Krystal Biotech Touts Positive KB407 CF Gene Therapy Data, Eyes Repeat Dosing and Registrational Path

Krystal Biotech (NASDAQ:KRYS) said it has generated positive interim clinical results for its inhaled cystic fibrosis (CF) ...

Krystal Biotech Announces Positive Interim Clinical Update from KB407 Phase 1 CORAL-1 Study with Confirmation of Wild-Type CFTR Delivery to the Lungs of Patients with Cystic ...

Confirmed wild-type CFTR delivery and expression in conducting airway cells of patients with class I mutations KB407 transduction confirmed in all six patients with successful bronchoscopies ...
Healio

Aerosol gene therapy safe, heightens CFTR protein levels in cystic fibrosis

Please provide your email address to receive an email when new articles are posted on . Patients given aerosolized 4D-710 had elevated CFTR protein levels that went over normal levels. Over 12 months, ...

Sionna Therapeutics to Present at the 44th Annual J.P. Morgan Healthcare Conference

A live webcast of the presentation will be available under the “Events” page within the Investors section of Sionna’s website ...
Kaleido Scope

Gene Expression Core

The Gene Expression Core was founded in 1994 and promotes translational, multidisciplinary CF research by furnishing expertise concerning recombinant CFTR and other proteins relevant to CF disease ...
News Medical

CFTR modulator can be safe, well-tolerated for younger children with common form of cystic fibrosis

Children ages two to five who have the most common form of cystic fibrosis (CF), caused by two copies of the F508 gene mutation, have not had any modulator treatments available to them until recently.
Business Wire

Vertex Announces Investigational New Drug (IND) Application for VX-522, mRNA Therapy for People With Cystic Fibrosis, Cleared by FDA

BOSTON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration has cleared its Investigational New Drug (IND) application for VX ...
Science Daily

Better understanding of the defective protein that causes cystic fibrosis

Scientists studying the protein that, when defective or absent, causes cystic fibrosis has made an important discovery about how that protein is normally controlled and under what circumstances it ...