manilatimes5d
4DMT Focuses Pipeline to Prioritize 4D-150 in Wet AMD & DME and 4D-710 in CF and Extends Cash Runway
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 ...
The Bakersfield Californian5d
4DMT Focuses Pipeline to Prioritize 4D-150 in Wet AMD & DME and 4D-710 in CF and Extends ...
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 ...
Daily5d
Lupin Cystic fibrosis drug gets tentative approval from USFDA
Ivacaftor Oral Granules, 25 mg, 50 mg and 75 mg per unit dose packet are indicated for cystic fibrosis treatment in patients ...
lifesciencesweden.se6d
FDA approves new cystic fibrosis therapy
A new treatment for cystic fibrosis has been approved by the U.S. Food and Drug Administration (FDA).
Le Lézard7d
Fair Therapeutics Completes Enrollment in Phase IIb CHOICES Trial of Novel CFTR Modulator for Ultra-Rare Variants of Cystic Fibrosis
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) ...
Newstalk11d
Luke O'Neill: The Science behind gene editing
New breakthroughs in gene editing mean that cures for genetic diseases such as cystic fibrosis could be developed in the near ...
Seeking Alpha19d
Krystal Biotech: Blockbuster Ambitions For Topical Gene Therapy
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.
Monthly Prescribing Reference20d
FDA Approval Increases Eligibility for Cystic Fibrosis Medication Trikafta
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 ...
clinicaladvisor.com20d
Alyftrek Gets FDA Approval for Cystic Fibrosis
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.
ophthalmologytimes21d
Gene Therapy
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.
Medindia22d
India’s Medical Milestone: Gene Therapy Success for Haemophilia A
Integrated gene therapy offers long-term relief for Haemophilia A Lentiviral vectors ensure safer treatment without immunosuppressants Clinical trial success boosts India’s global gene therapy ...
Healio22d
FDA approves Alyftrek for cystic fibrosis treatment
The FDA has approved Alyftrek for patients aged 6 years and older with cystic fibrosis with certain mutations ... these studies assessed use of the therapy taken once a day over 52 weeks in ...