Today's Rundown Sangamo slides on first genome-editing trial results Ramaswamy loses another key executive as Fouse jumps ship to Agios Sean Harper and Beth Seidenberg unite to run $320M VC fund Teva finally dumps laquinimod, ending failure-riddled saga With promising data, ProQR Therapeutics' RNA-based eye drug heads to phase 3 Radius Health hires oncology-focused CMO as it moves into breast cancer trials Fulcrum Therapeutics gets off $80M series B round PhaseBio nets $34M to advance Brilinta reversal agent, PAH drug Biohaven picks up AstraZeneca CNS drug, plans phase 3 Startup Gossamer Bio poaches Bristol-Myers Squibb executive as CSO Featured Story | | | Wednesday, September 5, 2018 Sangamo’s big chance has come to show it has a safe and efficient gene-editing technique to rival CRISPR—and the initial response has been a little mixed. |
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| This week's sponsor is Trianni. | | | |
Top Stories Wednesday, September 5, 2018 It has not been a great 12 months for Vivek Ramaswamy’s constellation of biotechs—seeing a major meltdown at Axovant with failures and a fleeing CEO—and now it’s losing another star biotech player in Jackie Fouse, Ph.D. Wednesday, September 5, 2018 Sean Harper, M.D., has revealed his next move. The former head of R&D at Amgen is teaming up with Beth Seidenberg, M.D., to lead investing activities at Westlake Village BioPartners, a new West Coast venture capital shop that has $320 million to pump into biotechs. Wednesday, September 5, 2018 Teva has finally given up on laquinimod. The aryl hydrocarbon receptor activator was once hailed as a blockbuster heir to Copaxone but suffered a string of late-phase failures that dragged down Teva and its partner Active Biotech. Wednesday, September 5, 2018 ProQR Therapeutics reported interim phase 1/2 data for its RNA-based treatment for LCA10, a severe form of blindness caused by a rare genetic disorder. The majority of patients had a “clinically meaningful” improvement in vision after three months of treatment, and the Dutch biotech is planning a pivotal trial for 2019. Tuesday, September 4, 2018 Endocrine therapy developer Radius Health has brought on Charles Morris to be its new chief medical officer and oversee the company’s expansion into oncology, with two breast cancer treatments currently in early-phase clinical trials. Wednesday, September 5, 2018 Fierce 15 winner Fulcrum Therapeutics has pulled in an impressive $80 million series B financing deal as it looks to push on with its lead drug into clinical testing. Wednesday, September 5, 2018 PhaseBio raised $34 million in its series D round, which will advance its lead assets: a treatment for pulmonary arterial hypertension (PAH) developed in-house and a reversal agent of AstraZeneca’s clot-busting Brilinta, licensed from the British pharma in 2017. Wednesday, September 5, 2018 Biohaven Pharmaceutical has licensed an experimental treatment of the neurodegenerative disorder multiple system atrophy from AstraZeneca. The deal follows a midphase trial that linked oral myeloperoxidase inhibitor AZD3241 to numerical improvements on a scale of MSA symptoms. Wednesday, September 5, 2018 Gossamer Bio has tapped Bristol-Myers Squibb’s immunology and cancer R&D executive Luisa Salter-Cid, Ph.D., as its new research lead. Resources Sponsored by: Patheon, part of Thermo Fisher Scientific When developing a parenteral, pharmaceutical scientists are faced with a variety of choices – choices that can be conflicting or present differing advantages. Sponsored by: Baxter Baxter BioPharma Solutions’ whitepaper offers a method to address the challenges of tech transfer for lyophilized products from lab-scale to commercial-scale. 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Drug Approval, Manufacturing Quality & Regulation 2018 | Online RESI HealthTech Week September 5-7, 2018 | Boston, MA 2018 Latin America Healthcare Compliance Certification Program September 24-27, 2018 | Sao Paulo, Brazil Drug Development Immersion September 26-27, 2018 | West Windsor, NJ FierceBiotech 3rd Drug Development Forum October 1–3, 2018 | Hilton Boston Logan Airport Hotel | Boston, MA Compliance Certification Program at Seton Hall Law October 8-11, 2018 | Newark, NJ BioImmersion: Biotech for the Non-Scientist October 16-18, 2018 | Chicago, IL Fierce Innovation Awards: Life Sciences Edition 2018 FierceBiotech Executive Summit: London October 30, 2018 | Royal Society of Medicine Understanding Commercialization Within Biopharma Nov 7-8, 2018 | Boston, MA Drug Development Boot Camp® 2018 – Register Now! 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