Visualizing molecular glues

Every cell in the human body is packed with proteins. These large molecules float around, bump into one another and make the stuff of life happen. They turn fuel into energy. They dispose of trash. They read genetic codes and coax cells to divide.

In some cells, naturally occurring “molecular glues” float among these proteins. Like crafty matchmakers, these small molecules stick proteins together. By doing so, they may change a cell’s destiny.

Drug hunters at the Novartis Institutes for BioMedical Research (NIBR) think these tiny matchmakers also have the potential to change …

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Caring for someone with heart failure

Available 24 hours a day or living 24 hours away – the carer of someone with heart failure comes in all guises. But where they are all the same is in the important role they play in helping someone with heart failure live with their condition.

Heart failure is complex. It is debilitating and potentially life-threatening. It causes the heart muscle to become stiff or weak, meaning it’s unable to pump enough blood to meet the demands of the body.1 This leaves heart failure patients exhausted and short of breath, severely limiting the activities they are able to do.1 …

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Novartis and life sciences companies commit expertise and assets to the fight against COVID-19 pandemic alongside Bill & Melinda Gates Foundation

  • Collaboration to address product development and scale up challenges posed by current pandemic 

Basel, March 26, 2020 — Today, Novartis and a consortium of life sciences companies announced an important collaboration to accelerate the development, manufacture and delivery of vaccines, diagnostics, and treatments for COVID-19 in response to the pandemic. The industry brings a range of assets, resources, and expertise needed to identify effective and scalable solutions to the pandemic that is affecting billions worldwide. Effects on health systems, …

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Zolgensma® data shows rapid, significant, clinically meaningful benefit in SMA including prolonged event-free survival, motor milestone achievement and durability now up to 5 years post-dosing

  • Interim SPR1NT data showed presymptomatic babies with SMA treated with Zolgensma® (onasemnogene abeparvovec-xioi) soon after birth achieved age-appropriate motor milestones
     
  • In addition to meeting both co-primary efficacy endpoints, nine of 22 patients in the completed pivotal STR1VE-US study demonstrated the “ability to thrive,” a stringent composite endpoint remarkable compared to untreated children with SMA Type 1
     
  • Ongoing START long-term follow-up study (cohort 2) demonstrated sustained durability of Zolgensma, …
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AveXis presents AVXS-101 IT data demonstrating remarkable increases in HFMSE scores and a consistent clinically meaningful response in older patients with SMA Type 2

  • SMA Type 2 patients between two and five years of age who received Dose B met the primary efficacy endpoint with a remarkable mean increase of 6.0 points in HFMSE scores from baseline to month 12, twice the clinically meaningful threshold, as measured by a validated, widely utilized and highly accepted scale developed specifically for older patients with SMA
     
  • Nearly all patients (92%) in this cohort achieved a clinically meaningful ≥3-point increase during the study period, demonstrating a consistent response following gene therapy
      …
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Novartis commits to donate up to 130 million doses of hydroxychloroquine to support the global COVID-19 pandemic response

  • Hydroxychloroquine and a related drug, chloroquine, are currently under evaluation in clinical trials for the treatment of COVID-19.
     
  • Novartis Sandoz division will pursue appropriate regulatory authorizations and, upon approval, will work with stakeholders to determine how best to get this medicine to the patients who need it
     
  • Commitment builds on recently announced USD 20 million Novartis COVID-19 Response Fund, drug discovery and development collaborations and essential medicines price stability
     
  • Novartis …
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Novartis receives approval from Japanese Ministry of Health, Labour and Welfare for Zolgensma® the only gene therapy for patients with spinal muscular atrophy (SMA)

  • Zolgensma® (onasemnogene abeparvovec), a one-time administered gene therapy, is approved for the treatment of SMA in patients under the age of two, including those who are pre-symptomatic at diagnosis
     
  • Zolgensma has demonstrated significant and clinically meaningful therapeutic benefit in symptomatic and pre-symptomatic SMA, including prolonged event-free survival and achievement of motor milestones unseen in natural history of the disease
     
  • Designated as an intractable disease in Japan, SMA is the leading …
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Novartis announces NEJM publication of three pivotal trials showing durable and potent efficacy of inclisiran, an investigational first-in-class siRNA cholesterol-lowering therapy

  • Inclisiran, an investigational medicine, showed durable and potent reduction of low-density lipoprotein cholesterol (LDL-C) in patients with atherosclerotic cardiovascular disease (ASCVD), ASCVD risk equivalents and heterozygous familial hypercholesterolemia (HeFH)1,2 

  • Inclisiran reduced LDL-C at 17 months by 52% in patients with ASCVD (ORION-10), 50% for ASCVD and ASCVD risk equivalents (ORION-11) and by 50% in HeFH patients (ORION-9); all of whom had elevated LDL-C levels despite maximally tolerated lipid-lowering therapy1,2

    …

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Novartis announces broad range of initiatives to respond to COVID-19 Pandemic; Creates USD 20 million global fund to support impacted communities

  • Novartis COVID-19 Response Fund will provide grants of up to USD 1 million to support communities around the world most impacted by the Coronavirus outbreak
     
  • Novartis to join collaborative R&D efforts with the Bill & Melinda Gates Foundation, Wellcome, and Mastercard -supported COVID-19 Therapeutics Accelerator and a partnership with the Innovative Medicines Initiative (IMI)
     
  • Commitments build on Sandoz pledge to maintain price stability of essential medicines and strong support of patients and healthcare systems …
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Novartis announces broad range of initiatives to respond to COVID-19 Pandemic; Creates USD 20 million global fund to support impacted communities

  • Novartis COVID-19 Response Fund will provide grants of up to USD 1 million to support communities around the world most impacted by the Coronavirus outbreak
     
  • Novartis to join collaborative R&D efforts with the Bill & Melinda Gates Foundation, Wellcome, and Mastercard -supported COVID-19 Therapeutics Accelerator and a partnership with the Innovative Medicines Initiative (IMI)
     
  • Commitments build on Sandoz pledge to maintain price stability of essential medicines and strong support of patients and healthcare systems …
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