Therapy Areas: Hereditary Disorders
Vertex Receives FDA Clearance of Investigational New Drug Application for VX-264, a Novel Encapsulated Cell Therapy for the Treatment of Type 1 Diabetes
16 March 2023 - - The US Food and Drug Administration has cleared the Investigational New Drug Application for VX-264, a stem cell-derived, fully differentiated pancreatic islet cell therapy encapsula...
Aluna Raises USD 15.3m in Series B Funding
6 February 2023 - - US-based lung health management platform Aluna has completed a USD 15.3m series B round of financing to continue growing its solution among doctors and patients managing asthma, cy...
First Patient Dosed in Adaptive Phage Therapeutics' PHAGE Clinical Trial Evaluating Phage Therapy in Cystic Fibrosis-related Respiratory Infection
13 January 2023 - - The first patient has been dosed in the PHAGE clinical trial, evaluating bacteriophage therapy in adults with cystic fibrosis who carry Pseudomonas aeruginosa (...
CF Foundation Invests Up to USD 15m in ReCode Therapeutics for Development of mRNA Therapy
11 January 2023 - - The Cystic Fibrosis Foundation will invest up to USD 15m in ReCode Therapeutics for the development of its messenger RNA therapy that could eventually provide a treatment option fo...
Markels Named to Arcturus Therapeutics Board of Directors
19 December 2022 - - US-based clinical messenger RNA medicines company Arcturus Therapeutics Holdings Inc. (NASDAQ: ARCT) has appointed John Markels, Ph.D., a pharmaceutical executive with over 35 yea...
SpliSense commences first-in-human, Phase 1/2 clinical trial of SPL84 to treat cystic fibrosis
15 December 2022 - SpliSense, an Israel-based biopharmaceutical company, announced on Wednesday that it has started a first-in-human, Phase 1/2 clinical trial of SPL84, the company's lead anti se...
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US FDA Approves Orkambi in Children with Cystic Fibrosis Ages 12 to <24 Months

Arcturus Reports Durability Data Against Omicron Variants, Including BA.5, at Six-Month Timepoint Following Booster with ARCT-154 COVID-19 Vaccine

EpiAxis Therapeutics and Peptilogics Enter Strategic Drug Discovery Partnership

Vicinitas Therapeutics Launches with USD 65m in Series A Financing

Endo Ventures signs development and distribution contract with Quoin Pharmaceuticals

ReCode Therapeutics Closes Series B Extension Financing Round

First Two Patients Dosed in Phase 1b/2a Study of BX004 for Treatment of Chronic Respiratory Infections in Patients with Cystic Fibrosis

Health Canada Grants Marketing Authorization for Trikafta in Children with Cystic Fibrosis Ages 6 Through 11 years With At Least One F508del Mutation

Cystic Fibrosis Foundation Invests USD 5m in Sionna Therapeutics to Develop New CFTR Modulator Therapy

Laurent Pharmaceuticals Starts Phase 3 Study with Once-a-Day Oral COVID-19 Antiviral Treatment Candidate in Hospitalized Moderate-to-Severe Patients

Cyclica Launches University of Toronto Spinout Perturba Therapeutics

Northwestern Medicine's study shows lung transplantation highly effective and successful in critically-ill COVID-19 patients