEO as it cuts more jobs and considers a sale, Surface Oncology to lay off 20% of staff in restructuring, A New Standard of Care: The Benefits of Continuous Temperature Monitoring and Early Fever Detection, The latest developments on the gene therapy frontier, This is biologys century. Seton Hall University is conducting a study to help understand how a mobile health technology app can assistadolescents and young adults with SCD with their transition to adult care and help them manage the disease as adults. FVIII expression in the highest-dose cohort = 94-140%. cDNA, complementary DNA; IM, intramuscular. Incyte Corporation is sponsoring a study to compare the efficacy of parsaclisib when combined with ruxolitinb versus placebo combined with ruxolitinib in patients with myelofibrosis. More. Patients with moderate hemophilia may bleed spontaneously and have pronounced bleeding after trauma, whereas in patients with mild hemophilia, bleeding is usually restricted to traumatic events; however, even these individuals have an increased risk for death from intracranial bleeding compared with the normal population.2. More. That figure could climb higher if underwriters exercise their right to buy another 1.5 million shares at the IPO price. More. More. Recently, companies have been more likely to try and prolong the cash For instance, the decrease in expression in the 4e13-vg/kg dose cohort was modest. Enrolled 2 patients in each cohort (9e11, 2e12, 1e13, and 3e13 vg/kg). Estimated enrollment is 108 participants. The found reduced levels of NPHP5 protein within the patient-derived retinal organoid cells, as well as reduced levels of another protein called CEP-290, which interacts with NPHP5 and forms the primary cilium gate. Manufacturers should consider how digital tools can further unlock operational challenges in production. Estimated enrollment is 12 participants. Achillion Pharmaceuticals is investigating the effects of ACH-4471, an orally administered complement factor D inhibitor, on complement C3 levels in patients with immune complex-mediated membranoproliferative glomerulonephritis (IC-MPGN). While only four in vivo viral-vector gene therapies are currently on the market, more than 100 gene-therapy assets are in clinical trials as of late 2020, with a far greater number in preclinical development.. 1.8 News and Social Media Coverage. More. Researchers at the University of Iowa Institute for Clinical and Translational Science will be gathering information from a large number of sarcoidosis patients located all around the world using a web-based survey. A cure for hemophilia seemed closer than ever. In preclinical studies in mice and nonhuman primates, scAAV vectors mediated a 10-fold increase in transduction efficiency compared with ssAAV vectors.19,20 Subsequently, we realized that the covalently closed hairpin structure of scAAV vector inhibited polymerase chain reaction amplification of vector genomes, resulting in an underestimation of vector titer. F. Hoffmann-La Roche AG, commonly known as Roche, is a Swiss multinational healthcare company that operates worldwide under two divisions: Pharmaceuticals and Diagnostics. The researchers discovered that a type of Leber congenital amaurosis (LCA) is caused by mutations in the NPHP5 (also called IQCB1) gene and leads to severe defects in the primary cilium, a structure found in The global tele intensive care unit market size reached US$ 4.2 Billion in 2021. Estimated enrollment is 149 participants. Keep up with the story. Elevation of ALT was observed in 2 of 3 patients between weeks 4 and 6 after gene transfer, requiring treatment with corticosteroids.34 No participant has developed a FVIII inhibitor. Catalyst Pharmaceuticals, Inc. is sponsoring a randomized, double-blind, controlled, outpatient two-period, two-treatment crossover study is designed to evaluate the efficacy and safety of amifampridine phosphate in patients (ages 2 to 70) diagnosed with certain genetic subtypes of CMS and demonstrated open label (amifampridine phosphate) or history of sustained amifampridine benefit from treatment.For more information visit. Sigilon Therapeutics, Inc. is sponsoring a first-in-human (FIH), phase 1/2, multi-centre, open-label, dose escalation study to assess the safety, tolerability, and preliminary efficacy of SIG-001 in adult patients with severe or moderately severe hemophilia A without inhibitors. Astellas Pharma Global Development, Inc. is sponsoring a phase 1 and phase 2 study of gilteritinib in combination with chemotherapy in children, adolescents and young adults with FMS-like tyrosine kinase 3 (FLT3)/internal tandem duplication (ITD) positive relapsed or refractory AML. For more information, visit: ClinicalTrials.gov:NCT035997. Ben Adams, FDA says Voyager can trek on as it removes trial hold for Huntingtons gene therapy test, Fierce Biotech, April 26, 2021. Katherine A. 29. Rocket Pharmaceuticals Inc. is sponsoring a non-randomized open-label phase 1 study to evaluate the safety and toxicity of gene therapy using a recombinant adeno-associated virus serotype 9 (AAV9) containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene (investigational product (IP), RP-A501) in male patients with Danon disease. Bayer was responsible for the commercial tasks. This natural history study is being conducted to better understand disease course and to determine clinically relevant outcome measures for use in future clinical trials. The industry-wide talent shortage could benefit from augmented and virtual reality. NORD offers financial support for study-related expenses and concierge travel arrangements to patients participating in. Though it shares some similarities with CRISPR, it can swap out specific DNA letters as well as edit out or add sequences of nucleotides. For products in the development stage, this means identifying high-potential innovations that could bring significant advancements early on, such as high-yield adapted cell lines or novel production systems. Caelum Biosciences, Inc. is sponsoring a study to determine the recommended dose of CAEL-101, to facilitate progression of further clinical trials, and evaluate safety and tolerability of CAEL-101 in combination with the standard of care (cyclophosphamide-bortezomib-dexamethasone (CyBorD) chemotherapy and daratumumab) in patients with AL Amyloidosis. More. Earlier this year, Roctavian became the first hemophilia A gene therapy to be approved in Europe, where its now sold at a list price of roughly 1.5 million euros. PTC Therapeutics is sponsoring a study to assess the efficacy and safety of vatiquinone for the treatment of patients with Friedreich ataxia. Estimated enrollment is 57 participants. More. More. The use of CDMOs is particularly suited to smaller companies or early-stage development activities, with several examples of strategic partnerships with contract manufacturers. Based on their portfolio strategy and the proportion and development stage of in-licensed assets, companies need to balance between investing in the development of proprietary technologies and selecting the most suitable systems and methods for each asset. BioCryst Pharmaceuticals is sponsoring a study to evaluate the efficacy and safety of BCX9930 monotherapy for the treatment of PNH compared to continued C5 inhibitor therapy in adult PNH patients with residual anemia despite treatment with a C5 inhibitor. ACADIA Pharmaceuticals Inc. is sponsoring a study to investigate the efficacy of treatment with oral trofinetide versus placebo in females with Rett syndrome. Therefore, hemophilia patients have to carefully plan periods of increased physical activities, such as sports, which people living without hemophilia can hardly imagine. Modis Therapeutics, Inc. and Zogenix, Inc. are sponsoring a study to evaluate the efficacy and safety of MT1621 in patients with genetic confirmation of TK2d who have never received MT1621 or nucleos(t)ides before entering the study. These stem cell samples were used to generate retinal organoids, cultured tissue clusters that possess many of the structural and functional features of actual, native retina. Additionally, the lower seroprevalence of AAV8 in humans (25% compared with >70% for AAV2) enabled exclusion of fewer subjects with preexisting humoral immunity to AAV8.23. Individuals with SCD age 18-30 are invited to participate in a short anonymous survey: https://www.surveymonkey.com/r/YHKHS8L. Estimated enrollment is 111 participants. Bluebird, November 2020. Thus, the therapeutic goal for gene therapy of hemophilia is modest in comparison with the majority of monogenetic disorders. Prop 30 is supported by a coalition including CalFire Firefighters, the American Lung Association, environmental organizations, electrical workers and businesses that want to improve Californias air quality by fighting and preventing wildfires and reducing air The hemophilias are ideally suited for gene therapy because a small increment in blood factor levels (5% of normal) is associated with significant amelioration of bleeding phenotype in severely affected patients. More. 16. Gene therapy offers the potential for a cure for patients with hemophilia by establishing continuous endogenous expression of factor VIII or factor IX (FIX) following transfer of a functional gene to replace the hemophilic patients own defective gene. and achieving patient access, and gene therapy companies will need to overcome key manufacturing challenges as they tailor their processes to their assets, while also making critical decisions at the asset and portfolio level that will allow them to leverage developments in viral-vector manufacturing to accelerate patient access (Exhibit 2). They are centered around the critical choice of production system; the ability to optimize downstream processing (DSP); and the development of standardized chemistry, manufacturing, and control (CMC) methods and quality assays. In spite of the many challenges facing biotechnology companies, a number of enterprises have risen to the top of the list. We'll email you when new articles are published on this topic. The precise pathophysiological basis for transaminitis remains unclear, in part because it has not been possible to recapitulate this toxicity in animal models.27,37 Clinical data show that the increase in ALT postAAV gene therapy is dependent on vector dose and possibly the number of CpG motifs, as discussed previously, but is independent of AAV capsid, genome configuration, transgene promoter, and method of manufacture. Recently, companies have been more likely to try and prolong the cash More. The decline in FIX expression and the liver toxicity were thought to be due to a capsid-specific cytotoxic T-cell response directed against the transduced hepatocytes prompted by the presentation of AAV2 capsid peptide in the context of MHC I molecules.17 Thus, in this study, humoral and cell-mediated immune responses limited persistent expression of FIX following liver-targeted administration of AAV vectors in humans. Copyright 2021 NORD - National Organization for Rare Disorders, Inc. All rights reserved. Spark Therapeutics is developing SPK-8016, a novel, internally developed investigational gene therapy for hemophilia A, or Factor VIII deficiency, to address the inhibitor market using a gene-based approach.Hemophilia A is a serious and rare inherited hematologic disorder, characterized by mutations in the F8 gene, which lead to deficient blood coagulation and an increased risk of Primary cilia play a role in cell cycle regulation. More. BioCryst Pharmaceuticals is sponsoring a first-in-human study of BCX9930 in healthy volunteers and patients with PNH. Prior to her role at Freeline, she served as head of clinical research and development at Spark Therapeutics, where she oversaw the clinical development of all programs. The global tele intensive care unit market size reached US$ 4.2 Billion in 2021. More. Spark Therapeutics is developing SPK-8016, a novel, internally developed investigational gene therapy for hemophilia A, or Factor VIII deficiency, to address the inhibitor market using a gene-based approach.Hemophilia A is a serious and rare inherited hematologic disorder, characterized by mutations in the F8 gene, which lead to deficient blood coagulation and an increased risk of Participants will receive the study drug and all other study procedures at no cost. This has engendered interest in targeting approaches.46 One such approach, GeneRide, entails the use of AAV vectors to deliver a promoterless FIX gene flanked by a guide DNA arm that is several hundred base pairs long and matches a specific locus (eg, albumin locus). this space, with a number of large acquisitions in the past few years (Exhibit 1). The FDA is also currently reviewing a hemophilia B gene therapy developed by UniQure, a Netherlands-based biotechnology company, with a decision anticipated in the next month or so. 65567. Retrophin is conducting the PROTECT study, a randomized, multicenter, double-blind, parallel-group, active-control study, to evaluate the potential benefit of sparsentan on kidney function by analyzing change in proteinuria (protein in urine) and estimated glomerular filtration rate (eGFR) as compared to current standard treatment. With $50 million in funding, Boston-based Ascidian Therapeutics claims its RNA exon editing approach could match the durability of gene therapy while avoiding some of the risks that come with editing DNA. New modified synthetic formulations of FVIII and FIX that are pegylated or fused to proteins with a long half-life, such as albumin or Fc, have greatly improved the stability profile for FX but have been less impressive for FVIII as a result of the dominant role of von Willebrand factor in determining its half-life.5 BIVV001, a novel FVIII fusion protein consisting of a single-chain recombinant FVIIIFc fused to the FVIII-binding DD3 domains of von Willebrand factor, as well as 2 XTEN linkers, has an increased half-life 38 hours, raising the prospects of extended protection in hemophilia A patients with weekly dosage.6 In patients with hemophilia B, extended half-life products allow reduction of injection frequency to once weekly or even once biweekly while maintaining higher trough levels (FIX > 5%), despite the reduction of injection frequency. The National Human Genome Research Institute is conducting this study to evaluate patients with MMA to learn more about the genetic causes of the various types of this inherited metabolic disorder and the medical complications associated with it. This study has been followed by unparalleled advances in gene therapy for hemophilia A and B, leading to clotting factor activity approaching normal or near-normal levels associated with a zero bleed rates in previously severely affected patients following a single administration of AAV vectors. More. The latest report by IMARC Group, titled Online Grocery Market: Global Industry Trends, Share, Size, Growth, Opportunity and Forecast 2022-2027 , offers a comprehensive analysis of the industry, which comprises insights on the global online grocery market The report also includes competitor and regional analysis, and contemporary advancements in the market. 24. Correction: A previous version of this story misstated David Lius first name. More. More. 21. This could result in more specific guidance on potency assays and clearer paths for switching production systems as analytical methods for comparability studies evolve. Incyte Corporation is sponsoring a study to assess the safety, tolerability, and efficacy of itacitinib immediate release tablets in patients with primary or secondary myelofibrosis who have received prior ruxolitinib and/or fedratinib monotherapy. The chart is updated daily and includes information about each individual agent or trade name of a drug or treatment, the sponsor company, the date of the designations public disclosure, the individual agents indication, or For more information visit:https://clinicaltrials.gov/ct2/show/NCT03301467?cond=NCT03301467&rank=1. For instance, AAV5 serotype pseudotyped vectors (AMT-060; UniQure Therapeutics, Amsterdam, The Netherlands) made using the insect cellbaculovirus method, but containing the same FIX gene cassette as that used in the St. Jude/UCL trial, resulted in mean FIX activity levels of 6.9%, despite using a log higher vector dose of 2e13 vg/kg.25 Increased ALT levels were observed in 3 of 10 patients recruited to AMT-060, requiring treatment with corticosteroids. Emily Capra is an associate partner in McKinseys Bay Area office; Andrea Gennari is a partner in the London office; and Alberto Loche is an associate partner in the Zurich office, where Carolin Temps is a consultant. Fuchsine and aniline became the company's most important products. Prior to her role at Freeline, she served as head of clinical research and development at Spark Therapeutics, where she oversaw the clinical development of all programs. Efficacy assessments in this study will evaluate motor, language, visual and cognitive function and each participant will be followed for safety and efficacy for 5 years after treatment. Regulatory guidance will likely evolve over time and become more detailed as more products come to market. Janssen Pharmaceuticals is sponsoring a study to evaluate whether the addition of selexipag to standard of care treatment delays disease progression in children with PAH in comparison to placebo. To learn more about clinical trials, why they matter and how to participate visit: Ovid Therapeutics Inc. is sponsoring a study to investigate the effect of TAK-935 on the frequency of motor seizures in patients with 15q duplication syndrome during the maintenance period. The National Cancer Institute is conducting a phase III study to determine how well pembrolizumab works compared to standard of care observation in treating patients with stage I-III Merkel cell cancer that has been completely removed by surgery. Estimated enrollment is 33 participants. Regeneron Pharmaceuticals is sponsoring a study to evaluate the effect of pozelimab and cemdisiran combination therapy on hemolysis, as assessed by lactate dehydrogenase, in patients with PNH who switch from eculizumab or ravulizumab therapy versus patients who continue eculizumab or ravulizumab therapy. For more information visit: https://clinicaltrials.gov/ct2/show/NCT03624374?cond=LBSL&rank=1. More. Center for Information and Study on Clinical Research Participation, Alpha-1 Antitrypsin Deficiency (A1ATD)-Associated Liver Disease, Alpha-1 Proteinase Inhibitor (A1PI) Deficiency and Chronic Pulmonary Disease (COPD), Atypical Hemolytic Uremic Syndrome (aHUS), Autoimmune Pulmonary Alveolar Proteinosis (aPAP), Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN), Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), Chronic Thromboembolic Pulmonary Hypertension (CTEPH), Congenital Sucrase-Isomaltase Deficiency (CSID), https://clinicaltrials.gov/ct2/show/record/NCT02927067, https://clinicaltrials.gov/ct2/show/record/NCT02931539, Eosinophilic Granulomatosis With Polyangiitis (EGPA), Epstein-Barr Virus (EBV) Associated Diseases, Fibrodysplasia Ossificans Progressiva (FOP), Focal Segmental Glomerulosclerosis (FSGS), Frontotemporal Dementia with Progranulin Mutations (FTD-GRN), Gastroenteropancreatic Neuroendocrine Tumor (GEP-NET), Hemolytic Disease of the Fetus and Newborn (HDFN), Hereditary Inclusion Body Myopathy (HIBM), Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN), Leukoencephalopathy with Axonal Spheroids and Pigmented Glia (Adult-Onset) (ALSP), Leukoencephalopathy with Brain Stem and Spinal Cord Involvement and Lactate Elevation (LBSL), Limb-Girdle Muscular Dystrophy 21 (LGMD2I), Neurofibromatosis Type 2 (NF2) Mutated Meningiomas, Paroxysmal Nocturnal Hemoglobinuria (PNH), Clinicaltrials.gov Identifier: NCT01410890, Post-Transplant Lymphoproliferative Disease, Primary Distal Renal Tubular Acidosis (dRTA), Primary Focal Segmental Glomerulosclerosis (FSGS), Pyruvate Dehydrogenase Complex Deficiency (PDCD), SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE), Short Bowel Syndrome with Intestinal Failure (SBS-IF), Transverse Myelitis (including Acute Flaccid Myelitis). 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