Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the ...

Novel Therapy Could Help People With Asthma, COPD, Cystic Fibrosis and Cancer-Related Lung ... may result in a greater risk of ... Altered Gene Expression and Cell Interactions Involved in COPD ...

Strategically focused pipeline optimizes resource allocation to progress two product candidates with strongest clinical proof of concept: 4D-150 for wet AMD and DME and 4D-710 for cystic fibrosis ...

Strategically focused pipeline optimizes resource allocation to progress two product candidates with strongest clinical proof of concept: 4D-150 for wet AMD and DME and 4D-710 for cystic fibrosisAfter ...

Ivacaftor Oral Granules, 25 mg, 50 mg and 75 mg per unit dose packet are indicated for cystic fibrosis treatment in patients ...

A new treatment for cystic fibrosis has been approved by the U.S. Food and Drug Administration (FDA).

Fair Therapeutics B.V. has completed enrollment of its Phase IIb CHOICES trial, evaluating the novel CFTR triple modulator therapy, Diponecaftor, for adults with ultra-rare cystic fibrosis (CF) ...

New breakthroughs in gene editing mean that cures for genetic diseases such as cystic fibrosis could be developed in the near ...

Krystal Biotech's VYJUVEK, a groundbreaking gene therapy for DEB ... In respiratory, KB407 is being investigated for cystic fibrosis, the most common inherited genetic disorder in the US.

Credit: Vertex. According to Vertex Pharmaceuticals, approximately 300 additional people with CF are now eligible to receive treatment. An additional 94 non-F508del CFTR mutations and a boxed ...

and the absolute change from baseline in the Cystic Fibrosis Questionnaire-Revised respiratory domain score through week 24 were also similar between both the Alyftrek and ELX/TEZ/IVA groups.

In the LIGHTHOUSE study, Atsena Therapeutics is evaluating ATSN-201 gene therapy for X-linked retinoschisis, leveraging AAV.SPR capsid for central retina transduction without foveal detachment risks.