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Xconomy 10/14/2020 06:00
Roche made a splash with its acquisition of Spark Therapeutics, whose Luxturna, a gene therapy for an inherited form of vision loss, was the first such therapy to win FDA approval. Now the Swiss pharmaceutical giant is looking to bolster its capabilities in liver and central nervous system (CNS) disorder gene therapies and it’s turning to Dyno Therapeutics to help. Dyno, a two-year-old startup based in Cambridge, MA, has never brought a gene therapy to the market, nor does it currently have its own drug pipeline. What it does have is a platform technology for engineering the viruses that ferry gene therapies to their cellular targets. Going further, it can design the capsids, the protein shells of these viruses that envelop a gene therapy a.
Xconomy 10/08/2020 15:16
A US Food and Drug Administration advisory committee meeting on Biogen, Inc.’s Alzheimer’s drug aducanumab will offer some insight into the amount of flexibility the agency and its external experts are willing to exercise for neurodegenerative diseases with high unmet need, particularly in the wake of a December 2019 guidance document on the quantum and [...]
Xconomy 10/08/2020 13:11
Arrowhead Pharmaceutical’s gene silencing treatment for a rare liver disease will move through the regulatory process and potential commercialization with the muscle of Takeda Pharmaceutical behind it. Takeda (NYSE: TAK) has agreed to pay $300 million up front in a partnership on Arrowhead (NASDAQ: ARWR) drug ARO-AAT as a treatment for alpha-1 antitrypsin-associated liver disease. […]
Xconomy 10/08/2020 09:41
Science is the way out of this pandemic—and scientific breakthroughs are on the ballot on November 3. To help you get ready for Election Day, BIO’s initiative Biotech Votes has resources to help you get registered and get informed. First, make sure you’re registered to vote. Check out this handy form to verify your voter […]
Xconomy 10/06/2020 16:02
The first generation of CRISPR-based therapies showed how the technology can edit DNA, making genetic fixes to unwanted mutations. But those treatments use the CRISPR system as scientists found it in nature, which means it still has limitations when applied to humans, says Benjamin Oakes, CEO of Scribe Therapeutics. Scribe aims to develop gene-editing therapies […]
Xconomy 10/06/2020 07:30
Scientists at microbiome therapy developers often talk about the importance of community, how the bugs in our gut act in concert in addition to their individual roles. That’s important for microbiome health and for microbe-based therapies, says Emily Drabant Conley, CEO of startup Federation Bio. Conley’s company is developing therapies that build on the understanding of […]
Xconomy 10/05/2020 09:35
CStone Pharmaceuticals has touted its experimental drug sugemalimab as potentially the best in its class of cancer immunotherapies. Pfizer is placing a bet on that promise by making a $200 million equity investment in CStone for rights to that drug in China. According to deal terms announced late Tuesday, Pfizer (NYSE: PFE) is purchasing nearly […]
Xconomy 10/05/2020 09:34
[Updated 10/1/2020. See below.] Genfit is halting all work on its lead drug in the fatty liver disorder nonalcoholic steatohepatitis (NASH) and implementing a corporate restructuring that will cut 40 percent of its staff, splitting the remaining workforce into two distinct business units. The announcement after the market close on Wednesday follows the May failure […]
Xconomy 10/05/2020 09:33
Solid Biosciences has answered the FDA’s outstanding questions about its Duchenne muscular gene therapy, clearing a clinical trial to now resume after patient complications last year led the agency to halt the study for a second time. Cambridge, MA-based Solid Bio (NASDAQ: SLDB) said Thursday that it expects dosing of patients in the Phase 1/2 […]
Xconomy 10/05/2020 09:31
Amid the bombast of Tuesday’s presidential debate, viewers caught some discussion about the response to the coronavirus pandemic and efforts to develop a COVID-19 vaccine. In the crosstalk, the president made an unsupported claim that we are weeks away from having vaccines. It’s true that some pharmaceutical companies are already manufacturing their COVID-19 vaccine candidates […]

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