Sarepta Therapeutics
🇺🇸United States
- Country
- 🇺🇸United States
- Ownership
- Private, Public
- Established
- 1980-01-01
- Employees
- 1.3K
- Market Cap
- $13.4B
- Website
- http://www.sarepta.com
- Introduction
Sarepta Therapeutics, Inc. is a commercial-stage biopharmaceutical company, which is engaged in the discovery and development of therapeutics for the treatment of rare diseases. The company was founded on July 22, 1980, and is headquartered in Cambridge, MA.
CRISPR Gene Editing Breakthrough Saves Baby with Ultra-Rare Metabolic Disorder
• Doctors at Children's Hospital of Philadelphia successfully treated a baby with severe CPS1 deficiency using a personalized CRISPR base-editing therapy, marking a first-of-its-kind approach for this rare metabolic disorder.
• The experimental treatment, developed within just six months of diagnosis, corrected the infant's specific genetic mutation by delivering edited DNA to liver cells via lipid nanoparticles, allowing him to reduce medication and process more dietary protein.
• This breakthrough demonstrates the potential for creating customized gene therapies for millions with rare genetic diseases, with researchers suggesting costs comparable to liver transplantation and possibilities for treating numerous other conditions.
Inozyme's INZ-701 Shows Promise in ENPP1 Deficiency Trial with Sustained Phosphate Increases
• Interim data from Inozyme Pharma's ENERGY 3 pivotal trial demonstrates INZ-701's potential to modify disease course in pediatric ENPP1 Deficiency patients, with sustained phosphate increases and favorable safety profile.
• The trial is progressing well with no patient dropouts, dose adjustments, or discontinuations, and remains on track for topline data in Q1 2026, positioning INZ-701 to potentially become the first approved therapy for this rare disease.
• Inozyme has appointed Petra Duda, M.D., Ph.D. as Chief Medical Officer, bringing over two decades of expertise in rare disease clinical development as the company advances toward late-stage development milestones.
Ten-Year APHINITY Data Shows Perjeta-Based Regimen Reduces Death Risk by 17% in HER2-Positive Early Breast Cancer
• Long-term follow-up data from the Phase III APHINITY trial demonstrates a statistically significant 17% reduction in risk of death when adding Perjeta (pertuzumab) to Herceptin (trastuzumab) and chemotherapy in early-stage HER2-positive breast cancer.
• The benefit was more pronounced in patients with lymph node-positive disease, showing a 21% reduction in death risk, reinforcing the regimen's value as a standard-of-care treatment in the curative setting.
• After ten years, 91.6% of patients receiving the Perjeta-based regimen were alive compared to 89.8% in the control group, with the previously reported invasive disease-free survival benefit maintained without new safety concerns.
Azafaros Secures €132M Series B Financing to Advance Rare Lysosomal Storage Disorder Treatments
• Azafaros has raised €132 million in an oversubscribed Series B financing round led by Jeito Capital and co-led by Forbion Growth to advance two Phase 3 clinical programs for nizubaglustat.
• Nizubaglustat, a first-in-class dual-acting drug candidate, will enter Phase 3 trials later this year for Niemann-Pick disease Type C and GM1/GM2 gangliosidoses, rare genetic disorders that cause progressive neurodegeneration.
• The drug has received multiple regulatory designations including Orphan Drug Designation in both the US and Europe and Fast Track status in the US, highlighting its potential to address significant unmet needs in these fatal pediatric conditions.
Taiwan's Formosa Pharmaceuticals Signs Exclusive Licensing Deal with Almac Discovery for ADC Development
• Taiwan-based Formosa Pharmaceuticals has entered into an exclusive licensing agreement with Northern Ireland's Almac Discovery to develop novel antibody-drug conjugates for cancer treatment.
• The partnership combines Formosa's antibody expertise with Almac's proprietary drug-linker technology, aiming to address unmet needs in oncology with more targeted therapeutic approaches.
• This collaboration represents a significant expansion of Taiwan's growing presence in the global biopharmaceutical sector, particularly in the rapidly evolving ADC market.
FDA Commissioner Makary Proposes New Approval Pathway for Rare Disease Drugs
• FDA Commissioner Marty Makary has announced plans for a new regulatory pathway to expedite approvals for rare disease treatments, potentially without requiring randomized controlled trials.
• The proposed "plausible mechanism" pathway would include conditional approvals for therapies with scientifically sound physiological rationales, coupled with a comprehensive patient surveillance system.
• Makary also addressed public distrust in health authorities, announcing plans to replace the self-reported VAERS system with a more robust "Health Information Exchange" to better monitor treatment complications.
Next-Generation Gene Therapies: Evolving Beyond Viral Vectors Towards More Affordable, Sustainable Solutions
• Despite 32 approved gene therapies globally, the industry faces significant challenges in safety, efficacy, and affordability, prompting development of novel delivery systems beyond traditional viral vectors.
• Companies are advancing non-viral delivery platforms including exosomes, lipid nanoparticles, and hydrophilic nanoparticles that offer cost-effective alternatives with reduced immunogenicity and potential for repeat dosing.
• Next-generation gene editing technologies like Prime Editing and CRISPR variants are emerging as more precise alternatives to traditional CRISPR-Cas9, with Prime Medicine's PM359 for chronic granulomatous disease advancing to clinical trials.
FDA's Cell and Gene Therapy Champion Peter Marks Departs, Leaving Industry at Critical Juncture
• Peter Marks, head of FDA's Center for Biologics Evaluation and Research since 2016, has resigned, leaving cell and gene therapy developers without their biggest regulatory advocate during a challenging investment period.
• Under Marks' leadership, the FDA approved dozens of cell and gene therapies including the first gene therapy, first cellular treatment for cancer, and first CRISPR gene editing medicine, establishing flexible regulatory frameworks for these novel treatments.
• Despite concerns about regulatory uncertainty following Marks' departure, incoming FDA Commissioner Marty Makary has signaled support for conditional approval pathways for rare disease treatments where randomized controlled trials aren't feasible.
New Report Reveals Critical Insights into Clinical-Stage Pharma Partnerships from 2020-2025
• A comprehensive new industry report analyzes over 1,670 clinical-stage partnering agreements in pharma and biotech from 2020-2025, providing unprecedented access to deal structures and financial terms.
• The report reveals detailed intelligence on how licensing agreements typically grant exclusive rights across Phase I-III trials, with multi-component structures involving collaborative R&D and commercialization strategies.
• Business development professionals can now access actual contract documents and payment triggers often missing from press releases, enabling more effective negotiation strategies and competitive deal structuring.
Sarepta's Gene Therapy for Limb-Girdle Muscular Dystrophy Shows Sustained 5-Year Safety Profile
• Bidridistrogene xeboparvovec (SRP-9003) demonstrated a favorable safety profile over 4-5 years in a phase 1/2 trial for limb-girdle muscular dystrophy 2E/R4, with most treatment-related adverse events occurring within 90 days post-infusion.
• The gene therapy showed robust beta-sarcoglycan expression (36.2% in low-dose cohort, 62.1% in high-dose cohort) and significant reductions in serum creatine kinase levels (>90%) that were maintained through 2 years post-treatment.
• Patients treated with bidridistrogene xeboparvovec demonstrated improved motor function compared to natural history cohorts, with the therapy now advancing to a phase 3 EMERGENE trial that completed enrollment in December 2024.
Muscular Dystrophy Research Reaches Turning Point: Regeneration Emerges as Next Frontier
• The Muscular Dystrophy Association's 75th anniversary conference in March 2025 will showcase remarkable progress in treatment options, with multiple approved drugs now able to slow or halt disease progression.
• Regenerative medicine has emerged as the next critical frontier, with dedicated sessions exploring muscle regeneration for muscular dystrophies and neural regeneration for ALS and spinal cord injuries.
• Gene therapy advancements will be prominently featured, including real-world clinical experiences with approved therapies like Zolgensma for SMA and Elevidys for Duchenne muscular dystrophy.
Sarepta Seeks Accelerated Approval for DMD Gene Therapy SRP-9001
• Sarepta Therapeutics has submitted SRP-9001 (delandistrogene moxeparvovec) to the FDA for accelerated approval to treat ambulatory Duchenne muscular dystrophy (DMD) patients.
• The filing is based on positive data from early-stage studies, showing improvements in clinical function and a consistent safety profile, while awaiting Phase 3 EMBARK results.
• SRP-9001, a one-time gene therapy, delivers a shortened dystrophin gene via an AAV vector, addressing the underlying genetic defect in DMD patients.
• If approved, SRP-9001 would offer a one-time treatment option for DMD, contrasting with Sarepta's existing chronic exon-skipping therapies.
Capricor Therapeutics Completes FDA Submission for Deramiocel in DMD Cardiomyopathy
• Capricor Therapeutics has completed its Biologics License Application (BLA) submission to the FDA for deramiocel to treat Duchenne muscular dystrophy (DMD) cardiomyopathy.
• The BLA is supported by data from Phase 2 HOPE-2 and HOPE-2 Open Label Extension (OLE) trials, showing attenuation of cardiac implications of DMD.
• The FDA has been requested to grant priority review, potentially reducing the review period to six months from the standard ten months.
• The BLA submission triggers a $10 million milestone payment to Capricor from its distribution partner, Nippon Shinyaku.
Elevidys Gene Therapy Shows Sustained Benefits in Duchenne Muscular Dystrophy Patients
• Sarepta Therapeutics' Elevidys demonstrates sustained benefits and disease stabilization in ambulatory Duchenne muscular dystrophy (DMD) patients, according to Phase 3 EMBARK trial results.
• Crossover-treated patients showed a 2.34-point improvement on the North Star Ambulatory Assessment (NSAA) compared to matched external controls after 52 weeks of Elevidys treatment.
• Patients treated with Elevidys in Part 1 of EMBARK maintained clinically meaningful improvements in NSAA, Time to Rise (TTR), and 10-meter walk/run (10MWR) at two years.
• Muscle biopsies showed consistent micro-dystrophin expression, and MRI scans indicated minimal muscle pathology progression, reinforcing Elevidys's long-term efficacy and safety.
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