XTalks

APRIL 24, 2023

Out of over 7,000 rare diseases, only 5 percent (or less) of rare diseases are thought to have approved treatment options, known as “orphan” therapies. Dr. Eagleton recently spoke on a webinar with his colleagues from Medpace about lessons learned from successful approaches from rare disease and gene therapy product approvals.

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XTalks

OCTOBER 20, 2023

Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.

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The Pharma Data

JUNE 7, 2023

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., a biotechnology company specializing in the development of lipid nanoparticle (LNP) delivery systems for molecular therapeutics.

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The Pharma Data

DECEMBER 13, 2020

which develops genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of diseases, today announced the appointment of Bo Zhang, Ph.D., as Head of Business Development. “Our 14, 2020 10:00 UTC. BEIJING & CAMBRIDGE, Mass.–( He received his B.S.

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Pharma Marketing Network

DECEMBER 21, 2020

Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e., those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies.

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Drug Discovery World

SEPTEMBER 14, 2023

Also in July 2023, Alexion, AstraZeneca Rare Disease (Alexion) and Pfizer entered into an agreement for Alexion to purchase and license the assets of Pfizer’s early-stage rare disease gene therapy portfolio for a total consideration of up to $1bn, plus tiered royalties on sales. billion, a decrease of $15.0 Vaccines were up 9.1%

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