ADVERTISEMENT

Rare Diseases

Sangamo Nearing Fabry Gene Therapy Deal To Fund Its Pipeline

Low on cash when Pfizer handed back rights to its hemophilia A gene therapy, Sangamo is talking to partners for its Fabry disease gene therapy to fund its two lead programs through proof-of-concept.

Deals In Depth: December 2024

Nine $1bn+ alliances were penned in December, and two exceeded $2bn.

Teva Leapfrogs One ANDA Sponsor But Faces Decade Wait On US Firdapse Opportunity

Catalyst saw its share price jump by around 14% after striking what the market termed a favorable patent-litigation settlement agreement with ANDA sponsor Teva over its rare disease drug Firdapse.

Innovation And Accessibility: India’s Advanced Therapy Pricing Challenge

As India prepares to commercialize its first advanced therapies it must engage key stakeholders to ensure successful implementation. Learning from global best practices and managing pricing strategies will be crucial to maximize access, deliver high-quality outcomes, and sustain innovation through reinvestment in R&D.

EU CHMP Opinions And MAA Updates

This is an update of recommendations from the European Medicines Agency's Committee for Medicinal Products for Human Use on the authorization of new medicines in the EU, and updates on EU marketing authorization changes recommended by the CHMP.

Data: The Clinical Trials Space Rebounds

The complex landscape of clinical trials, fraught with pressures and risks, and exacerbated by the adoption of new technologies, regulatory burdens, and drug pricing negotiations, is rebounding with aplomb.

Circio’s Vision For Long-Lasting Nucleic Acid Therapeutics

Circular RNA has several advantages, but the field is young. Scandinavian startup Circio Holdings believes its version of the technology will prove the most robust.

ASH: Sickle Cell Treatment Decisions In The Post-Oxbryta, Genetic Medicine Era

Hematologists discussed the still limited treatment options for sickle cell disease at ASH, weighing the risks and benefits of disease-modifying versus curative therapies after the withdrawal of Pfizer’s Oxbryta (voxelotor).

Podcast: Treating Autoimmune Neuropsychiatric Diseases

In this episode of the In Vivo podcast, Peter Flynn, CEO of Arialys Therapeutics, discusses the exciting field of autoimmune neuropsychiatry and the company’s monoclonal antibody, which it is investigating for the treatment of a rare condition called anti-NMDA receptor encephalitis and potentially other indications.

Outlook 2025: Will Geopolitics Dampen Pharma’s Comeback Party?

The financial macroenvironment may be looking more favorable for 2025 but will pharma growth be muted by elements beyond its control? In Vivo discusses the headwinds and drivers for pharma in the coming 12 months.