BioCentury | Oct 6, 2020
Product Development

FDA pushing Makena, early drug pricing target, off the market

FDA has proposed the withdrawal of AMAG’s Makena, sending the drug, and the women who have counted on it to prevent premature births, into what could be the final turn in a roller-coaster ride that...
BioCentury | Oct 2, 2020
Regulation

Oct. 1 Quick Takes: Setback at FDA for Mesoblast; plus AMAG, Solid, COVID updates from Gilead, AZ and more

Bucking ODAC recommendation, FDA wants another trial from MesoblastMesoblast Ltd. (ASX:MSB; NASDAQ:MESO) said FDA issued a complete response letter to a BLA for remestemcel-L to treat pediatric steroid-refractory acute GvHD. FDA’s Oncologic Drugs Advisory Committee had voted 9...
BioCentury | Aug 28, 2020
Distillery Therapeutics

Potassium channel-vesicle interaction as a target in ischemic stroke

DISEASE CATEGORY: Neurology INDICATION: Stroke Interrupting the interaction between the potassium channel KCNB1 and VAPA, a vesicle membrane protein, could treat ischemic stroke. In cultured rat cortical...
BioCentury | May 13, 2020
Emerging Company Profile

QurAlis taking three ALS programs toward clinic with $42M series A

After launching with a modest seed round in 2018, neurology company QurAlis is driving toward the clinic with a $42 million series A round to fund development of three therapies to treat amyotrophic lateral sclerosis...
BioCentury | Dec 3, 2019
Company News

Xenon licenses sodium channel inhibitors to Neurocrine to fund more advanced epilepsy programs

Xenon has exclusively licensed its sodium channel inhibitors for epilepsy to Neurocrine to focus on advancing its potassium channel modulators through the clinic. The deal adds a new indication to Neurocrine's neurology portfolio and gives...
BioCentury | Oct 30, 2019
Company News

Oct. 29 Company Quick Takes: Incyte’s JAK inhibitor beats expectations; plus Sumitomo-Intercept, Merck KGaA-Pfizer, AMAG, Regeneron-Sanofi

Incyte surges on Jakafi sales, guidance On the strength of 3Q19 sales of Jakafi ruxolitinib that beat estimates and resulted in an increase in full-year guidance, Incyte Corp. (NASDAQ:INCY) climbed $3.49 to $83.35 Tuesday, adding...
BioCentury | Jun 27, 2019
Product Development

Gene therapy’s next frontier lies beyond rare, monogenic diseases

...widen that window, said Kullmann. His UCL lab is developing a gene therapy that delivers KCNA1...
...Mass. Voyager Therapeutics Inc. (NASDAQ:VYGR), Cambridge, Mass. Targets DDC (AADC) - Dopa decarboxylase KCNA1 - Potassium channel Kv1.1...
...decarboxylase (DDC) (AADC) GATA binding protein 4 (GATA4) Microtubule-associated protein tau (tau) (MAPT) (FTDP-17) Myocyte enhancer factor 2C (Mef2c) Potassium channel Kv1.1 (KCNA1) T-box...
BioCentury | Jun 4, 2019
Financial News

June 4 Financial Quick Takes: Lung Therapeutics' $36M series C; plus Strand, Hansoh, Inhibrx and more

Lung Therapeutics closes $36M series C Lung Therapeutics Inc. (Austin, Texas) raised $36 million in a series C round led by Bios Partners with participation by undisclosed new and existing investors. Lung has raised $53...
BioCentury | May 15, 2019
Translation in Brief

Lentiviral gene therapy could offer relief to refractory epilepsy patients

...Journal of Neuroscience paper published last month, the authors showed that a gene therapy encoding KCNA1...
...commercial partners are underway. Targets: CAMK2A - Calcium calmodulin-dependent protein kinase II α; KCNA1 - Potassium channel Kv1.1 Elizabeth...
...Potassium channel Kv1.1 Elizabeth S. Eaton, Staff Writer Medical Research Council University College London Calcium calmodulin-dependent protein kinase II alpha (CAMK2A) Potassium channel Kv1.1 (KCNA1) Epilepsy gene...
BioCentury | May 6, 2019
Distillery Therapeutics

KCNA1 gene therapy for epilepsy

...Rat studies suggest KCNA1-based gene therapy could help treat epilepsy. The gene therapy consisted of KCNA1...
...steps include clinical testing of the lentiviral vector-encoded therapy in refractory neocortical epilepsy patients. TARGET/MARKER/PATHWAY: Potassium channel Kv1.1 (KCNA1)...
...same affiliation as above email: m.walker@ucl.ac.uk Elizabeth S. Eaton University College London Calcium calmodulin-dependent protein kinase II alpha (CAMK2A) Potassium channel Kv1.1 (KCNA1) Epilepsy AAV...
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