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 PepGen.com. Follow PepGen on LinkedIn and X.
View source version on businesswire.com: https://www.businesswire.com/news/home/20250310990282/en/
Contacts
Investor Dave Borah, CFASVP, Investor Relations and Corporate Communicationsdborah@pepgen.com
Media Julia DeutschLyra Strategic AdvisoryJdeutsch@lyraadvisory.com
Hashtags

Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Yahoo
14 minutes ago
- Yahoo
Amid cost pressures, US employers are shifting their benefit strategy, WTW survey finds
More companies expanding choice, tackling high cost and reallocating benefit spend NEW YORK, June 10, 2025 (GLOBE NEWSWIRE) -- As U.S. employers grapple with heightened economic uncertainty and greater financial pressures on budgets, steering the right course on benefit strategy is more challenging than ever. This is according to a survey by WTW (NASDAQ: WTW), a leading global advisory, broking and solutions company. As a result, companies are turning to smarter spending, sharper focus and using benefits as a strategic tool to drive engagement, retention and purpose. The 2025 Benefits Trends Survey found rising benefit costs to be the top issue (90%) influencing U.S. employers' benefit strategies in 2025, up from 67% in 2023. Other top concerns include competition for talent (52%), expectations for an enhanced employee experience (43%), cost of living (39%) and rising mental health issues (32%). 'After a long period of high benefits inflation and in the face of a possibly weakening economy, employers are taking a step back and looking to focus on what drives real value for employees and the business. That means targeting support and spending on the benefits that matter most, enabling personalization and helping employees make better decisions,' said Jeff Levin-Scherz, Population Health Leader, North America, Health & Benefits. As the cost of medical care continues to show double digit growth in the U.S., employers face greater challenges in delivering their strategy in key areas such as health benefits (44%), wellbeing programs (44%), and leave benefits (36%). To address these concerns, employers are shifting their strategy. Few are expanding their benefit portfolio, choosing to instead focus on extracting value from their current offerings and improve financing, employee experience, analytics and administration. Compared to just 8% in the past year, 63% of employers plan to reallocate or rebalance spend in the next three years. A majority (73%) plan to tackle high costs by enhancing value or switch to better-value vendors across health, retirement and risk benefits. Just under half (44%) plan to tackle high-cost medical conditions and 37% plan to adopt a network of preferred medical providers. Eager to address employee pressure points, companies are also looking to improve the following priority areas over the next three years: maximizing value, mental health, health benefits, financial wellbeing and family support. Many plan to increase their use of communication and use nudges and navigation solutions to influence behaviors and enhance the employee experience. Regularly reviewing vendor performance, including employee feedback, is also a key action employers are taking. 'Organizations are facing more pressure than ever to deliver the right benefits strategy. Finding innovative solutions for old and new challenges and reallocating spend on benefits that deliver true value is a good start. There is still a long way to go to address these pressure points, but employers are headed in the right direction by focusing on what matters most to their employees,' said Levin-Scherz. About the survey The 2025 Benefit Trends Survey was conducted from early March to mid-April. Respondents include 696 U.S. employers, representing a broad range of industries in both the private and public sector. About WTW At WTW (NASDAQ: WTW), we provide data-driven, insight-led solutions in the areas of people, risk and capital. Leveraging the global view and local expertise of our colleagues serving 140 countries and markets, we help organizations sharpen their strategy, enhance organizational resilience, motivate their workforce and maximize performance. Working shoulder to shoulder with our clients, we uncover opportunities for sustainable success—and provide perspective that moves you. Media contactIleana Feoli: +1 212 309
Yahoo
19 minutes ago
- Yahoo
JetBlue Vacations Expands Insider Experience to San Juan, Strengthening Commitment to Puerto Rico
JetBlue enhances vacation packages to San Juan with local insider support DANIA BEACH, Fla., June 10, 2025--(BUSINESS WIRE)--JetBlue Vacations (Nasdaq: JBLU), powered by Paisly, today announced the expansion of its Insider Experience program to San Juan, Puerto Rico, enhancing the customer experience with personalized service and reinforcing the airline's long-standing commitment to the island as its largest airline. Customers booking a JetBlue Vacations flight + hotel package to San Juan at select hotels will now enjoy the trusted, concierge-style service of local Insiders, who are available to enhance their travel experience from arrival to departure. Launched in 2020 and currently available in Aruba, Cancún, Montego Bay, Punta Cana, Nassau, St. Lucia and Tulum, the Insider Experience puts customers in the hands of knowledgeable, on-the-ground experts who provide airport meet-and-greet services, free airport transfers, and 24/7 support throughout their stay. JetBlue operates more scheduled flights and offers more seats to and from the island than any other carrier—making this latest expansion a natural next step in deepening its presence and support for the destination. Travelers to San Juan will now have access to a dedicated local Insider who can assist with recommendations, booking activities, and addressing any in-destination needs. As part of this expansion, all JetBlue Vacations customers booking a flight + hotel package to San Juan will also receive a free guided walking tour of Old San Juan, in partnership with the Municipality of San Juan. This immersive tour invites travelers to explore the city's 500-year history, walk its iconic blue cobblestone streets, and discover legendary landmarks such as San Juan Bautista Cathedral, San Felipe del Morro Fort, and Casa Blanca Museum, once home to Juan Ponce de León. "Puerto Rico has always been a favorite destination for our JetBlue Vacations customers, and we're excited to bring even more value to their trips with the Insider Experience," said Jamie Perry, president of Paisly, JetBlue's travel subsidiary. "With local Insiders offering personalized support and great perks like a complimentary Old San Juan walking tour, we're making it easier for travelers to explore and enjoy everything the island has to offer," Perry added. The JetBlue Vacations Insider Experience in San Juan will provide: Meet-and-greet service upon arrival at Luis Muñoz Marín International Airport (SJU) Complimentary airport transfers to and from customers' hotels 24/7 access to local Insiders via phone or WhatsApp for assistance throughout their stay Personalized recommendations and booking support for activities, dining, and excursions Expedited issue resolution, with Insiders empowered to handle on-the-ground requests quickly "The expansion of JetBlue Vacations' Insider Experience program to San Juan reaffirms the Island's continued development as a world-class travel destination. We are proud to collaborate on this initiative, which offers visitors authentic and meaningful experiences while supporting local businesses and promoting the rich culture and history of our capital city," said Willianette Robles, Executive Director of the Puerto Rico Tourism Company. Through this initiative, JetBlue continues to support Puerto Rico's tourism industry, promote cultural experiences, and enhance the overall travel journey for its customers. With a growing network of flights and the recent launch of a new crew base in San Juan, JetBlue remains deeply committed to strengthening its ties with the local community and creating unforgettable travel experiences. To book a JetBlue Vacations package to San Juan and take advantage of the Insider Experience and free Old San Juan walking tour, visit About JetBlue VacationsJetBlue Vacations offers flexible travel packages including JetBlue flight + hotel, JetBlue flight + cruise, hotel + points, and standalone cruise bookings. Customers get access to the lowest available JetBlue airfare when they book as part of a package, delivering exceptional value for money. Each trip can be customized with add-ons like cars, transfers, and activities. Vacation packages also earn TrueBlue points and tiles, helping customers get closer to Perks You Pick® & Mosaic status. Select JetBlue Vacations packages include exclusive benefits through the Very Important Perks (VIP) and Insider Experience programs (available in select destinations), bringing JetBlue's signature customer service into every step of the travel journey. About PaislyPaisly, LLC is a tech-enabled managed travel services company that is committed to delivering personalized, human-first experiences at scale. Paisly, a wholly-owned subsidiary of JetBlue Airways Corporation, leverages a proprietary technology platform that is purpose-built for the airline industry, and delivers personalized travel offers based on customer behavior and real-time context. Unlike traditional platforms, Paisly manages all services in-house, from hotel partnerships and marketing to customer care, ensuring a seamless experience. Its model is built on three core pillars: great products powered by innovative tech, exceptional customer service delivered by its 24/7 Helpful Humans team and Insider Experience program, and unbeatable value through personalized recommendations. With over 300 dedicated crewmembers, Paisly is redefining how travelers research, plan, book, and enjoy their trips - making every journey better. About JetBlueJetBlue is New York's Hometown Airline®, and a leading carrier in Boston, Fort Lauderdale-Hollywood, Los Angeles, Orlando, and San Juan. JetBlue carries customers to more than 100 cities throughout the United States, Latin America, Caribbean, Canada, and Europe. For more information and the best fares, visit View source version on Contacts JetBlue Corporate Communications Tel: +1.718.709.3089corpcomm@ Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data


Business Wire
24 minutes ago
- Business Wire
JetBlue Vacations Expands Insider Experience to San Juan, Strengthening Commitment to Puerto Rico
BUSINESS WIRE)--JetBlue Vacations (Nasdaq: JBLU), powered by Paisly, today announced the expansion of its Insider Experience program to San Juan, Puerto Rico, enhancing the customer experience with personalized service and reinforcing the airline's long-standing commitment to the island as its largest airline. Customers booking a JetBlue Vacations flight + hotel package to San Juan at select hotels will now enjoy the trusted, concierge-style service of local Insiders, who are available to enhance their travel experience from arrival to departure. Launched in 2020 and currently available in Aruba, Cancún, Montego Bay, Punta Cana, Nassau, St. Lucia and Tulum, the Insider Experience puts customers in the hands of knowledgeable, on-the-ground experts who provide airport meet-and-greet services, free airport transfers, and 24/7 support throughout their stay. JetBlue operates more scheduled flights and offers more seats to and from the island than any other carrier—making this latest expansion a natural next step in deepening its presence and support for the destination. Travelers to San Juan will now have access to a dedicated local Insider who can assist with recommendations, booking activities, and addressing any in-destination needs. As part of this expansion, all JetBlue Vacations customers booking a flight + hotel package to San Juan will also receive a free guided walking tour of Old San Juan, in partnership with the Municipality of San Juan. This immersive tour invites travelers to explore the city's 500-year history, walk its iconic blue cobblestone streets, and discover legendary landmarks such as San Juan Bautista Cathedral, San Felipe del Morro Fort, and Casa Blanca Museum, once home to Juan Ponce de León. 'Puerto Rico has always been a favorite destination for our JetBlue Vacations customers, and we're excited to bring even more value to their trips with the Insider Experience,' said Jamie Perry, president of Paisly, JetBlue's travel subsidiary. 'With local Insiders offering personalized support and great perks like a complimentary Old San Juan walking tour, we're making it easier for travelers to explore and enjoy everything the island has to offer,' Perry added. The JetBlue Vacations Insider Experience in San Juan will provide: Meet-and-greet service upon arrival at Luis Muñoz Marín International Airport (SJU) Complimentary airport transfers to and from customers' hotels 24/7 access to local Insiders via phone or WhatsApp for assistance throughout their stay Personalized recommendations and booking support for activities, dining, and excursions Expedited issue resolution, with Insiders empowered to handle on-the-ground requests quickly 'The expansion of JetBlue Vacations' Insider Experience program to San Juan reaffirms the Island's continued development as a world-class travel destination. We are proud to collaborate on this initiative, which offers visitors authentic and meaningful experiences while supporting local businesses and promoting the rich culture and history of our capital city,' said Willianette Robles, Executive Director of the Puerto Rico Tourism Company. Through this initiative, JetBlue continues to support Puerto Rico's tourism industry, promote cultural experiences, and enhance the overall travel journey for its customers. With a growing network of flights and the recent launch of a new crew base in San Juan, JetBlue remains deeply committed to strengthening its ties with the local community and creating unforgettable travel experiences. To book a JetBlue Vacations package to San Juan and take advantage of the Insider Experience and free Old San Juan walking tour, visit About JetBlue Vacations JetBlue Vacations offers flexible travel packages including JetBlue flight + hotel, JetBlue flight + cruise, hotel + points, and standalone cruise bookings. Customers get access to the lowest available JetBlue airfare when they book as part of a package, delivering exceptional value for money. Each trip can be customized with add-ons like cars, transfers, and activities. Vacation packages also earn TrueBlue points and tiles, helping customers get closer to Perks You Pick® & Mosaic status. Select JetBlue Vacations packages include exclusive benefits through the Very Important Perks (VIP) and Insider Experience programs (available in select destinations), bringing JetBlue's signature customer service into every step of the travel journey. About Paisly Paisly, LLC is a tech-enabled managed travel services company that is committed to delivering personalized, human-first experiences at scale. Paisly, a wholly-owned subsidiary of JetBlue Airways Corporation, leverages a proprietary technology platform that is purpose-built for the airline industry, and delivers personalized travel offers based on customer behavior and real-time context. Unlike traditional platforms, Paisly manages all services in-house, from hotel partnerships and marketing to customer care, ensuring a seamless experience. Its model is built on three core pillars: great products powered by innovative tech, exceptional customer service delivered by its 24/7 Helpful Humans team and Insider Experience program, and unbeatable value through personalized recommendations. With over 300 dedicated crewmembers, Paisly is redefining how travelers research, plan, book, and enjoy their trips - making every journey better. About JetBlue JetBlue is New York's Hometown Airline®, and a leading carrier in Boston, Fort Lauderdale-Hollywood, Los Angeles, Orlando, and San Juan. JetBlue carries customers to more than 100 cities throughout the United States, Latin America, Caribbean, Canada, and Europe. For more information and the best fares, visit