ADDRESSING THE VALUE OF GENE THERAPY: ENHANCING PATIENT ACCESS TO TRANSFORMATIVE TREATMENTS

May 1, 2018

Gene therapy is a radical shift in our approach to disease treatment. By modifying the expression of a patient’s genes or repairing abnormal genes, gene therapy often addresses the root cause of diseases. While several new gene and cell therapies have received FDA approvals over the past 20 years, the field has recently experienced a turning point. Three gene therapies were approved for human medical use in the U.S. in 2017, including the first in the country for an inherited condition, and many more are likely on the way.

Spotlight

Heat Biologics

Heat Biologics is a clinical-stage company focused on developing its proprietary ImPACT™ (Immune Pan-Antigen Cytotoxic Therapy), a fully allogeneic (“off-the-shelf”) cell-based immunotherapy for use in the treatment of a wide range of cancers.

OTHER WHITEPAPERS
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Leveraging innovations in plasmid manufacturing to bring advanced therapies to market

whitePaper | December 13, 2022

The global demand for plasmid DNA has increased dramatically in recent years, fueled by a surge in the clinical development of next-generation cell and gene therapy products and more recently by the success of COVID-19 vaccines.

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Application of Single-Use Technologies(SUT) in Biosimilar Development

whitePaper | December 20, 2022

The current processing paradigm of large manufacturing facilities dedicated to singleproduct production is no longer an effective approach for best manufacturing practices.

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The US Bioeconomy

whitePaper | April 14, 2022

In the nearly 50 years since the first genetic engineering experiments, the United States has become the world’s biotechnology powerhouse,

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Cell Lysate Sample Handling with Simoa Bead-Based Assays

whitePaper | November 12, 2019

This document describes a novel Quanterix lysate diluent for running lysate samples with Simoa assays diluent.

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IoT To The Rescue For Biotech & Pharma Manufacturing

whitePaper | April 7, 2020

The Internet of Things (IoT) could not be timelier for biotech/pharma. With fierce cost pressures, the push for value-based outcomes, and the need for flexible manufacturing solutions, the IoT is the much-needed shot in the arm to bring about transformational change. Sensors, actuators, and devices (“things”) embedded in production equipment and networked through computer systems can generate an enormous amount of data. The data can be mined for insights and opportunities to drive production efficiency, automate monitoring and controlling functions, and enable flexible manufacturing systems.

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2022 Global Life Sciences Outlook

whitePaper | January 7, 2022

While valuations for the life sciences sector in this past year have been mixed, the underlying performance and outlook for the sector is healthy

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Spotlight

Heat Biologics

Heat Biologics is a clinical-stage company focused on developing its proprietary ImPACT™ (Immune Pan-Antigen Cytotoxic Therapy), a fully allogeneic (“off-the-shelf”) cell-based immunotherapy for use in the treatment of a wide range of cancers.

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