Papillon Therapeutics

PPL-002, our experimental treatment for Danon disease, has been granted #RarePediatricDisease designation from the FDA! Danon disease is a rare, X-linked genetic disorder that causes a range of life-threatening symptoms, including severe cardiomyopathy, physical impairment and retinal abnormalities. Therapeutics intended to treat serious or life-threatening rare diseases that affect individuals under the age of 18 are eligible for Rare Pediatric Disease designation. Read more: https://bwnews.pr/3YxqptI

We are thrilled to announce that the FDA has granted Rare Pediatric Disease designation to PPL-001! PPL-001 is an experimental treatment for Friedreich's ataxia, a rare, inherited neurodegenerative disorder. The FDA grants Rare Pediatric Disease designation to therapeutics intended to treat serious or life-threatening rare diseases that affect individuals under the age of 18. Read the full press release here: https://bit.ly/4eZ3Kwd

We're in Phoenix, Arizona, for the Alliance for Regenerative Medicine's Cell & Gene Meeting on the Mesa! Join us at 9:30 am on Monday, October 7 for our company presentation with CEO Carter Cliff. Find us on the agenda here: www.meetingonthemesa.com #CGMesa24 #cellandgenetherapy

Our pipeline has received another #OrphanDrugDesignation! PPL-002 is a gene therapy in development to treat Danon disease, a rare, X-linked genetic disorder. People living with Danon disease experience a range of life-threatening symptoms, including severe cardiomyopathy, physical impairment and retinal abnormalities. PPL-002's treatment approach involves targeting multiple organ systems simultaneously in patients, offering the potential to modify and reverse disease progression. Learn more: https://bwnews.pr/3zEaz6S

We're excited to be one of the 2024 Connect Cool Companies participating in San Diego Innovation Day tomorrow! Hear from our CEO Carter Cliff, who will be sharing the story of Papillon at several events throughout the day, including the Venture Summit, JLABS Fireside Chat, and the Connect and TL Foundation Pitches. Visit sandiegoisbetter.com for more info! #IDSD24 

Here is an Advanced Regenerative Manufacturing Institute (ARMI) | BioFabUSA Member success story hot off the press: Papillon Therapeutics Receives Orphan Drug Designation from the U.S. Food and Drug Administration for PPL-001 for the Treatment of Friedreich's Ataxia. ARMI’s BioFabConsulting group drafted and submitted Papillon Therapeutics’ Orphan Drug Designation application, and we are pleased to announce that the FDA has granted an Orphan Drug Designation to gene therapy in development for Friedreich’s ataxia, a rare inherited neurodegenerative disorder. Friedreich’s ataxia often begins in childhood or adolescence, causing progressive issues with walking, speech, and motor functions. Our therapy takes a multi-systemic approach, targeting multiple organ systems simultaneously and offering the potential to modify and reverse disease progression. Join us in Congratulating Papillon Therapeutics! Read the full Business Wire Article Here: https://bwnews.pr/4gGUKx1 #Friedreichsataxia #medicalInnovation #BioFabConsulting

We are pleased to announce that the FDA has granted Orphan Drug Designation to our gene therapy in development for Friedreich’s ataxia, a rare inherited neurodegenerative disorder. Friedreich’s ataxia often begins for patients in childhood or adolescence, causing progressive issues with walking, speech, and motor functions. Our therapy takes a multi-systemic approach, targeting multiple organ systems simultaneously and offering the potential to modify and reverse disease progression. Learn more: https://bwnews.pr/4gGUKx1

Exciting news! The TL Fund announces four new investments of $100,000 each into four local startups! Congratulations to Condor, Looq AI, Papillon Therapeutics, and Resolute Science! Want to see these companies pitch on the big stage, and experience the ultimate convergence of life science and tech in San Diego? Join us for the Petco Park takeover on September 24th! https://bit.ly/3QWaQaJ

Exciting news! Our scientific co-founder Dr. Stephanie Cherqui is presenting tomorrow at #DIA2024. Join Dr. Cherqui and other leading experts in the field to gain valuable insights into regulatory considerations in trial design and the path from bench-to-bedside for a cell and gene therapy product. https://bit.ly/3Xowpon

Our CEO Carter Cliff was recently featured on the Life Science & Biotech CEO Stories podcast! In this episode, Carter shares his journey building #biotech companies like Papillon Therapeutics, and offers valuable insights for aspiring company founders. Thanks to Timothy Dougherty for hosting such an engaging conversation. Listen to the full episode here: https://bit.ly/3Xi5B9h