A Phase 3, Single-arm, Open-label, Multicenter Study of the Safety and Efficacy of Dirloctocogene Samoparvovec (SPK 8011, Adeno-associated Viral Vector With B-domain Deleted Human Factor VIII Gene) in Adults With Severe or Moderately Severe Hemophilia A

The purpose of this study is to evaluate the efficacy of SPK-8011 in preventing bleed episodes compared with FVIII prophylaxis in participants with hemophilia A without FVIII inhibitors on routine FVIII prophylaxis.

Start Date13 Mar 2024

Sponsor / Collaborator

Spark Therapeutics, Inc.

NCT03432520

/ Active, not recruitingNot Applicable

A Multi-Center Evaluation of the Long-Term Safety and Efficacy of Spark-sponsored Gene Therapies in Males With Hemophilia A

This long-term follow-up study will continue to evaluate the long-term safety and efficacy of SPK-8011 and SPK-8016 in males with hemophilia A, who have received a single intravenous administration of SPK-8011 or SPK-8016 in any Spark-sponsored SPK-8011 or SPK-8016 study.

Start Date14 Aug 2018

Sponsor / Collaborator

Spark Therapeutics, Inc.

NCT03003533

/ CompletedPhase 1/2

Gene-transfer, Open-label, Dose-escalation Study of SPK-8011 [Adeno-associated Viral Vector With B-domain Deleted Human Factor VIII Gene] in Individuals With Hemophilia A

This clinical research study is being conducted by Spark Therapeutics, Inc. to determine the safety and efficacy of the factor VIII gene transfer treatment with SPK-8011 in individuals with hemophilia A.

Start Date26 Jan 2017

Sponsor / Collaborator

Spark Therapeutics, Inc.

100 Clinical Results associated with SPK-8011

100 Translational Medicine associated with SPK-8011

100 Patents (Medical) associated with SPK-8011

Literatures (Medical) associated with SPK-8011

01 Mar 2022·Molecular therapy. Methods & clinical developmentQ2 · MEDICINE

Preclinical assessment of an optimized AAV-FVIII vector in mice and non-human primates for the treatment of hemophilia A

Q2 · MEDICINE

ArticleOA

Author: Silverberg, Joseph ; High, Katherine A ; Frick, Jennifer ; Wang, Yuhuan ; Kutza, Stephanie ; Toso, Raffaella ; Davidson, Robert J ; DiPietro, Marti ; Elkouby, Liron ; Sabatino, Denise E ; Armour, Sean M ; Nguyen, Giang N ; Wang, Chuansong ; Anguela, Xavier M ; Crosariol, Marco ; Willet, Mallory

Extensive clinical data from liver-mediated gene therapy trials have shown that dose-dependent immune responses against the vector capsid may impair or even preclude transgene expression if not managed successfully with prompt immune suppression. The goal of this preclinical study was to generate an adeno-associated viral (AAV) vector capable of expressing therapeutic levels of B-domain deleted factor VIII (FVIII) at the lowest possible vector dose to minimize the potential Risk of a capsid-mediated immune response in the clinical setting. Here, we describe the studies that identified the investigational agent SPK-8011, currently being evaluated in a phase 1/2 study (NCT03003533) in individuals with hemophilia A. In particular, the potency of our second-generation expression cassettes was evaluated in mice and in non-human primates using two different bioengineered capsids (AAV-Spark100 and AAV-Spark200). At 2 weeks after gene transfer, primates transduced with 2 × 10¹² vg/kg AAV-Spark100-FVIII or AAV-Spark200-FVIII expressed FVIII antigen levels of 13% ± 2% and 22% ± 6% of normal, respectively. Collectively, these preclinical results validate the feasibility of lowering the AAV capsid dose for a gene-based therapeutic approach for hemophilia A to a dose level orders of magnitude lower than the first-generation vectors in the clinic.

18 Nov 2021·The New England journal of medicineQ1 · MEDICINE

Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A

Q1 · MEDICINE

Article

Author: High, Katherine A ; Sullivan, Spencer K ; Anguela, Xavier M ; Samelson-Jones, Benjamin J ; George, Lindsey A ; Kuranda, Klaudia ; MacDougall, Amy ; Jaworski, Kristen ; Noble, Robert ; Monahan, Paul E ; Recht, Michael ; Rasko, John E J ; Reape, Kathleen Z ; Croteau, Stacy E ; Chang, Tiffany ; Eyster, M Elaine ; Ragni, Margaret V ; Mingozzi, Federico ; Curran, Marla

BACKGROUND:

The goal of gene therapy for patients with hemophilia A is to safely impart long-term stable factor VIII expression that predictably ameliorates bleeding with the use of the lowest possible vector dose.

METHODS:

In this phase 1-2 trial, we infused an investigational adeno-associated viral (AAV) vector (SPK-8011) for hepatocyte expression of factor VIII in 18 men with hemophilia A. Four dose cohorts were enrolled; the lowest-dose cohort received a dose of 5 × 10¹¹ vector genomes (vg) per kilogram of body weight, and the highest-dose cohort received 2 × 10¹² vg per kilogram. Some participants received glucocorticoids within 52 weeks after vector administration either to prevent or to treat a presumed AAV capsid immune response. Trial objectives included evaluation of the safety and preliminary efficacy of SPK-8011 and of the expression and durability of factor VIII.

RESULTS:

The median safety observation period was 36.6 months (range, 5.5 to 50.3). A total of 33 treatment-related adverse events occurred in 8 participants; 17 events were vector-related, including 1 serious adverse event, and 16 were glucocorticoid-related. Two participants lost all factor VIII expression because of an anti-AAV capsid cellular immune response that was not sensitive to immune suppression. In the remaining 16 participants, factor VIII expression was maintained; 12 of these participants were followed for more than 2 years, and a one-stage factor VIII assay showed no apparent decrease in factor VIII activity over time (mean [±SD] factor VIII activity, 12.9±6.9% of the normal value at 26 to 52 weeks when the participants were not receiving glucocorticoids vs. 12.0±7.1% of the normal value at >52 weeks after vector administration; 95% confidence interval [CI], -2.4 to 0.6 for the difference between matched pairs). The participants had a 91.5% reduction (95% CI, 88.8 to 94.1) in the annualized bleeding rate (median rate, 8.5 events per year [range, 0 to 43.0] before vector administration vs. 0.3 events per year [range, 0 to 6.5] after vector administration).

CONCLUSIONS:

Sustained factor VIII expression in 16 of 18 participants who received SPK-8011 permitted discontinuation of prophylaxis and a reduction in bleeding episodes. No major safety concerns were reported. (Funded by Spark Therapeutics and the National Heart, Lung, and Blood Institute; ClinicalTrials.gov numbers, NCT03003533 and NCT03432520.).

News (Medical) associated with SPK-8011

21 Apr 2025

·www.prnewswire.com

Roche's HEMLIBRA Achieves Significant Uptake Across Key Hemophilia A Markets | DelveInsight

HEMLIBRA has strong market potential due to its unique, subcutaneous prophylactic treatment for Hemophilia A, including patients with and without factor VIII inhibitors. With increasing global diagnosis rates and expanding access in emerging markets, HEMLIBRA is poised for continued growth. LAS VEGAS, April 21, 2025 /PRNewswire/ -- DelveInsight's " HEMLIBRA Market Size, Forecast, and Market Insight Report" highlights the details around HEMLIBRA, a bispecific factor IXa- and factor X-directed antibody indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients ages newborn and older with hemophilia A (congenital factor VIII deficiency) with or without factor VIII inhibitors. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of HEMLIBRA. The report also highlights the historical and forecasted sales from 2020 to 2034 segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Chugai/Genentech/Roche's HEMLIBRA (emicizumab) Overview HEMLIBRA is a bispecific antibody that targets both factor IXa and factor X, two key proteins in the body's natural blood clotting process. Bridging these proteins it helps restore clotting function in individuals with hemophilia A. HEMLIBRA is used as a preventive treatment and is administered as a ready-to-use subcutaneous injection. It can be given once a week, every two weeks, or every four weeks—following an initial four-week period of weekly doses. Developed by Chugai Pharmaceutical Co., Ltd., HEMLIBRA is co-developed internationally by Chugai, Roche, and Genentech. In the U.S., Genentech markets the drug under the name HEMLIBRA (emicizumab-kxwh), with "kxwh" assigned per FDA guidance on naming biological products. HEMLIBRA can be prescribed in various dosage regimens. The initial loading dose is 3 mg/kg administered via subcutaneous injection once weekly for the first four weeks. This is followed by one of the following maintenance dosing options: 1.5 mg/kg once every week, 3 mg/kg once every two weeks, or 6 mg/kg once every four weeks The choice of maintenance dose should be guided by the healthcare provider's judgment, taking into account the potential for improved patient adherence with certain regimens. The prophylactic use of bypassing agents should be stopped the day before initiating HEMLIBRA prophylaxis. Factor VIII (FVIII) prophylaxis may be continued during the first week of HEMLIBRA treatment. Learn more about HEMLIBRA projected market size for hemophilia A @ HEMLIBRA Market Potential Hemophilia A is marked by bleeding episodes, though in mild and moderate cases, these initial episodes are often less intense, which can lead to delays in diagnosis as patients may not seek medical attention promptly. Moreover, early detection is frequently hindered by the lack of access to adequate diagnostic facilities. In 2023, the number of treated prevalent Hemophilia A cases across the 7MM was estimated at around 45,000, with projections indicating growth throughout the forecast period, according to DelveInsight. Among hemophilia A patients—whether or not they have inhibitors—those without inhibitors represent a larger portion of the population. The standard treatment approach focuses on replacing the missing clotting factor, FVIII, to support normal clot formation and minimize or prevent bleeding episodes. Initially, FVIII replacement was sourced from whole blood and plasma, but advances in biotechnology led to the development of recombinant FVIII (rFVIII) therapies, significantly enhancing disease management by offering more precise and efficient options. More recently, innovative therapies using novel mechanisms to prevent bleeding are expanding the treatment arsenal. Several approved therapies for hemophilia A include ALTUVIIIO (Sanofi/Sobi), ROCTAVIAN (BioMarin), OBIZUR (Takeda), ALHEMO (Novo Nordisk), HEMLIBRA (Chugai/Genentech/Roche), ESPEROCT (Novo Nordisk), JIVI (Bayer), WILATE (Octapharma), and ADYNOVATE (Takeda), among others. Notably, ROCTAVIAN, a gene therapy developed by BioMarin, represents a major advancement—it offers a one-time treatment by introducing functional FVIII genes into the patient's cells, potentially restoring normal clotting ability and reducing the need for regular injections. With a wholesale acquisition cost of USD 2.9 million, BioMarin has already facilitated reimbursement in Europe and administered treatment to a patient in Germany in Q2 2023 at a discounted price of approximately USD 900,000. Looking ahead, the hemophilia A treatment landscape is expected to evolve significantly from 2024 to 2034, fueled by the introduction of new therapies currently in development. According to DelveInsight, the hemophilia A market across the 7MM is projected to grow from USD 11.1 billion in 2023 at a robust CAGR through 2034. Nonetheless, factors like delayed diagnosis, limited awareness, and high treatment costs may pose barriers to market growth. Discover more about the hemophilia A market in detail @ Hemophilia A Market Report Emerging Competitors of HEMLIBRA Emerging therapies such as SPK-8011 (Roche [Spark Therapeutics]), fitusiran (Sanofi), marstacimab (Pfizer), Mim8 (Novo Nordisk), and others are being developed to provide safe and effective treatment options. Companies like GeneVentiv Therapeutics are also working on advancements like Adeno-associated Virus (AAV)-based universal gene therapy for hemophilia (GENV-HEM [AAV8.Fva]). Fitusiran is a subcutaneously administered small interfering RNA (siRNA) therapy under development for the prophylactic treatment of hemophilia A and B, regardless of the presence of inhibitors. Its mechanism involves reducing levels of antithrombin—a protein that inhibits clot formation—to enhance thrombin generation, restore hemostatic balance, and prevent bleeding episodes. In January 2018, Sanofi and Alnylam Pharmaceuticals restructured their RNAi therapeutics collaboration to better align and accelerate the development and commercialization of select treatments for rare genetic diseases. Through this revised agreement, Sanofi obtained global rights to develop and commercialize fitusiran. Marstacimab (PF-06741086) is a monoclonal IgG1 antibody that targets the Kunitz 2 domain of the Tissue Factor Pathway Inhibitor (TFPI). It is being developed to prevent or reduce bleeding events in patients with severe hemophilia A or B (defined as <1% activity of Factor VIII or IX), whether or not they have inhibitors. The FDA granted Fast Track Designation to marstacimab in September 2019 for its potential use in patients with inhibitors. In December 2023, Pfizer announced that the FDA had accepted its Biologics License Application (BLA) for marstacimab, supported by data from the Phase III BASIS study involving patients without FVIII or FIX inhibitors. BBM-H803 is Belief BioMed's first gene therapy candidate for hemophilia A and its second product to gain Investigational New Drug (IND) approval from China's National Medical Products Administration (NMPA). In December 2022, the U.S. FDA granted Orphan Drug Designation to BBM-H803. To know more about the number of competing drugs in development, visit @ HEMLIBRA Market Positioning Compared to Other Drugs Key Milestones of HEMLIBRA In June 2022, Chugai received approval in Japan for acquired Hemophilia A based on a domestic Phase III AGEHA study. In October 2018, HEMLIBRA was approved by the US FDA for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages newborn and older, with hemophilia A without factor VIII inhibitors (FVIII). However, this therapy, which was co-developed by Genentech, Chugai, and Roche, was already FDA-approved in 2017 for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with hemophilia A with FVIII inhibitors In April 2018, the US FDA granted breakthrough therapy designation to Genentech's HEMLIBRA for people with hemophilia A without factor VIII inhibitors. In October 2014, the US FDA granted orphan drug designation to HEMLIBRA for the treatment of Hemophilia A. Discover how HEMLIBRA is shaping the hemophilia A treatment landscape @ HEMLIBRA Cost HEMLIBRA Market Dynamics HEMLIBRA developed by Roche/Genentech, has significantly reshaped the hemophilia A treatment landscape since its approval. Designed as a bispecific monoclonal antibody, HEMLIBRA mimics the function of factor VIII by bridging activated factor IX and factor X, offering prophylactic treatment for patients with hemophilia A with or without factor VIII inhibitors. Its subcutaneous administration and less frequent dosing schedule (weekly, biweekly, or monthly) offer considerable convenience compared to traditional intravenous factor replacement therapies, making it a preferred option for both patients and caregivers. The market dynamics for HEMLIBRA have been largely influenced by its clinical efficacy, improved patient compliance, and strong safety profile. Since its launch, it has rapidly gained market share, especially among inhibitor patients where treatment options were limited and complex. In non-inhibitor patients as well, the shift away from frequent IV infusions has led to strong adoption rates, allowing HEMLIBRA to cannibalize traditional recombinant factor VIII therapies. Furthermore, its entry has pressured pricing and market share dynamics across the hemophilia therapeutic space, triggering strategic shifts by legacy players like Takeda, Sanofi, and Bayer. However, HEMLIBRA's market leadership is being challenged by the evolving competitive landscape. The emergence of gene therapies, which offer the potential for long-term or even curative outcomes, introduces a new dimension to treatment decisions. While some gene therapies are still in early stages of adoption and face challenges related to durability, safety, and patient eligibility, they represent a looming threat to the long-term dominance of HEMLIBRA, particularly in younger, eligible patient populations. Going forward, the market trajectory for HEMLIBRA will depend on several factors: expanding label indications (such as pediatric use), long-term safety and efficacy data, continued pricing negotiations with payers, and how effectively Roche can defend its position against next-generation therapies. Additionally, with increasing emphasis on patient-centric outcomes and value-based care, HEMLIBRA's ability to demonstrate long-term cost-effectiveness versus both traditional and novel competitors will be key to sustaining its market share. Dive deeper to get more insight into HEMLIBRA's strengths & weaknesses relative to competitors @ HEMLIBRA Market Drug Report Table of Contents Related Reports Hemophilia A Market Hemophilia A 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 hemophilia A companies, including BioMarin Pharmaceutical, Roche (Spark Therapeutics), ApcinteX, Novo Nordisk, Sanofi (Genzyme), Alnylam Pharmaceuticals, Pfizer, Sangamo Therapeutics, Bayer, Ultragenyx Pharmaceutical, among others. Hemophilia A Pipeline Hemophilia A Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key hemophilia B companies, including UniQure Biopharma B.V., CSL Behring, Pfizer, Spark Therapeutics, Genzyme, a Sanofi Company, Alnylam Pharmaceuticals, Novo Nordisk, ApcinteX Ltd, Freeline Therapeutics, Sangamo Therapeutics, among others. Hemophilia B Market Hemophilia B Market Insight, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of market trends, market drivers, market barriers, and key hemophilia B companies such as Gem Pharmaceuticals, Lytix Biopharma, Incyte Corporation, Daiichi Sankyo, NantPharma, Iovance Biotherapeutics, Agenus, Eli Lilly and Company, Adaptimmune, Aadi, AVEO Pharmaceuticals, Amgen, among others. Hemophilia B Pipeline Hemophilia B Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key hemophilia B companies, including Shanghai Vitalgen BioPharma, Belief BioMed, TiumBio, Jiangsu Gensciences, Centessa Pharmaceuticals, Takeda, Sanofi, GC Biopharma Corp, Equilibra Bioscience LLC, Regeneron Pharmaceuticals, ISU Abxis, 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

Orphan DrugDrug ApprovalPhase 3Fast TrackBreakthrough Therapy

11 Mar 2025

·www.biospace.com

Roche Absorbs $2.4B Impairment in Overhaul of Spark Gene Therapy Unit

iStock, Besiki Kavtaradze Roche acquired Spark Therapeutics in 2019 for $4.8 billion. Roche is currently enacting a “ fundamental reorganisation ” of its gene therapy subsidiary Spark Therapeutics amid broader shifts in its pharmaceuticals business. The move, first revealed in the company’s financial report published in January 2025, comes after a strategic review of Spark’s prospects in the second half of 2024. Roche at the time was simultaneously implementing “restructuring activities” at Spark, which cost the company about $185 million last year. Roche has yet to settle on the specifics of the reorganization initiative, though it will involve the integration of some of Spark’s operations into the group’s broader Pharmaceuticals Division, as per the financial report. Roche’s preliminary estimate puts its restructuring costs at around $341 million. In connection with Spark’s overhaul, Roche will incur approximately $2.4 billion in goodwill impairment costs. This sum takes into consideration the $4.8 billion it paid to acquire the gene therapy specialist in 2019, as well as the current and future revenues and costs associated with the unit. “There was no surplus from the estimated future revenues of the Spark Therapeutics business to support the carrying value of the goodwill,” Roche wrote in its financial report, “neither were there any significant future synergistic benefits to other products within the Pharmaceuticals Division.” Roche’s decision to acquire Spark in 2019 rested heavily on two assets: the FDA-approved gene therapy Luxturna, indicated for a rare and heritable form of blindness, and the investigational SPK-8011, a one-time treatment for hemophilia A. At the time, SPK-8011 had just elicited a 97% response rate in patients with the bleeding disorder, giving Spark the push it needed to take the asset into late-stage studies. In December 2024, however, Roche shelved SPK-8011, as per an update to its federal clinical trials page that tags the Phase III trial of the asset as “withdrawn.” In a statement to Fierce Biotech at the time, a Roche spokesperson said that the decision to discontinue the SPK-8011 study is connected to a newer investigational therapy with an “enhanced function FVIII variant.” Roche also previously axed the Spark assets SPK-8016 for hemophilia A and SPK-3006 for Pompe disease, a rare genetic condition that damages muscles and nerves. Spark is also conducting research into eye, liver and neurodegenerative diseases, according to its pipeline . Luxturna has similarly been disappointing. In 2024, the gene therapy brought in just under $20.5 million , representing a 59% year-on-year decline in sales. Elsewhere in its gene therapy efforts, Roche in October 2024 put $1 billion on the line —on top of a $50 million upfront payment—in a back-heavy deal with Dyno Therapeutics to develop next-generation gene therapies for neurological indications.

AcquisitionGene TherapyPhase 3

16 Dec 2024

·www.fiercebiotech.com

Roche mothballs another hemophilia A gene therapy under Spark amid plans to debut new hematologic asset

The move comes after a phase 3 study of the gene therapy candidate was listed as “withdrawn” on ClinicalTrials.gov last week.\n Following the apparent elimination of a late-stage gene therapy study last week, Swiss drug juggernaut Roche has confirmed that it’s axing a hemophilia candidate inherited in its 2019 buyout of Spark Therapeutics.In an emailed statement, a Roche spokesperson told Fierce Biotech that the company is discontinuing further development of Spark’s experimental hemophilia A gene therapy dirloctocogene samoparvovec, also known as SPK-8011.The move comes after the entry for the candidate’s phase 3 study was listed as “withdrawn” last week on the U.S.’ online clinical trial repository. The entry on ClinicalTrials.gov attributes the withdrawal to a \"strategic decision\" by Roche. The terminated study, dubbed KEYSTONE 1, was launched in March but never actually enrolled any patients, according to ClinicalTrials.gov.Roche’s spokesperson confirmed the termination of the study and explained that the company is mothballing SPK-8011 as it introduces a new, enhanced function factor VIII (FVIII) hemophilia A candidate to its gene therapy pipeline.“This decision is based on our belief that an enhanced function FVIII variant has the potential to address remaining unmet needs and reduce the treatment burden for patients,” the spokesperson explained. “This decision builds on the promising results seen in the phase 1/2 dirloctocogene samoparvovec study, which assessed the safety and efficacy of the factor VIII gene transfer treatment in individuals with hemophilia A, demonstrating favorable safety, durability and predictability using a low-dose approach.”The pharma has not yet incorporated this new program into its online pipeline, which was last updated Oct. 23 and still lists SPK-8011. Roche first got its hands on SPK-8011 and Spark itself when it purchased the Philadelphia gene therapy specialist for $4.3 billion in 2019. That said, the Roche unit’s gene therapy ambitions have run into turbulence on multiple occasions since then.Last year, Roche had to write down (PDF) the value of the now-terminated SPK-8011, incurring a charge of 519 million Swiss francs ($606 million at the time) over revised sales forecasts around the personalized medicine candidate.Shortly thereafter, the pharma removed a different hemophilia A gene therapy, known as RG6358 or SPK-8016, from its pipeline. The axed candidate had been in early clinical-stage development for use in up to 30% of people with severe or moderately severe hemophilia A who have inhibitors to FVIII.More recently, Roche punted Spark’s Pompe disease gene therapy candidate SPK-3006 from its pipeline this July, which the company credited to a “strategic portfolio prioritization within Spark.”At the time, a Roche spokesperson added that Pompe disease already boasts an established standard of care and multiple different companies are pushing potential gene therapy solutions through the clinic. Elsewhere on the personalized medicine front, Roche in October inked a new deal with Dyno Therapeutics that could be worth more than $1 billion. Under the pact, Dyno—with whom Roche struck a separate gene therapy accord in 2020—is on deck to design novel adeno-associated virus vectors with “improved functional properties” as delivery tools for gene therapies.Roche hopes to use Dyno’s technologies, which leverage artificial intelligence and high-throughput in vivo data, to target neurological diseases.

AcquisitionGene TherapyPhase 3License out/in

100 Deals associated with SPK-8011

R&D Status

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Hemophilia A	Phase 3	United States	Spark Therapeutics, Inc.	14 Feb 2019

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03432520 \| NCT03003533 (ISTH2023)	Phase 1/2	Hemophilia A FVIII activity	-	Dirloctocogene samoparvovec (SPK-8011)	zdyquprpcd(pqdubvjtgr) = Some HJHS components showed improvements, with proportions of participants with severe symptoms decreasing over time rzmhjhaziv (quqthtvajh )	-	24 Jun 2023
NCT03432520 \| NCT03003533 (ASH2022) Manual	Phase 1/2	Hemophilia A	21	SPK-8011	gpbpddzmse(rremvyncrf) = in saliva, semen, serum, urine, and PBMCs was 2 (1-2) weeks, 2 (1-2) weeks, 2 (2-3) weeks, 2 (1-2) weeks, and 5 (4-6) weeks, respectively hycvjbifju (dhruzjzmcd )	Positive	12 Dec 2022
NCT03432520 \| NCT03003533 (ASH 12022 \| ASH 22022) Manual	Phase 1/2	Hemophilia A	23	SPK-8011	pdwkugccqh(uwijabfiza) = kwzgknsrry tdcfvpyghz (rokmobtpwf ) View more	Positive	15 Nov 2022
NCT03003533 (ISTH2021) Manual	Phase 1/2	Hemophilia A	17	SPK-8011	osgseusqoh(tqmfkgtuwx) = rprcskweai hdkpslmmnw (awnblveaia ) View more	Positive	17 Jul 2021
ISTH2021	Phase 1/2	Hemophilia A	17	SPK-8011	pochmnoveb(ptpptnyixa) = wggiemrsau qfrvqixpql (gbujpxdlkg ) View more	-	17 Jul 2021
ASH 12018 \| ASH 22018 Manual	Phase 1/2	Hemophilia A	12	SPK-8011	dlzryxbjfa(rccerdsjei) = nltuyewupq ixjhnuctog (qwcclvvweg )	Positive	29 Nov 2018
ASH 12018 \| ASH 22018 Manual	Phase 1/2	Hemophilia A	12	SPK-8011	dlzryxbjfa(rccerdsjei) = wgepoxfojk ixjhnuctog (qwcclvvweg )	Positive	29 Nov 2018

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

AI Agents Built for Biopharma Breakthroughs

Accelerate discovery. Empower decisions. Transform outcomes.

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis