Latest news with #ClinicalDevelopment
Yahoo
10-03-2025
- Business
- Yahoo
PepGen Announces Presentations at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference
BOSTON, March 10, 2025--(BUSINESS WIRE)--PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases, today announced that the Company will be presenting two oral presentations as well as five posters at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, being held March 16-19, 2025 in Dallas, TX. The oral presentations feature data from the ongoing CONNECT1-EDO51 clinical trial in Duchenne muscular dystrophy (DMD) and from the ongoing FREEDOM-DM1 clinical trial in myotonic dystrophy type 1 (DM1), which the Company recently announced. Oral Presentations Title: CONNECT1-EDO51: A 12-Week Open-Label Phase 2 Study to Evaluate PGN-EDO51 Safety and Efficacy in People with Duchenne Amenable to Exon 51 SkippingSession: Clinical Trial UpdatesDate & Time: Wednesday, March 19th at 8:45-9:00 a.m. CTPresenter: Paul Streck, MD, MBA, Executive Vice President, R&D Title: FREEDOM-DM1: A Phase 1, Placebo-Controlled Single Ascending Dose Study to Evaluate PGN-EDODM1 in People with Myotonic Dystrophy Type 1 (DM1)Session: Clinical Trial UpdatesDate & Time: Wednesday, March 19th at 12:45-1:00 p.m. CTPresenter: Jane Larkindale, DPhil, Vice President, Clinical Science Poster Presentations Posters will be displayed beginning at 6:00-8:00 p.m. CT on Sunday, March 16th through Tuesday, March 18th in the conference exhibit hall. Title: Mechanistic Characterization of Enhanced Delivery Oligonucleotide (EDO) PlatformPoster Number: #P21Presenter: Jane Larkindale, DPhil, Vice President, Clinical Science Title: Nonclinical Data for PGN-EDODM1 Demonstrated Mechanistic and Meaningful Activity for the Potential Treatment of DM1Poster Number: #P48Presenter: Ashling Holland, PhD, Director, Research & Preclinical Development Title: Single- and Repeat-Dose Nonclinical Data for PGN-EDO51 Demonstrated Favorable Pharmacology and Safety Profiles for the Treatment of DMDPoster Number: #P49Presenter: Ashling Holland, PhD, Director, Research & Preclinical Development Title: FREEDOM-DM1: A Phase 1, Placebo-Controlled Single Ascending Dose Study to Evaluate PGN-EDODM1 in People with Myotonic Dystrophy Type 1 (DM1)Poster Number: #O45Presenter: Jennifer Shoskes, PharmD, Associate Director, Clinical Development Title: CONNECT1-EDO51: A 12-Week Open-Label Phase 2 Study to Evaluate PGN-EDO51 Safety and Efficacy in People with Duchenne Amenable to Exon 51 SkippingPoster Number: #O74Presenter: Bassem Morcos, MD, Medical Director, Clinical Development Following the conference, the presentations presented at the MDA Clinical & Scientific Conference will be available on the Investors page of PepGen's website under Scientific Publications. About PGN-EDODM1 PGN-EDODM1, PepGen's investigational candidate in development for the treatment of DM1, utilizes the Company's proprietary EDO technology to deliver a therapeutic oligonucleotide that is designed to restore the normal splicing function of MBNL1, a key RNA splicing protein. PGN-EDODM1 is designed to directly address the deleterious effects of cytosine-uracil-guanine (CUG) repeat expansion in the DMPK transcripts which sequester MBNL1, by binding to the pathogenic CUG trinucleotide repeat expansion present in the DMPK transcripts, disrupting the binding between the CUG repeat expansion and MBNL1. We believe this mechanism will allow the DMPK transcripts to continue performing its normal function within the cell, while also liberating MBNL1 to correct downstream mis-splicing events. We believe that this innovative therapeutic approach has considerable advantages over oligonucleotide modalities that rely on knockdown or degradation of the DMPK transcripts. The FDA has granted PGN-EDODM1 both Orphan Drug and Fast Track Designations for the treatment of patients with DM1. About PGN-EDO51 PGN-EDO51, PepGen's investigational candidate in development for the treatment of DMD, utilizes the Company's proprietary EDO technology to deliver a therapeutic oligonucleotide that is designed to target the root cause of this devastating disease. PGN-EDO51 is designed to skip exon 51 of the dystrophin transcript, an established therapeutic target for approximately 13% of DMD patients, thereby aiming to restore the open reading frame and enabling the production of a truncated, yet functional dystrophin protein. The FDA has granted PGN-EDO51 both Orphan Drug and Rare Pediatric Disease Designations for the treatment of patients with DMD amenable to an exon-51 skipping approach. About PepGen PepGen is a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases. PepGen's EDO platform is founded on over a decade of research and development and leverages cell-penetrating peptides to improve the uptake and activity of conjugated oligonucleotide therapeutics. Using these EDO peptides, we are generating a pipeline of oligonucleotide therapeutic candidates designed to target the root cause of serious diseases. For more information, please visit Follow PepGen on LinkedIn and X. View source version on Contacts Investor Dave Borah, CFASVP, Investor Relations and Corporate Communicationsdborah@ Media Julia DeutschLyra Strategic AdvisoryJdeutsch@
Yahoo
30-01-2025
- Business
- Yahoo
Science Exchange Announces New Clinical Development Solution
Addressing the complexity of FSP and FSO outsourcing models, this new solution, powered by Science Exchange's supplier orchestration platform, modernizes clinical supplier collaboration and budget management. PALO ALTO, Calif., January 30, 2025--(BUSINESS WIRE)--Science Exchange, the leading SaaS provider of supplier orchestration solutions for the life sciences industry, today announced the launch of its Clinical Development Solution. Designed to address the unique challenges faced by clinical operations and procurement teams managing outsourced clinical studies, the platform provides pharmaceutical sponsors with the tools to efficiently orchestrate the entire contracting and collaboration process across suppliers—whether in functional service provider (FSP) or full-service outsourcing (FSO) models. The Business Process Challenge in Clinical Development Clinical trials, especially large-scale studies such as oncology trials, involve increasingly complex outsourcing arrangements. The shift from full-service outsourcing (FSO) to functional service provider (FSP) models has created new operational burdens for clinical operations teams. In an FSP model, sponsors directly manage a diverse set of specialized suppliers responsible for various trial components, requiring greater coordination and oversight. Key business process challenges include: Study Intake and RFI/RFP Management: Coordinating RFIs and RFPs for specialized services becomes time-intensive, often managed via email and spreadsheets. Budget Lifecycle and Rate Card Validation: Ensuring supplier budgets align with pre-negotiated rate cards in MSAs requires meticulous review and introduces risks of errors. Collaboration Between Stakeholders: Communication across sponsors and multiple vendors often lacks a central hub and is done via email, leading to potential communication silos and downstream budget errors. Tracking Change Orders: Managing budget amendments and ensuring proper documentation is a manual, fragmented process that often relies on complex Excel models and PDFs. Spend and Payment Processing Transparency: Consolidating data from diverse suppliers for reporting and analysis is a significant challenge, particularly around invoices and attribution of invoices to specific budget lines. These challenges are common across the full lifecycle of clinical trials and highlight the critical need for a digital supplier orchestration platform purpose-built for clinical development operations. Modernizing Clinical Operations with Supplier Orchestration Software The Science Exchange Clinical Development Solution addresses these pain points head-on, empowering sponsors with a centralized platform to manage the complexities of FSP and FSO models. Key features include: Digital Workspaces: Streamline all supplier engagements, budgets, and contracts on a single platform, providing visibility and control across the trial lifecycle. On-Cloud Budget Grids: Digital budget grids enable strong budget compliance and reduce version control errors. Rate Card Compliance Automation: Automatically validate budget lines against supplier-specific rate cards in MSAs, flagging discrepancies to ensure compliance at the line level. Real-Time Collaboration: Foster seamless communication between sponsors and vendors, eliminating the silos that often delay trials. Change Order Log Management: Digitize the process of tracking and approving budget modifications, ensuring an audit trail for compliance. Spend Data Mining: Centralize and validate supplier spend data at the budget line level to generate actionable insights and ensure alignment with trial budgets, whether at the study or program levels. "In an era where R&D budget complexity threatens strategic decision-making, Science Exchange's clinical development solution acts like a digital Rosetta Stone. By converting fragmented PDF and Excel data into structured, actionable intelligence, they're not just digitizing budgets—they're transforming how life sciences organizations unlock hidden financial insights. Imagine having instant, granular visibility into every budget line item, enabling precision cost optimization that was previously impossible," said Wynn Bailey, a former partner at PwC, Life Sciences Operations practice. The Science Exchange Clinical Development Solution is a transformative step forward for the industry, addressing the operational burdens of increasingly complex outsourcing models. By enabling sponsors to bring their supplier engagements, budgets, and collaboration processes onto a single technology platform, Science Exchange is paving the way for faster, more efficient clinical trials. "The shift to FSP outsourcing models has introduced unprecedented challenges for clinical operations teams, particularly in managing a growing network of specialized suppliers," said Elizabeth Iorns, CEO and Co-Founder at Science Exchange. "Our orchestration platform is purpose-built to address these challenges, providing sponsors with the technology to digitize, automate, and streamline their workflows across all components of clinical development. Whether it's a central labs study or a complex Phase 3 oncology trial, the solution is designed to deliver efficiency, transparency, and process compliance." Explore our new solution Join our webinar: Supplier Orchestration in Action: Modernizing Clinical Operations through Technology | Wednesday, February 26 | 10:00 AM PT / 1:00 PM ET Read our blog post: Navigating the Shift to FSP Models: Addressing the Complexity of Clinical Development with Supplier Orchestration. Visit our website: Clinical Development Solution page. About Science Exchange Science Exchange is the first procurement and supplier orchestration cloud software platform for life sciences companies. The platform digitizes and streamlines complex procurement workflows spanning the entire purchasing lifecycle, from vendor and product discovery through purchasing and payment. Many of the world's top pharma and biotech organizations rely on Science Exchange to help accelerate their research, automate manual processes, and advance their programs faster. For additional information on Science Exchange, please visit View source version on Contacts Media Contact: Kelly Science Exchange Sign in to access your portfolio
