PTC Therapeutics

Rylae-Ann, diagnosed with AADC deficiency, received gene therapy in a clinical trial, transforming her life. Her parents founded Teach RARE to support other families. The FDA's recent approval of the therapy marks a significant milestone, offering hope to many.

BioTech Breakthrough announces 4th annual awards recognizing top life sciences and biotechnology innovations, with winners including Leyden Labs, Rocket Pharmaceuticals, Phathom Pharmaceuticals, and more.

BioTech Breakthrough announced winners of its 4th annual awards, recognizing top biotech companies and innovations in areas like BioPharma, Genomics, Therapeutics, and AI. The program attracted thousands of nominations from 14 countries, highlighting advancements in biological sciences and AI, such as CRISPR and precision medicine.

The FDA approved Kebilidi (eladocagene exuparvovec-tneq) for AADC deficiency, making it the first brain-administered therapy in the US. Developed by PTC Therapeutics, Kebilidi is a gene replacement therapy targeting the putamen, restoring dopamine synthesis and improving motor development. The approval is based on ongoing clinical trial data, with EU and UK approvals as Upstaza in 2022 and 2023, respectively.

FDA approved eladocagene exuparvovec-tneq (Kebilidi), the first gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency, administered via four infusions into the brain. Clinical study showed improved gross motor function in 8 out of 12 treated pediatric patients. FDA also authorized the SmartFlow Neuro Cannula for delivering the therapy.

FDA's increased flexibility in drug approvals, particularly for rare diseases, could boost biotech stocks. However, this trend raises concerns about taxpayer funding for potentially ineffective drugs and patient safety. Four biotech firms—Applied Therapeutics, Biohaven, PTC Therapeutics, and Soleno Therapeutics—may benefit from this flexibility, potentially driving stock gains.

FDA's increased flexibility in drug approvals benefits biotech stocks, with 4 companies—Applied Therapeutics, Biohaven, PTC Therapeutics, and Soleno Therapeutics—poised for gains if their drugs for rare diseases are approved.

FDA fast-tracks approval for Kebilidi, a gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency, the first U.S. gene therapy injected directly into the brain. Indicated for children and adults, it boosts AADC to restore dopamine production, improving movement-related symptoms. Long-term studies and additional benefits proof needed for full FDA approval. Side effects include dyskinesia, anemia, fever, and surgical risks like cerebrospinal fluid leaks and infections.

FDA clears icobrain aria, the first AI tool for detecting ARIA in Alzheimer treatment; approves expanded age indication for Theranica's Nerivio for acute migraine in pediatric patients; grants accelerated approval to PTC Therapeutics' gene therapy eladocagene exuparvovec-tneq for AADC deficiency.

The FDA approved Kebilidi, the first gene therapy for AADC deficiency, showing motor function improvement in 8 of 12 treated pediatric patients. Common adverse reactions include dyskinesia and procedural complications. Approved via Accelerated Approval, it uses the SmartFlow Neuro Cannula for administration.