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11:25 AM, Oct 12, 2018  |  BC Week In Review | Clinical News  |  Preclinical Results

Combinations of CF correctors from McGill could rescue more mutations

Researchers from McGill University discovered three novel correctors of cystic fibrosis transmembrane conductance regulator (CFTR) and showed that a combination of the small molecules could correct the most common CF mutation, F508del, as well as...
12:27 PM, Oct 05, 2018  |  BC Week In Review | Clinical News  |  Preclinical Results

Blueprint debuts selective ALK2 inhibitor for rare musculoskeletal disease

Blueprint Medicines Corp. (NASDAQ:BPMC) provided the first glimpse of its preclinical ALK2 inhibitor program, BLU-782, when it presented data at the American Society for Bone and Mineral Research (ASBMR) meeting in Montreal on Sept. 30....
3:41 PM, Sep 13, 2018  |  BC Week In Review | Clinical News  |  Preclinical Results

4D's MRx0004 improves inflammation in asthma model

4D Pharma plc (LSE:DDDD) said once-daily oral MRx0004 protected against airway inflammation by decreasing infiltration of both neutrophils and eosinophils in the lung bronchoalveolar lavage fluid of a house dust mite mouse model of severe...
10:43 AM, Sep 07, 2018  |  BC Week In Review | Clinical News  |  Preclinical Results

Exonics shows CRISPR treats DMD in dogs

Exonics Therapeutics Inc. (Boston, Mass) is one step closer to bringing its SingleCut CRISPR gene editing Duchenne muscular dystrophy treatment to the clinic after preclinical data showed the therapy led to dystrophin restoration of up...
9:45 AM, Sep 07, 2018  |  BC Week In Review | Clinical News  |  Preclinical Results

Bridge Biotherapeutics's BBT-877 reduces lung fibrosis in mouse model of IPF

Bridge Biotherapeutics Inc. (Seongnam, South Korea) said BBT-877 reduced lung fibrosis as measured by Ashcroft scores and deposition of collagen in a mouse model of bleomycin-induced idiopathic pulmonary fibrosis (IPF). Data were presented at the...
9:45 AM, Sep 07, 2018  |  BC Week In Review | Clinical News  |  Preclinical Results

Bifunctional agonist could treat pain, opioid dependency

A team from Astraea Therapeutics LLC (Mountain View, Calif.) and Wake Forest Baptist Medical Center developed a bifunctional agonist targeting two opioid receptors that could treat pain without opioid-related side effects and reverse opioid dependency. Opioids...
10:41 AM, Aug 31, 2018  |  BC Week In Review | Clinical News  |  Preclinical Results

SAB's SAB-139 protects non-human primates from Ebola virus infection

SAB Biotherapeutics Inc. (Sioux Falls, S.D.) reported data from 12 rhesus macaques challenged with a lethal dose of Ebola virus showing that SAB-139 given one or three days postinfection conferred 100% protection against Ebola virus...
8:30 PM, Aug 16, 2018  |  BC Week In Review | Clinical News  |  Preclinical Results

SOD1 antisense oligonucleotide improves survival in ALS model

Researchers at Biogen Inc. (NASDAQ:BIIB), Ionis Pharmaceuticals Inc. (NASDAQ:IONS) and colleagues reported data from a rat model of superoxide dismutase 1 (SOD1)-mutated amyotrophic lateral sclerosis (ALS) showing that a single bolus dose of intrathecal IONIS-SOD1Rx...
11:55 AM, Jul 13, 2018  |  BC Week In Review | Clinical News  |  Preclinical Results

Cold Harbor JV developing copper chelator for Wilson's disease

DepYmed Inc., a joint venture between Cold Spring Harbor Laboratory and Ohr Pharmaceutical Inc. (NASDAQ:OHRP), is developing its protein tyrosine phosphatase 1B (PTP-1B; PTPN1) inhibitor DPM-1001 as a copper chelator for Wilson's disease. In a...
9:29 AM, May 04, 2018  |  BC Week In Review | Clinical News  |  Preclinical Results

uniQure reports preclinical data for Huntington's disease candidate

uniQure N.V. (NASDAQ:QURE) reported preclinical data showing that AMT-130 decreased mutant huntingtin (HTT) protein in animal models of Huntington's disease. Data were presented at the American Academy of Neurology meeting in Los Angeles. In a pig...

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