What a $2 million per dose gene therapy reveals about drug pricing
Vincent Gaynor remembers, almost to the minute, when he realized his part in birthing the breakthrough gene therapy Zolgensma ...
Boehringer Ingelheim and partners start clinical development of a first-in-class, inhaled gene therapy for people with cystic fibrosis
Boehringer Ingelheim, IP Group, the UK Respiratory Gene Therapy Consortium (GTC) 1 and OXB, 1 today announce the start of LENTICLAIR TM 1, a Phase I/II trial of BI 3720931, a novel, first-in-class, ...
IFLScience on MSN1d
Inhalable Cystic Fibrosis Gene Therapy 24 Years In The Making Enters Clinical Trials
A new inhalable gene therapy for cystic fibrosis (CF) has entered Phase I clinical trials, a major milestone on the journey ...
AskBio Receives FDA Regenerative Medicine Advanced Therapy designation for Parkinson’s disease investigational gene therapy
Berlin, Germany, and Research Triangle Park, N.C., USA, Feb. 19, 2025 (GLOBE NEWSWIRE) -- Not intended for UK MediaRegenerative Medicine ...
uniQure: Gene Therapy Specialist Looks Set For Upside As Huntington's Approval Beckons
Qure's AMT-130 shows 80% Huntington's progress reduction, eyeing FDA approval. Click here to read why I rate QURE stock a Buy ...
Parents of boy with rare eye condition hail ‘amazing’ results of gene therapy
Jace’s mum DJ, 35, told the PA news agency: “Pre-surgery, at around two years old, you could have held up any object, even a ...
News Medical2d
The Latest Advances in Cell and Gene Therapy
The evolution of cell and gene therapy is reshaping drug discovery, providing new hope for rare diseases and cancer through innovative gene editing approaches.
GlobalData on MSN2d
Boehringer and partners commence gene therapy trial for cystic fibrosis
Boehringer Ingelheim, the UK Respiratory Gene Therapy Consortium (GTC), IP Group and Oxford BioMedica (OXB) have commenced ...
Scientific Research Publishing2d
BDNF Gene Therapy: A Potential Solution for Neurodegenerative Diseases ()
A Potential Solution for Neurodegenerative Diseases. Open Access Library Journal, 12, 1-16. doi: 10.4236/oalib.1112714 . Alzheimer’s Disease (AD), a complex neurodegenerative condition, significantly ...