Clinical Development Disease Science 
Drug Target Review
APRIL 7, 2025
As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. 2 This progress has implications for diagnosis, therapeutic efficacy, and potentially establishing clinically relevant endpoints.
Drug Target Review
JANUARY 8, 2024
It is no mystery that as we age our health starts to deteriorate, and we become increasingly susceptible to diseases. Through years of scientific exploration and research, we now know there are several biological changes that make our bodies more susceptible to disease or injury, and we can target those with therapeutic interventions.
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PPD
JANUARY 15, 2025
As clinical trials become increasingly complex, particularly in decentralized trials and rare disease studies, sponsors experience increased challenges in site selection, forecasting and resourcing, and patient recruitment and enrollment.
KIF1A
SEPTEMBER 30, 2023
Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. KIF1A-Related Research In the rare disease space, a common and straightforward question is: How rare is the disease?
The Pharma Data
NOVEMBER 29, 2020
Prior to joining Gannex, Dr. Palmer was Head of Liver Disease Clinical Development at Takeda Pharmaceuticals. She has also held senior leadership positions at Shire Pharmaceuticals and Kadmon Corporation, where she was in charge of the global development of NASH and other liver disease programs.
Drug Target Review
MARCH 17, 2025
He has contributed to more than 130 granted patents and advanced over 12 clinical drug candidates across various therapeutic areas, including cognition, depression, pain and other disorders. Now in Phase II development at Gate for depression and other synaptopathies, zelquistinel represents a key part of his current work.
LifeSciVC
JULY 17, 2024
By Mike Cloonan, Chief Executive Officer of Sionna Therapeutics, as part of the From The Trenches feature of LifeSciVC The drug development process in rare diseases is rife with challenges especially when companies target significant differentiation or first-in-class targets.
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