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2 Diseases | | 10 Products |
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- || Translarna (ataluren) / PTC Therap, Exondys 51 (eteplirsen) / Sarepta Therap
Review, Journal: Factors Associated with Respiratory Health and Function in Duchenne Muscular Dystrophy: A Systematic Review and Evidence Grading. (Pubmed Central) - Jan 9, 2024
Yet, more research is needed to further delineate sources of respiratory heterogeneity, in particular the genotype-phenotype association and the impact of novel DMD therapies in a real-world setting. Our synthesis and grading should be helpful to inform clinical practice and future research of this heavily burdened patient population.
- || Elevidys (delandistrogene moxeparvovec-rokl) / Sarepta Therap
Enrollment open: ENVOL: A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) - Dec 12, 2023
P2, N=21, Recruiting, Sponsor: Hoffmann-La Roche
Our synthesis and grading should be helpful to inform clinical practice and future research of this heavily burdened patient population. Not yet recruiting --> Recruiting
- | Elevidys (delandistrogene moxeparvovec) / Sarepta Therap, Roche
Journal, Gene therapy: Development of capsid- and genome-modified optimized AAVrh74 vectors for muscle gene therapy. (Pubmed Central) - Dec 4, 2023
Not yet recruiting --> Recruiting The use of the first generation of two different AAV serotype vectors (AAV9 and AAVrh74) in four separate clinical trials failed to be effective in patients with Duchenne muscular dystrophy, although some efficacy was observed in a subset of patients with AAVrh74 vectors leading to US Food and Drug Administration approval (Elevidys)...Here, we report that capsid- and genome-modified optimized AAVrh74 vectors are significantly more efficient in transducing primary human skeletal muscle cells in
- | vesleteplirsen (SRP-5051) / Sarepta Therap, Exondys 51 (eteplirsen) / Sarepta Therap
Trial completion date: MOMENTUM: Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Efficacy (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (clinicaltrials.gov) - Dec 1, 2023
P2, N=62, Active, not recruiting, Sponsor: Sarepta Therapeutics, Inc.
The use of the first generation of two different AAV serotype vectors (AAV9 and AAVrh74) in four separate clinical trials failed to be effective in patients with Duchenne muscular dystrophy, although some efficacy was observed in a subset of patients with AAVrh74 vectors leading to US Food and Drug Administration approval (Elevidys)...Here, we report that capsid- and genome-modified optimized AAVrh74 vectors are significantly more efficient in transducing primary human skeletal muscle cells in Trial completion date: Mar 2025 --> Jan 2029
- ||| Idefirix (imlifidase) / Hansa Biopharma, Sarepta Therap
Enrollment closed, Enrollment change: A Study to Investigate DSA Rebound in Patients Treated with Imlifidase Prior to Transplantation (clinicaltrials.gov) - Dec 1, 2023
P2, N=3, Active, not recruiting, Sponsor: Hansa Biopharma AB
Trial completion date: Mar 2025 --> Jan 2029 Recruiting --> Active, not recruiting | N=12 --> 3
- |||| Exondys 51 (eteplirsen) / Sarepta Therap
Enrollment closed: A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON) (clinicaltrials.gov) - Nov 29, 2023
P3, N=160, Active, not recruiting, Sponsor: Sarepta Therapeutics, Inc.
Recruiting --> Active, not recruiting | N=12 --> 3 Recruiting --> Active, not recruiting
- | Translarna (ataluren) / PTC Therap, Exondys 51 (eteplirsen) / Sarepta Therap
Journal, HEOR: Early Cost-Utility Analysis of Ataluren and Eteplirsen in the Treatment of Duchenne Muscular Dystrophy in Egypt. (Pubmed Central) - Nov 14, 2023
Recruiting --> Active, not recruiting Based on these models, there is a huge gap between the prices of orphan drugs and their value-based prices, which highlights the need for major policy reforms in the assessment and pricing of orphan drugs.
- || Elevidys (delandistrogene moxeparvovec-rokl) / Sarepta Therap
New P2 trial: ENVOL: A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) - Nov 13, 2023
P2, N=21, Not yet recruiting, Sponsor: Hoffmann-La Roche
- Idefirix (imlifidase) / Hansa Biopharma, Sarepta Therap, Rituxan (rituximab) / Biogen, Zenyaku Holdings, Roche
Alloantibody Levels, Immunoglobulin Degradation Products, and Platelet Transfusion Response over Successful Desensitization of Severe Alloimmune Platelet Refractoriness with a Novel IgG-Targ... (SDCC - Halls G-H) - Nov 3, 2023 - Abstract #ASH2023ASH_3601;
METHODS With IRB approval, patient data and samples (D-16; D-1 post-conventional desensitization with rituximab and therapeutic plasma exchange; D0 post-imlifidase dose 1; D2 post-imlifidase dose 2; and D7, D14 and D29 follow-up samples) were collected. These results suggest imlifidase is capable of effective desensitization despite profound baseline sensitization, with rapid cleavage of intact IgG to F(ab
- |||||| Elevidys (delandistrogene moxeparvovec-rokl) / Sarepta Therap
Enrollment open: EXPEDITION: A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study (clinicaltrials.gov) - Oct 30, 2023
P3, N=400, Enrolling by invitation, Sponsor: Sarepta Therapeutics, Inc.
These results suggest imlifidase is capable of effective desensitization despite profound baseline sensitization, with rapid cleavage of intact IgG to F(ab Not yet recruiting --> Enrolling by invitation
- || Elevidys (delandistrogene moxeparvovec) / Sarepta Therap, Roche
Trial completion, Trial completion date: A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) - Oct 25, 2023
P1/2, N=41, Completed, Sponsor: Sarepta Therapeutics, Inc.
Not yet recruiting --> Enrolling by invitation Active, not recruiting --> Completed | Trial completion date: Apr 2026 --> Aug 2023
- | Elevidys (delandistrogene moxeparvovec) / Sarepta Therap, Roche
Journal, Gene therapy: Delandistrogene moxeparvovec gene therapy in ambulatory patients (aged ?4 to <8?years) with Duchenne muscular dystrophy: 1-year interim results from Study SRP-9001-103 (ENDEAVOR). (Pubmed Central) - Oct 22, 2023
P1, P1/2
One-year post-treatment, delandistrogene moxeparvovec was well tolerated, and demonstrated stabilized or improved motor function, suggesting a clinical benefit for patients with Duchenne muscular dystrophy. ANN NEUROL 2023;94:955-968.
- Immunomonitoring requirements in AAV based gene therapy clinical trials: where do we stand? () - Oct 15, 2023 - Abstract #ESGCT2023ESGCT_1057;
Promising results have been obtained with several rAAV products as illustrated by six drugs currently approved (Luxturna, Zolgensma, Roctavian, Hemgenix, Upstaza and Elevidys) in Europe and USA...Finally, we will describe the general work?flow to implement clinical immune monitoring, in line with Good Clinical Laboratory Practices (GCLP) and other regulatory guidelines. Beyond the method validation itself, other steps, such as definition of responsibilities, personnel training or data and material traceability have to be implemented and controlled by the immunology laboratory to assess safety and efficacy endpoints, while ensuring the quality and integrity of the data.
- Idefirix (imlifidase) / Hansa Biopharma, Sarepta Therap
IgG depletion with imlifidase ? a potential way to increase the eligible patient population for AAV based gene therapies () - Oct 15, 2023 - Abstract #ESGCT2023ESGCT_1051;
All in all, antibody depletion with imlifidase could be a solution for patients excluded from participation in gene therapy clinical trials because of pre?formed immunogenicity towards the AAV vector. The therapeutic potential of imlifidase as pre?treatment to gene therapy is currently being evaluated for AAV based gene therapies in collaboration with Sarepta Therapeutics, Genethon and AskBio.*EU/EEA, UK and Switzerland
- Exondys 51 (eteplirsen) / Sarepta Therap
Cas12 DNA editing restores dystrophin expression and muscle function in mouse model of Duchenne muscular dystrophy and demonstrates high editing efficiency in NHPs () - Oct 15, 2023 - Abstract #ESGCT2023ESGCT_930;
Here, we generated a novel humanized DMD mouse model harboring human DMD exon51, which exhibited highly similar phenotypes in patients with DMD...In addition, no in vivo toxicities were observed, including nerve, liver, and kidney injury, in the humanized DMD mice and WT NHPs. Unlike antisense?oligonucleotide?based exon skipping therapy administering throughout a patient's lifetime or AAV gene replacement therapy expressing micro?dystrophins for partial dystrophin functionality, our innovative CRISPR?based hfCas12Max?mediated single?cut approach modifying genomic DNA offers a promising, long?lasting, and "one?and?done" new treatment modality that targets the underlying cause of DMD.
- Elevidys (delandistrogene moxeparvovec-rokl) / Sarepta Therap
T?cell response to SRP?9001 dystrophin transgene in a patient treated with delandistrogene moxeparvovec: A case of immune?mediated myositis () - Oct 15, 2023 - Abstract #ESGCT2023ESGCT_337;
P1
We hypothesize that this combination of factors led to the lack of SRP?9001 protein immunologic tolerance and subsequent IMM in this patient.These results are consistent with findings from clinical trials of other investigational micro?dystrophin GTs, which show that patients with deletions in regions of the DMD gene overlapping those expressed in a given micro?dystrophin may be at an increased risk of a myositis event following GT. Work is currently underway to better understand these risk factors and to find ways to safely administer delandistrogene moxeparvovec to patients with potentially higher?risk DMD mutations.
- Vyondys 53 (golodirsen) / Sarepta Therap, Amondys 45 (casimersen) / Sarepta Therap
Trial completion: A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications. (clinicaltrials.gov) - Oct 12, 2023
P2, N=3, Completed, Sponsor: Kevin Flanigan
Work is currently underway to better understand these risk factors and to find ways to safely administer delandistrogene moxeparvovec to patients with potentially higher?risk DMD mutations. Active, not recruiting --> Completed
- || Review, Journal: Advances in Dystrophinopathy Diagnosis and Therapy. (Pubmed Central) - Oct 4, 2023
Other antisense oligonucleotide drugs in the pipeline include casimersen for exon 45, suvodirsen for exon 51, and golodirsen for exon 53 skipping. Advances in the diagnosis and therapy of dystrophinopathies offer new perspectives for their early discovery and care.
- | Elevidys (delandistrogene moxeparvovec) / Sarepta Therap, Roche
Journal: Delandistrogene moxeparvovec (Elevidys) for Duchenne muscular dystrophy. (Pubmed Central) - Sep 27, 2023
Advances in the diagnosis and therapy of dystrophinopathies offer new perspectives for their early discovery and care. No abstract available
- | bidridistrogene xeboparvovec (SRP-9003) / Sarepta Therap
Enrollment closed, Trial completion date, Trial primary completion date: A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [?-SG] Deficiency) (clinicaltrials.gov) - Sep 21, 2023
P1, N=6, Active, not recruiting, Sponsor: Sarepta Therapeutics, Inc.
No abstract available Enrolling by invitation --> Active, not recruiting | Trial completion date: Jan 2027 --> Aug 2028 | Trial primary completion date: Jan 2026 --> Aug 2028
- || Exondys 51 (eteplirsen) / Sarepta Therap
Review, Journal: The Regulatory Repercussions of Approving Muscular Dystrophy Medications on the Basis of Limited Evidence. (Pubmed Central) - Sep 20, 2023
Despite recent legislative and regulatory changes to the FDA's accelerated approval pathway, the history of eteplirsen and similar drugs points to the need for additional reforms to better balance evidence generation with patient safety and access to promising medications. Lawmakers and regulators should take further action to limit excessive spending on unproven therapies and ensure that drug sponsors conduct robust and timely confirmatory trials after receiving accelerated approval.
- | Elevidys (delandistrogene moxeparvovec) / Sarepta Therap, Roche
Preclinical, Journal: Expression and function of Four AAV-based constructs for dystrophin restoration in the mdx mouse model of duchenne muscular dystrophy. (Pubmed Central) - Sep 19, 2023
Muscle environment was normalized (i.e., reductions in central nucleation), indicating functional and histological advantages of rAAVrh74.MHCK7.micro-dystrophin. Thus, promoter choice and transgene design are critical for optimal dystrophin expression/distribution for maximal functional improvement.
- Elevidys (delandistrogene moxeparvovec) / Sarepta Therap, Roche
Phase classification: A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) - Sep 15, 2023
P1/2, N=41, Active, not recruiting, Sponsor: Sarepta Therapeutics, Inc.
Thus, promoter choice and transgene design are critical for optimal dystrophin expression/distribution for maximal functional improvement. Phase classification: P2 --> P1/2
- || Elevidys (delandistrogene moxeparvovec) / Sarepta Therap, Roche
Review, Journal, Gene therapy: Paving the way for future gene therapies: A case study of scientific spillover from delandistrogene moxeparvovec. (Pubmed Central) - Sep 8, 2023
This impact is even more profound in rare diseases, where developing therapies in isolation may not be possible. This review describes some instances of scientific spillover in healthcare, and specifically gene therapy, using delandistrogene moxeparvovec (SRP-9001), a gene therapy recently approved by the US Food and Drug Administration for the treatment of ambulatory pediatric patients aged 4-5 years with Duchenne muscular dystrophy with a confirmed mutation in the DMD gene, as a case study.
- | Exondys 51 (eteplirsen) / Sarepta Therap
Journal, HEOR, Real-world evidence, Real-world: Real-world evidence of eteplirsen treatment effects in patients with Duchenne muscular dystrophy in the USA. (Pubmed Central) - Sep 1, 2023
Other assessed outcomes favored eteplirsen numerically but did not all reach statistical significance. Eteplirsen-treated patients had reduced rates of multiple healthcare resource utilization measures versus matched controls.
- | Vyondys 53 (golodirsen) / Sarepta Therap, Amondys 45 (casimersen) / Sarepta Therap
Enrollment change, Trial completion date, Trial termination, Trial primary completion date: An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy (clinicaltrials.gov) - Aug 21, 2023
P3, N=171, Terminated, Sponsor: Sarepta Therapeutics, Inc.
N=260 --> 171 | Trial completion date: Aug 2026 --> Jul 2023 | Active, not recruiting --> Terminated | Trial primary completion date: Aug 2026 --> Jul 2023; Participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment. There were no safety concerns with this study.
- || Elevidys (delandistrogene moxeparvovec) / Sarepta Therap, Roche
P1/2 data, Journal, Gene therapy: Long-term safety and functional outcomes of delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy: A phase 1/2a nonrandomized trial. (Pubmed Central) - Aug 14, 2023
P1/2
Gene transfer therapy with delandistrogene moxeparvovec treatment is well tolerated, with a favorable safety profile. Functional improvements are sustained through 4?years, suggesting delandistrogene moxeparvovec may positively alter disease progression.
- || Elevidys (delandistrogene moxeparvovec) / Sarepta Therap, Roche
Review, Journal: Delandistrogene Moxeparvovec: First Approval. (Pubmed Central) - Aug 11, 2023
Delandistrogene moxeparvovec is undergoing clinical development in several countries/regions, including the EU and Japan. This article summarizes the milestones in the development of delandistrogene moxeparvovec leading to this first approval in the USA for the treatment of ambulatory paediatric patients aged 4 through 5
- | DMD exon 52 skipping program / Wave Life Sciences
Preclinical, Journal: Prevention of early-onset cardiomyopathy in Dmd exon 52-54 deletion mice by CRISPR-Cas9-mediated exon skipping. (Pubmed Central) - Aug 7, 2023
Here we employed CRISPR-Cas9 delivered intravenously by adeno-associated virus (AAV) vectors to restore functional dystrophin expression via excision or skipping of exon 55. Remarkably, our exon skipping approach completely prevented onset of the cardiac phenotype in treated mice up to 12
- || Elevidys (delandistrogene moxeparvovec) / Sarepta Therap, Roche
Trial completion date, Trial primary completion date: ENDEAVOR: A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) - Aug 1, 2023
P1, N=58, Enrolling by invitation, Sponsor: Sarepta Therapeutics, Inc.
Remarkably, our exon skipping approach completely prevented onset of the cardiac phenotype in treated mice up to 12 Trial completion date: Jan 2028 --> Jul 2026 | Trial primary completion date: Aug 2023 --> Nov 2024
- |||||||||| Elevidys (delandistrogene moxeparvovec-rokl) / Sarepta Therap
New P3 trial: EXPEDITION: A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study (clinicaltrials.gov) - Aug 1, 2023
P3, N=400, Not yet recruiting, Sponsor: Sarepta Therapeutics, Inc.
- || Exondys 51 (eteplirsen) / Sarepta Therap
PK/PD data, Journal: Safety, tolerability and pharmacokinetics of eteplirsen in young boys aged 6-48 months with Duchenne muscular dystrophy amenable to exon 51 skipping. (Pubmed Central) - Jul 31, 2023
P2
Eteplirsen pharmacokinetics were consistent between both cohorts and with previous clinical experience in boys with DMD > 4 years of age. These data support the safety and tolerability of eteplirsen at the approved 30-mg/kg dose in boys as young as 6 months old.
- | Elevidys (delandistrogene moxeparvovec) / Sarepta Therap, Roche
Journal, Gene therapy: Expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment with delandistrogene moxeparvovec gene therapy in individuals with Duchenne muscular dystrophy. (Pubmed Central) - Jul 27, 2023
P2
These data support the safety and tolerability of eteplirsen at the approved 30-mg/kg dose in boys as young as 6 months old. Results indicate robust expression of SRP-9001 dystrophin and overall stabilization in NSAA up to 2
- |||| Idefirix (imlifidase) / Hansa Biopharma, Sarepta Therap
Enrollment open: PAES-LTFU: A Long-term Follow-up Trial of Patients Previously Treated With Imlifidase Prior to Kidney Transplantation and a Non-comparative Transplanted Patient Group (clinicaltrials.gov) - Jul 20, 2023
P3, N=150, Recruiting, Sponsor: Hansa Biopharma AB
Results indicate robust expression of SRP-9001 dystrophin and overall stabilization in NSAA up to 2 Not yet recruiting --> Recruiting
- | DMD exon 52 skipping program / Wave Life Sciences, Exondys 51 (eteplirsen) / Sarepta Therap
Preclinical, Journal: Systemic deletion of DMD exon 51 rescues clinically severe Duchenne muscular dystrophy in a pig model lacking DMD exon 52. (Pubmed Central) - Jul 14, 2023
Our findings indicate that ubiquitous deletion of DMD exon 51 in DMD?52 pigs largely rescues the rapidly progressing, severe muscular dystrophy and the reduced cardiac function of this model. Long-term follow-up studies of DMD?51-52 pigs will show if they develop symptoms of the milder BMD.
- |||||||| Idefirix (imlifidase) / Hansa Biopharma, Sarepta Therap
New P3 trial: PAES-LTFU: A Long-term Follow-up Trial of Patients Previously Treated With Imlifidase Prior to Kidney Transplantation and a Non-comparative Transplanted Patient Group (clinicaltrials.gov) - Jul 10, 2023
P3, N=150, Not yet recruiting, Sponsor: Hansa Biopharma AB
- ||||| Vyondys 53 (golodirsen) / Sarepta Therap, Amondys 45 (casimersen) / Sarepta Therap
Enrollment closed: An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy (clinicaltrials.gov) - Jul 6, 2023
P3, N=260, Active, not recruiting, Sponsor: Sarepta Therapeutics, Inc.
Long-term follow-up studies of DMD?51-52 pigs will show if they develop symptoms of the milder BMD. Enrolling by invitation --> Active, not recruiting
- | Elevidys (delandistrogene moxeparvovec) / Sarepta Therap, Roche
Trial primary completion date: ENDEAVOR: A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) - Jul 5, 2023
P1, N=46, Enrolling by invitation, Sponsor: Sarepta Therapeutics, Inc.
Enrolling by invitation --> Active, not recruiting Trial primary completion date: May 2023 --> Aug 2023
- |||| Elevidys (delandistrogene moxeparvovec) / Sarepta Therap, Roche
Enrollment open: ENVISION: A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) - Jul 5, 2023
P3, N=148, Recruiting, Sponsor: Sarepta Therapeutics, Inc.
Trial primary completion date: May 2023 --> Aug 2023 Not yet recruiting --> Recruiting
- Idefirix (imlifidase) / Hansa Biopharma, Sarepta Therap
Enrollment open: DINKY: Imlifidase Prior to Kidney Transplant in Highly Sensitised Children (clinicaltrials.gov) - Jun 22, 2023
P2, N=10, Recruiting, Sponsor: Hansa Biopharma AB
Not yet recruiting --> Recruiting Not yet recruiting --> Recruiting
- || Review, Journal: The potential role and mechanism of circRNA/miRNA axis in cholesterol synthesis. (Pubmed Central) - Jun 20, 2023
Suppressing HMGCR, SQLE, and miR-122 with circRNA_ABCA1, circ-PRKCH, circEZH2, circRNA-SCAP, and circFOXO3 are the promising therapeutic target for drug development, specifically the circFOXO3. This review focuses on the role and mechanism of the circRNA/miRNA axis in cholesterol synthesis in the hope of providing knowledge to identify new targets.
- | LYS-SAF302 / Lysogene, Sarepta Therap
Journal, Gene therapy: Focal lesions following intracerebral gene therapy for mucopolysaccharidosis IIIA. (Pubmed Central) - Jun 19, 2023
This alters extracellular matrix composition, depletes heparan sulfate, impairs astrocyte and oligodendrocyte function, and causes cystic white matter degeneration at the site of highest gene expression. The AAVance trial results will reveal the potential benefit-risk ratio of this therapy.
- | Exondys 51 (eteplirsen) / Sarepta Therap, Kyndrisa (drisapersen) / BioMarin
Journal: Next Generation Exon 51 Skipping Antisense Oligonucleotides for Duchenne Muscular Dystrophy. (Pubmed Central) - Jun 8, 2023
These dystrophin levels allowed for normalization of creatine kinase (CK) and lactate dehydrogenase (LDH) levels, and improved motor function in hDMDdel52/mdx mice. As no major safety observation was obtained, the improved therapeutic index of these next generation AONs is encouraging for further (pre)clinical development.
- | Exon 43 / Sarepta Therap
Preclinical, Journal: Rapid and Reliable Quantification of Prime Editing Targeting Within the Porcine ABCA4 Gene Using a Bioluminescence Resonance Energy Transfer-Based Sensor. (Pubmed Central) - Jun 8, 2023
We identified nine working pegRNAs, and in combination with ngRNAs, we achieved a correction rate of up to ?92% measured with the BRET-based reporter system. Our data prove the high efficiency of prime editors to correct mutations and highlight the importance of optimal ngRNA design, thus offering a promising editing tool to correct ABCA4 mutations in the disease context.
- | delandistrogene moxeparvovec (SRP-9001) / Sarepta Therap, Roche
Journal, Gene therapy: Assessing the value of delandistrogene moxeparvovec (SRP-9001) gene therapy in patients with Duchenne muscular dystrophy in the United States. (Pubmed Central) - Jun 1, 2023
Varying the benefit discount rate to account for the single administration increased the estimated value to #$5M, assuming $500,000/evLYG. In this early economic model, delandistrogene moxeparvovec increases evLYs versus SoC and begins to inform its potential value from a healthcare perspective.
- |||||||| Elevidys (delandistrogene moxeparvovec) / Sarepta Therap, Roche
New P3 trial: ENVISION: A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) - May 31, 2023
P3, N=148, Not yet recruiting, Sponsor: Sarepta Therapeutics, Inc.
- delandistrogene moxeparvovec (SRP-9001) / Sarepta Therap, Roche
Practical considerations for delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy (DMD) (Foyer 3B, 3rd Floor) - May 29, 2023 - Abstract #EPNS2023EPNS_832;
P1, P1/2,
The practical considerations of delandistrogene moxeparvovec treatment provided are based on clinical trial data and aim to aid physicians treating patients with DMD. Implementation of these practical considerations is recommended.
- delandistrogene moxeparvovec (SRP-9001) / Sarepta Therap, Roche
One-year data from ENDEAVOR, a Phase 1b trial of delandistrogene moxeparvovec in patients with DMD (Foyer 3B, 3rd Floor) - May 29, 2023 - Abstract #EPNS2023EPNS_831;
P1
Implementation of these practical considerations is recommended. Cohort 1 data suggest the safety and efficacy profile of intended commercial process delandistrogene moxeparvovec material is consistent with that of clinical process material, with no new safety signals identified, and robust SRP-9001 dystrophin expression and positive functional benefit observed.
- Vyondys 53 (golodirsen) / Sarepta Therap, Exondys 51 (eteplirsen) / Sarepta Therap, Amondys 45 (casimersen) / Sarepta Therap
Interim Analysis of EVOLVE: A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice (Foyer 3B, 3rd Floor) - May 29, 2023 - Abstract #EPNS2023EPNS_815;
Cohort 1 data suggest the safety and efficacy profile of intended commercial process delandistrogene moxeparvovec material is consistent with that of clinical process material, with no new safety signals identified, and robust SRP-9001 dystrophin expression and positive functional benefit observed. These real-world data from the interim analysis of EVOLVE support the safety profiles and will continue to describe long-term clinical outcomes of eteplirsen, golodirsen, and casimersen.
- delandistrogene moxeparvovec (SRP-9001) / Sarepta Therap, Roche
EMBARK, a Phase 3 trial evaluating safety and efficacy of delandistrogene moxeparvovec in DMD: Study design and baseline characteristics (Foyer 3B, 3rd Floor) - May 29, 2023 - Abstract #EPNS2023EPNS_798;
P3
These real-world data from the interim analysis of EVOLVE support the safety profiles and will continue to describe long-term clinical outcomes of eteplirsen, golodirsen, and casimersen. EMBARK will provide placebo-controlled information on the efficacy and safety of delandistrogene moxeparvovec in a large population of ambulatory patients with DMD aged ?4 to <8 years.