IFLScience on MSN6h
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 towards seeing this treatment in the clinic. For this first phase, the aim ...
pharmaphorum10h
Trial of Boehringer's cystic fibrosis gene therapy imminent
An inhaled gene therapy for cystic fibrosis developed by Boehringer Ingelheim is due to start clinical testing shortly, hoping to become a first-in-class treatment for the genetic disorder.
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, ...
Hosted on MSN2mon
Next-in-Class Once-Daily Pill Approved for Cystic Fibrosis
According to Vertex, the next-in-class drug is active against an additional 31 mutations in the CFTR gene ... predicted forced expiratory volume in 1 second (ppFEV1), the primary endpoint.
EurekAlert!1d
Novel inhalable gene therapy trialled for people with cystic fibrosis
An inhalable medicine with the potential to improve lung disease in people with cystic fibrosis, irrespective of their mutation type, is being tested in human trials in the UK and Europe.
Kaleido Scope5y
CFTR Protein Production Core
1. “The allosteric effects of the F508del mutation in human CFTR elucidated by cryo-EM enabled by protein engineering,” presented by John F. Hunt, Columbia University, at the 16th ECFS Basic Science ...
The Scientist5mon
A Gene Editing System Corrects Cystic Fibrosis Mutation
In their 2019 paper, Liu’s team used prime editing to alter the gene mutations causing sickle cell disease and Tay-Sachs disease. Liu explained that they attempted to edit mutated CFTR as well but ...
precisionmedicineonline1d
Boehringer Ingelheim Kicks Off Inhaled Cystic Fibrosis Gene Therapy Phase I/II Trial With Partners
Boehringer Ingelheim, IP Group, UK Respiratory Gene Therapy Consortium, and OXB launched the first-in-human clinical trial, known as LENTICLAIR 1.
The Scientist9mon
A New Delivery System Offers Hope for Cystic Fibrosis
The patient’s cells must produce the CFTR protein for the existing therapies to work, which leaves patients with nonsense mutations without options. Since its initial discovery, researchers believed ...
Kaleido Scope6y
Gene Expression Core
These activities are organized through three Core functions: Core Function #1: Clone new CFTR constructs and other CFTR regulatory proteins The Core has the capabilities and skill to clone new CFTR ...
ADVFN1mon
Vertex Announces US FDA Approval of ALYFTREK™, a Once-Daily Next-in-Class CFTR Modulator for the Treatment of Cystic Fibrosis
ALYFTREK™ is approved for patients 6 years and older with at least one responsive mutation ... next-in-class triple combination cystic fibrosis transmembrane conductance regulator (CFTR ...