PTC Therapeutics, Inc.

Today, we announced our submission of the vatiquinone New Drug Application (NDA) for the treatment of children and adults living with Friedreich ataxia (FA) to the U.S. Food and Drug Administration (FDA). Learn more: https://bit.ly/4gzoGdC

Supporting the next generation of talent and leaders in the life sciences industry is important to us at PTC. Since 2020, our Talent & Pipeline Program (TPP) has given recent grads the opportunity to participate in a year-long fellowship program that provides comprehensive training and real-world work experience across a range of functions. We're thrilled to launch a new cohort for 2025 and look forward to helping individuals make the next meaningful step in their careers. 👉 Click the link to learn more and apply: https://bit.ly/4gHhHiI

PTC was excited to learn, share and collaborate with other leading RNA experts at the recent 7th Annual RNA-Targeted Drug Discovery & Development Summit in Boston, MA, and the RNA at the Bench and Bedside IV conference in La Jolla, CA. Anu Bhattacharyya, Kerstin Effenberger, and Marla Weetall from our research team presented on ways PTC is leveraging its leading splicing platform to identify promising therapies for rare diseases with high unmet need. Learn more about our splicing platform here: https://bit.ly/4fi4w6L

Our team was excited to celebrate with the #FriedreichAtaxia (FA) community at the annual FARA Energy Ball, a fundraiser for important research on FA. Our CEO, Matthew Klein, MD, MS, FACS is pictured here with Friedreich's Ataxia Research Alliance (FARA) Founder and President Ron Bartek and CEO Jen Farmer. In addition, at the FA Research Reception we had the opportunity to provide an update on our vatiquinone New Drug Application which is scheduled to be submitted before the end of the month.

Pam and her daughter, Kelsey, live with phenylketonuria (#PKU), a rare, inherited, metabolic disease that affects the brain. Despite the challenges of managing this condition every day, they inspire one another. In honor of #PKUAwarenessDay, we’re sharing a glimpse into how their PKU journey has formed an incredible bond between a mother and her daughter both living with a #raredisease. Head to our website to watch and learn more: https://lnkd.in/d-SyjiaY

Today we are pleased to announce that PTC has entered into a Global License and Collaboration Agreement with Novartis for the PTC518 Huntington’s disease program. “PTC518 is the leading oral disease-modifying therapy in development for Huntington's disease and the economics of this agreement are consistent with the promise of this treatment," said Matthew Klein, MD, MS, FACS, Chief Executive Officer. "This collaboration combines PTC’s expertise in developing small molecule splicing therapies with Novartis’s expertise in global development and commercialization of neuroscience therapies. We are excited to collaborate with Novartis to accelerate the potential of PTC518 for the hundreds of thousands of HD patients worldwide in need of a therapy designed to be well-tolerated and an effective disease-modifying therapy. PTC will use the proceeds of this transaction to expand our splicing platform as well as to support commercial and development portfolio activities.” Learn more: https://bit.ly/3ViarS2

Today we announced that PTC has entered into an agreement to sell its Rare Pediatric Disease Priority Review Voucher for $150 million upon the closing of the transaction. Learn more: https://bit.ly/4g99ZOa

Caregiving is a profound act of love and commitment, but it can also be an incredibly challenging journey when you're caring for someone with a #raredisease. From navigating complex medical systems to providing round-the-clock care, #caregivers shoulder immense responsibilities that often go unnoticed by the broader public. In honor of National Family Caregivers Month, we're sharing just a few of their personal stories as a reminder of the critical role they serve in managing and caring for those with a rare condition. 📖 Read more: https://lnkd.in/erDh2rT9 #NFCM

PTC set forth a range of ambitious goals at the start of 2024, and as Chief Executive Officer Matthew Klein, MD, MS, FACS, recently shared during the Jefferies Global Healthcare Conference in London, we’re proud to be on track to executing all of our plans and upcoming regulatory milestones. Learn more about our latest business achievements and plans for the year ahead: https://bit.ly/3AXW5iD

Congratulations team great recognition!