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) ...
MarketWatch

Cystic Fibrosis Therapeutics Market Set to Reach USD 35.09 Billion by 2033 as Precision CFTR Modulator Therapies Transform Patient Outcomes

The MarketWatch News Department was not involved in the creation of this content. Cystic Fibrosis Therapeutics Market Set to Reach USD 35.09 Billion by 2033 as Precision CFTR Modulator Therapies ...

Vertex Provides Pipeline and Business Updates in Advance of Upcoming Investor Meetings

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced business and program updates ahead of upcoming investor ...
Healio

Cystic fibrosis triple therapy outcomes improve regardless of prior modulators

Please provide your email address to receive an email when new articles are posted on . Triple therapy improved lung function, BMI and other factors. Patients naïve to cystic fibrosis transmembrane ...
Nasdaq

Sionna Therapeutics Presents Preclinical Data on Novel CFTR Modulators at the 48th European Cystic Fibrosis Conference

Sionna Therapeutics, Inc. has announced the presentation of promising preclinical data at the 48th European Cystic Fibrosis Conference in Milan, showing that its nucleotide-binding domain 1 (NBD1) ...

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 mutationsKB407 transduction confirmed in all ...
MarketWatch

ReCode Therapeutics Receives U.S. FDA Orphan Drug Designation for RCT2100 for the Treatment of Cystic Fibrosis

ReCode Therapeutics, a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA and gene correction therapeutics, today announced that the U.S. Food and ...
Seeking Alpha

Vertex Announces European Commission Approval of ALYFTREK®, a New Once-Daily CFTR Modulator for the Treatment of Cystic Fibrosis

- ALYFTREK ® (deutivacaftor/tezacaftor/vanzacaftor) approved in the EU for people with CF 6 years and older with at least one non-class I mutation in the CFTR gene ...
Healio

CFTR modulator use during pregnancy preserves maternal lung function

Please provide your email address to receive an email when new articles are posted on . “[These findings] help clinicians be more supportive of a female with CF becoming pregnant,” Raksha Jain, MD, ...
EurekAlert!

Research shows positive impact of CFTR modulators on pregnancy outcomes in people with cystic fibrosis

DENVER - A recent retrospective study published in CHEST(Opens in a new window) evaluated the effects of cystic fibrosis transmembrane conductance regulator (CFTR) modulator use on pregnancy outcomes ...
Business Wire

Cystic Fibrosis Foundation Invests $5 Million in Sionna Therapeutics to Develop New CFTR Modulator Therapy

BETHESDA, Md.--(BUSINESS WIRE)--The Cystic Fibrosis Foundation today announced its investment of $5 million in Sionna Therapeutics, a company founded in 2019. The company is focused exclusively on ...
Nasdaq

Sionna Therapeutics to Present Preclinical Data on CFTR Modulators at European Cystic Fibrosis Conference

Sionna Therapeutics, Inc. announced that preclinical data on its novel cystic fibrosis (CF) treatment candidates, SION-451 and SION-719, will be presented at the European Cystic Fibrosis Society’s ...