Rare Diseases

CEO Djordje Filipovic tells Scrip that after talks with "multiple other potential partners,” the firm chose the Japanese group for its expertise and marketing capabilities in rare diseases” to be the licensing partner for tadekinig alfa in the US.

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.

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

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.

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.

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.

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.

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.

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).

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.