Surprising no one, Jennifer Doudna and Emmanuelle Charpentier won the 2020 Nobel Prize in Chemistry “for the development of a method for genome editing,” that is, the CRISPR/Cas9 system. CRISPR has transformed many aspects of biological research in the eight years since the 2012 publication of Charpentier’s and Doudna’s paper describing the programmable use of tracrRNA, which Charpentier and her team had described in 2011, to target specific DNA sequences for editing. The work was more or less instantly recognized as worthy of a Nobel Prize. Certainly, Charpentier and Doudna were clear contenders for the award. But so was someone who might be considered the missing third awardee: Feng Zhang, whose laboratory was the first to use the technology to edit mammalian cells.

Genor raises $347M via HK IPO, aims to strengthen breast cancer franchise

BEIJING – Biologics developer Genor Biopharma Co. Ltd. raised HK$2.69 billion (US$347 million) via a pre-revenue IPO on the Hong Kong Stock Exchange on Oct. 7, with shares trading 16.25% higher to close at HK$27.95. Backed by Goldman Sachs, JP Morgan and Jefferies, the Shanghai-based biopharma issued nearly 119.9 million shares at HK$24 apiece, the top end of its proposed price range. It is the latest pre-revenue biotech IPO to be highly sought after by retail investors in Hong Kong, after Ocumension Therapeutics Ltd.’s $190 million IPO, which was oversubscribed by nearly 1,900 times in July.

Lilly seeks EUA for COVID-19 antibody treatment

With an ongoing phase II trial showing that a combination of two SARS-CoV-2 neutralizing antibodies from Eli Lilly and Co. has so far reduced viral load, symptoms and COVID-19-related hospitalization and ER visits, Lilly has asked the FDA to consider an emergency use authorization for one of the components, bamlanivimab, also known as LY-CoV555. Lilly said it expects to seek an EUA for the combination therapy in November. Separately, Taiwan Liposome Co. Ltd. (TLC) said it has received regulatory blessings to start a phase I trial of TLC-19, the company's liposomal suspension of hydroxychloroquine for inhalation in 30 healthy volunteers. Data from the trial will serve as a basis for subsequent trials in patients with COVID-19. TLC's U.S.-listed shares (NASDAQ:TLC) were up 32.5% by midday.

Vaccine guidance, adcom all a matter of raising public trust

With COVID-19 cases once again surging across the globe and several countries considering targeted lockdowns, vaccines remain the best hope of restoring a sense of normalcy amidst the pandemic. For vaccines to work though, people must have enough confidence in their efficacy and safety that they’re willing to get vaccinated. That’s why the guidance the FDA released yesterday on what it will take for a SARS-CoV-2 vaccine to be authorized for emergency use is so important. On its face, the guidance is aimed at advising vaccine sponsors through the emergency use authorization (EUA) process. But at the core, the guidance is intended to ensure the public that vaccine EUAs will be granted based on robust scientific data and a rigorous review process that includes advice from an independent advisory committee of vaccine and medical experts.

Growing concerns over decline in non-COVID-19 clinical testing, says U.K.’s ABPI

LONDON – Industry is calling on the government to set out a plan for the safe and sustainable restart of non-COVID-19 clinical studies that were put on hold when the pandemic struck. Although restrictions were lifted in May, only 45% of trials that were suspended had reopened to recruitment as of Sept. 9, new trials are not starting and there is increasing concern about progress. Problems remain with patient recruitment, trial oversight, long waiting times for non-COVID referrals and a lack of research staff.

Joint adcom to take up Alkermes’ antipsychotic; briefing docs flag opioid use

Briefing documents for the meeting Oct. 9 of the FDA’s Psychopharmacologic Drugs Advisory Committee and the Drug Safety and Risk Management Advisory Committee have been posted. The joint session will review Dublin-based Alkermes plc’s NDA for ALKS-3831 (olanzapine/samidorphan), an oral, once-daily atypical antipsychotic candidate for adults with schizophrenia and for adults with bipolar I disorder. Evercore ISI analyst Umer Raffat called the documents “fairly benign,” with the main concern involving whether concomitant opioid use with ALKS-3831 might lead to withdrawal symptoms or an opioid overdose.

Bone Therapeutics inks Asian market deals for bone cell therapy platform

HONG KONG – Belgium-based Bone Therapeutics SA has inked development and commercialization deals for its bone cell therapy platform in Chinese and Southeast Asian markets. Shenzhen Pregene Biopharma Co. Ltd. has the exclusive rights to clinically develop and commercialize the Allob platform for the treatment of human bone disorders in greater China (including Hong Kong and Macau), while China-based Link Health Pharma Co. Ltd. will get the same rights for Taiwan, Singapore, South Korea and Thailand. In return, Bone Therapeutics is eligible to receive up to €55 million (US$65 million) in development, regulatory and commercial milestone payments ,including €10 million in up-front and milestone payments anticipated in the next 24 months.

Janpix targets STAT3/5 with a $10M series B

Janpix Inc., of Cambridge, Mass., has raised a $10 million series B designed to progress its monovalent small-molecule protein degraders of STAT3 and STAT5 into final preclinical studies and eventually into the clinic to treat various hematological and solid tumor cancers. Both targets are associated with the proliferation of tumor cells, their survival and resistance. STAT3 has been known to regulate genes implicated in oncogenesis, tumor immune evasion, inflammation and fibrosis. The science behind Janpix is based on the work of Patrick Gunning, a company co-founder and professor of chemistry at the University of Toronto Mississauga. The round was led by Medicxi, which seeded and funded Janpix’s $19 million 2017 series A funding.

Astrazeneca strikes deal with Impedimed for Sozo platform in heart failure, kidney disease trial

PERTH, Australia – Astrazeneca plc is leasing 175 Sozo devices from Impedimed Ltd. and plans to put them in 20 countries to run phase II trials for a combination drug that looks at fluid imbalance in the human body in patients with heart failure and chronic kidney disease.

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