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Q&A: One scientist’s bold vision to make on-demand treatments routine for life-threatening rare genetic diseases

Broad Institute

Based on a technology developed by Broad Institute core member David Liu’s laboratory, the treatment is the first in a series of new medicines being tested to treat rare diseases by repairing patients’ particular genetic misspellings. s to be produced at scale, making them the standard of care for life-threatening rare genetic diseases.

Treatment 133
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Price of World’s First Malaria Vaccine for Children in Endemic Countries Slashed by Over 50%, Now Under $5

The Pharma Data

This announcement was made as part of their pledges to Gavi, the Vaccine Alliance, ahead of Gavi’s sixth replenishment cycle (Gavi 6.0) scheduled for 2026 to 2030. In a landmark move, Bharat Biotech will progressively reduce the price of the RTS,S vaccine to below $5 per dose by 2028—a reduction of more than 50%.

Vaccine 52
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COVAX ANNOUNCES ADDITIONAL DEALS TO ACCESS PROMISING COVID-19 VACCINE CANDIDATES; PLANS GLOBAL ROLLOUT STARTING Q1 2021

The Pharma Data

COVAX now has agreements in place to access nearly two billion doses of several promising vaccine candidates, and laid the groundwork for further doses to be secured through contributions from donors. billion donor-funded doses of approved vaccines in 2021 to the 92 low- and middle-income economies eligible for the COVAX AMC.

Vaccine 52
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World Health Assembly charts course for COVID-19 response and global health priorities

The Pharma Data

For the first time, the world has rallied behind a plan to accelerate the development of the vaccines, diagnostics and therapeutics we need, and to ensure they are available to all countries on the basis of equity. The Access to COVID-19 Tools (ACT) Accelerator is delivering real results. The WHA will consider a draft resolution ( EB146.R10

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Boehringer Ingelheim increases commitments to sustainable development, investing in health innovation and collaborations to expand access and improve healthcare for 50 million people in vulnerable communities by 2030

The Pharma Data

It further evolves Boehringer Ingelheim’s commitments and significantly increases the company’s donation to appreciatively impacting health, societal and environmental issues, in alignment with the United Nations 2030 Sustainable Development Goals.

Vaccine 52
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Bayer expects dynamic growth with higher profitability in the coming years and is accelerating its transformation

The Pharma Data

In the pharmaceutical sector, new technologies could help us not only to treat but probably cure or even prevent diseases,” Baumann said. Sustainability forms an integral part of Bayer’s business strategy, growth plans and incentive systems, Baumann stressed, citing as an example the company’s decision to become carbon-neutral by 2030.

Science 52
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communication, collaboration and complementarity to address real-life problems

The Pharma Data

Many neglected tropical diseases (NTDs) result from diverse types of interactions and often complex cycles of transmission between humans and animals, both vertebrates and invertebrates, and changing social and environmental conditions.

Disease 52