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.
PPD
JUNE 20, 2025
Patients diagnosed with idiopathic pulmonary fibrosis (IPF) — a progressive, rare lung disease marked by scarring and worsening lung function — often face a poor prognosis. While two approved treatments, pirfenidone and nintedanib, slow disease progression, there currently is no treatment that reverses the effects or offers a cure for IPF.
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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.
Drug Target Review
JULY 1, 2025
Clinical trials are expensive, slow and often limited by outdated design constraints. Placebo arms, in particular, create ethical and logistical hurdles, especially in areas like rare disease and oncology. They offer patient-specific outcome predictions, generated using machine learning models trained on real historical clinical data.
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.
The Pharma Data
JUNE 25, 2025
Novartis Finalizes Acquisition of Regulus Therapeutics, Strengthening Its Renal Disease Portfolio with Promising ADPKD Therapy Novartis AG, a leading global pharmaceutical company, has officially completed its acquisition of Regulus Therapeutics Inc. , a biotechnology firm known for its expertise in microRNA-targeting therapies.
The Pharma Data
JUNE 18, 2025
AMVUTTRA’s approval has not only underscored the therapeutic potential of RNAi-based medicines but also signaled Alnylam’s readiness to scale its capabilities and deepen its impact across a wider range of disease areas. As we begin this exciting new chapter, I am energized about what lies ahead for Alnylam R&D and for patients,” he said.
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