Idorsia Pharmaceuticals Ltd. has described cystic fibrosis transmembrane conductance regulator (CFTR) modulators reported to be useful for the treatment of cystic fibrosis.
After a midstage trial showed no benefit from adding ELX-02 (exaluren) to Kalydeco (ivacaftor, Vertex Pharmaceuticals Inc.) for certain cystic fibrosis (CF) patients, Eloxx Pharmaceuticals Inc. said it will discuss next steps for the program, its lead candidate, with the trial's sponsor, the CF Foundation. A proof-of-concept trial for EXL-02 in the rare kidney disease Alport syndrome will start later this year, the company said.
A study published in Nature Communications revealed a new antisense oligonucleotide therapy applicable to the W1282X mutation of the cystic fibrosis transmembrane conductance regulator gene in cystic fibrosis.
Although there has been huge progress in treatment of cystic fibrosis over the last decade, with Vertex Pharmaceuticals Inc. becoming the first to address the underlying cause of the disease with its Kalydeco (ivacaftor), approved in 2012, there are still many patients who aren’t eligible for treatment.
Just nine months after a $55 million series B fundraising round, Billiontoone Inc. delivered an oversubscribed series C of $125 million. Demand for the company’s Unity prenatal test, which can assess fetal risk for common recessive conditions and aneuploidies using one maternal blood sample, tripled over the last year.
While full details of the underlying science are due to be published in a peer reviewed journal, Kither Biotech Srl has announced its closure of an €18.5 million (US$20.4 million) series B financing with which it aims to translate its approach to rare respiratory diseases into the clinic.
Swiss biotech Polyphor AG, left hindered by failures in advanced clinical programs it had pursued in breast cancer and bacterial pneumonia, has found a path forward in a planned merger with Enbiotix Inc. The combination would put Enbiotix Chairman and CEO Jeff Wager in charge of a publicly listed company advancing a combined portfolio of programs for rare disease and cancer, including Polyphor’s inhaled antibiotic, murepavadin, for infections in people with cystic fibrosis.
Just weeks after announcing plans to invest billions in an mRNA therapeutics research center, Sanofi SA has acquired Translate Bio Inc. for $3.2 billion, building on a development partnership focused around the technology.
TORONTO – What do ER doctors want most for their patients? Never to return to the ER, said Giovanni Ferrara, a professor at Edmonton’s University of Alberta Hospital's Division of Pulmonary Medicine. Ferrara is heading a feasibility project to see if a wearable device developed by Rochester, N.Y.-based Heath Care Originals Inc. can predict with scientific certainty when the condition of a patient with lung disease is worsening and requires another visit to the hospital.
