Rewriting the Script for Rare Diseases
Sarepta Therapeutics, headquartered in Cambridge, Massachusetts, with its Genetic Therapies Center of Excellence (GTCOE) located in Columbus, Ohio, is a global leader in precision genetic medicine. The company is driven by its mission to transform the lives of patients with rare diseases, including those with life-limiting genetic disorders such as Duchenne muscular dystrophy (Duchenne) and limb-girdle muscular dystrophies (LGMDs).
Under the leadership of Louise Rodino-Klapac, Ph.D., Executive Vice President, Head of R&D and Chief Scientific Officer, Sarepta has established a legacy of groundbreaking science and relentless commitment to advancing genetic therapies that hold the promise of having a transformative impact. As head of Sarepta’s vast multidisciplinary team in research and clinical development, Dr. Rodino-Klapac is forging new and innovative pathways for the next generation of medicines to treat complex genetic diseases, as she plays a leading role in establishing Columbus as a thriving innovation hub. Today, Sarepta’s work in gene therapy is among the most promising and clinically advanced in the world and is rooted in Columbus.
At Sarepta, we believe in challenging the impossible. Every breakthrough is a testament to the courage of the patients we serve and the dedication of our team to improving their lives.
Louise Rodino-Klapac, Ph.D.Executive Vice President, Head of R&D & Chief Scientific Officer, Sarepta Therapeutics
Through its innovative RNA-targeted therapies, gene therapies, and gene editing programs, Sarepta has developed a robust pipeline of treatments addressing the underlying causes of genetic diseases. The company’s exon-skipping treatments for Duchenne, the first such therapies approved by the U.S. FDA, are designed to restore the production of dystrophin, a protein essential for muscle health, giving patients and their families renewed hope for the future. Sarepta also developed the first gene therapy to treat Duchenne, a groundbreaking treatment that was co-invented by Dr. Rodino-Klapac and advanced from a hope to reality thanks to her dedication and tenacity and that of her team. It was because of her scientific ingenuity and decades of commitment that individuals with Duchenne now have a chance to rewrite their futures. For Sarepta, it paved the way for other gene therapies in its pipeline, securing the company’s position as a leader not only in Duchenne but in the field of gene therapy.
Beyond Duchenne, Dr. Rodino-Klapac co-founded and served as Chief Scientific Officer of Myonexus Therapeutics, a Columbus, Ohio-based gene therapy company focused on LGMDs acquired by Sarepta in 2019. The company’s LGMD programs from that acquisition are now advancing steadily through development.
Partnerships Powering Progress
Sarepta’s work extends far beyond the lab. The company has cultivated strategic global partnerships to accelerate the development and accessibility of promising therapies. A recent collaboration with Arrowhead Pharmaceuticals* exemplifies Sarepta’s commitment to leveraging complementary expertise to expand its therapeutic pipeline. Through its collaboration with Arrowhead, Sarepta is advancing numerous therapies, including four clinical-stage programs, three pre-clinical programs, and six research targets across neuromuscular, CNS, cardiomyopathy, and pulmonary diseases.
*Subject to Federal Trade Commission clearance
Innovation thrives in collaboration. By partnering with visionary and innovative organizations, we can amplify our impact and move closer to achieving our ultimate goal: a world where no patient is left behind.
Louise Rodino-Klapac, Ph.D.Executive Vice President, Head of R&D & Chief Scientific Officer, Sarepta Therapeutics
Ohio, particularly Columbus, is vital to Sarepta for numerous strategic and operational reasons:
- The biotechnology ecosystem, which is alive and thriving in Ohio, plays a central and strategic role in supporting Sarepta’s mission to patients and its position as the leader in precision genetic medicine
- The intersection of academic, leading treatment practitioners, and industry make Columbus the epicenter of biomedical innovation, particularly in gene therapy
- The people advancing Sarepta’s work are pleased to be a part of the Columbus community, and Sarepta is dedicated to growing its presence in Columbus
- In October 2021, Sarepta announced the official grand opening of its GTCOE in Columbus and its commitment to the region is stronger than ever
- Sarepta is building the infrastructure and providing the resources necessary to support Ohio’s prominence in the life sciences, including expanding the GTCOE’s footprint and attracting top talent from across the country
Sarepta’s connections to Ohio have bolstered its mission by fostering research, innovation, and talent development in the field of genetic medicine.
Charting New Frontiers in Genetic Medicine
Sarepta’s vision for the future is one of bold ambition. The company is actively exploring advancements in genetic medicine and continues to push the boundaries of what’s possible.
Our vision is clear. We aim to rewrite the future for patients with genetic diseases. By harnessing the power of genetic medicine, we’re not just treating symptoms—we’re targeting the root causes and providing real hope for lasting change.
Louise Rodino-Klapac, Ph.D.Executive Vice President, Head of R&D & Chief Scientific Officer, Sarepta Therapeutics
The company’s dedication to patients and innovation ensures that Sarepta remains a leader in the life sciences. With a relentless drive to improve lives, Sarepta is redefining the possibilities of modern medicine.
Sarepta is more than a biotechnology company; it’s a lifeline for patients with rare genetic disorders. Guided by a profound commitment to science and humanity, Sarepta continues to make strides in genetic medicine, offering hope to patients worldwide. As the company advances its groundbreaking work, it strengthens its position as a beacon of innovation and compassion in the life sciences.
