Olivia Vizier

ProQR’s RNA therapy on track to pivotal tests in rare, inherited form of vision loss

ProQR Therapeutics has encouraging early data from a small study that show its experimental RNA-based therapy for a rare, inherited form of vision loss helped patients recover some of their sight. Based on those results, the company is planning to advance its therapy to two pivotal studies that could start later this year.

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Targeting and Modulating CRAC Channels to Treat Acute Pancreatitis and COVID-19 with Dr. Sudarshan Hebbar CalciMedica

Dr. Sudarshan Hebbar, Chief Medical Officer, CalciMedica talks about developing drugs that are focused on targeting CRAC channels, calcium release activated calcium channels, in order to modulate these channels to help treat diseases that have a significant inflammatory component.  Initially developed drugs for acute pancreatitis targeting both the pancreas and the lung. Now conducting large

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Clene is Using Nanocrystals of Pure Elements to Change Neurodegenerative Treatment

That is the mission of Clene Nanomedicine, which has combined cell biology, solid state physics, and materials science to pioneer the development of Clean-Surfaced Nanocrystal (CSN) therapeutics. The company’s goal is to use this process to enhance the energetic support in neurons and change the way neurodegenerative diseases are treated. PM360 spoke with President and CEO Rob

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How to Fake a Professional Headshot at Home

It’s hard to believe that this month marks one year of the pandemic. This past year has been an arduous journey that none of us could have ever anticipated. Throughout that journey, I believe we all have learned a tremendous amount about ourselves and how resourceful we are. Prior to the pandemic, most of us

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C&G Therapy Manufacturing Automation: Making The Most Of Today’s Opportunities

SOTIO — highlighted a variety of ways the industry could address the high cost of making these therapies. Despite the fact each of these experts is focused on different types of cell therapies (allogenic and/or autologous) and therapeutic indications, their mindset was strikingly aligned around just how innovative a simplified C&G therapy manufacturing process would

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endpoints

Taiwan’s Acepodia closes Series B with the promise of conjugating antibodies and off-the-shelf NK cells

Acepodia closed its Series B round Tuesday morning, hoping to build upon CEO Sonny Hsiao’s work from when he was a biologist at UC Berkeley. The biotech plans to use the funds to advance its lead program through the end of a Phase I trial and launch a Phase II study, as well as to

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Pharma's Almanac

Q: Putting aside COVID-19 vaccines and therapeutics, what do you think will be the most significant development in the industry in 2021?

A relevant topic in the healthcare space that has arisen amid the COVID-19 pandemic is the alarming drop in cancer screening rates globally due to limited access of patients to hospitals settings and cancellations of non-elective procedures, which will inevitably result in increased cancer deaths in coming years. The pandemic has reemphasized the relevance of

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Developing Accessible Monoclonal Antibodies to Treat COVID-19 with Vu Truong Aridis Pharmaceuticals

Vu Truong, CEO, Aridis Pharmaceuticals is taking on the challenge of making an inhaled monoclonal antibody to treat COVID-19 to allow for the easier patient access.  Drawing on their work in infectious diseases and with a number of monoclonal antibodies candidates to treat bacteria infection associated with superbugs and antibiotic resistance, Aridis is  finding ways

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Aveo teams with Bristol Myers after surprise FDA approval; CalciMedica lines up $21M raise to advance Covid-19 candidate

California-based CalciMedica closed a Series D round on Thursday to advance its portfolio of calcium release-activated calcium (CRAC) inhibitors, including Auxora, which is being tested in both Covid-19 and acute pancreatitis patients.

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The US Has 3 Covid Vaccines—Testing New Ones Is Complicated

The push abroad raises fresh concerns over clinical trials exploiting economically developing nations. Vaxart, maker of a potential Covid vaccine that comes in tablet form, is gearing up for a Phase II trial. The company plans to consult foreign ministries of health and ethics committees to set up ethical trials. That might include a crossover study that

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endpoints

Looking to take advantage of ‘silenced’ enzymes, SalioGen emerges from stealth with eyes set on gene therapy 3.0

That’s the question a new biotech is aiming to answer, as SalioGen Therapeutics emerges from stealth Monday morning with a $20 million Series A. And they believe they’ve found a new delivery system that can more precisely deliver genes in vivo than the relatively large adeno-associated virus or CRISPR system: mammalian-derived enzymes.

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The Fight Against Fungi

Cidara Therapeutics is exploring echinocandin derivatives. The company’s clinical candidate rezafungin is a derivative of anidulafungin, a compound originally discovered at Eli Lilly and Company and currently marketed by Pfizer. Chemists placed a choline group at a critical point on the molecule, a feature that stabilizes the echinocandin ring and prevents metabolic breakdown of the molecule,

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