Category: Rare Diseases
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Arkin Bio Ventures III: $100 million fund for transformative therapies
Fund lead, Dr. Pini Orbach, talks about the fund targeting oncology, immunology, inflammation, rare diseases, and their aim to be an active partner from day one.
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Genesipre’s rare disease therapy gets orphan drug status in the US and Europe
The gene therapy is being developed to treat a rare pediatric metabolic disease, methylmalonic acidemia (MMA).
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Sanofi’s ASH 2025: Redefining rare blood disorder care
Sanofi’s head of rare diseases, Jeff Schaffnit, outlines the company’s dual approach: redefining hemophilia care and pioneering immune modulation for rare blood disorders.
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Chiesi licenses AbbVie unit’s brain-targeting tech for rare diseases
The partnership with Aliada Therapeutics centers on a platform technology designed to ferry large-molecule therapies across the protective blood-brain barrier (BBB) for lysosomal storage disorders (LSDs).
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AAVantgarde closes $141 million Series B for genetic disease therapies
The company plans to use the Series B funding to progress clinical trials for two genetic eye disorders, Stargardt disease and Usher 1B syndrome.
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BOOST Pharma raises $3.5 million for rare disease stem cell therapy
The Danish company plans to use the funds to advance its stem cell therapy, BT-101, for osteogenesis imperfecta, also known as brittle bone disease.
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ERS 2025: Boehringer shares additional data for lung fibrosis therapy
Pooled Phase III data show nerandomilast may reduce death risk in idiopathic pulmonary fibrosis (IPF) and progressive pulmonary fibrosis (PPF).
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PureTech’s deupirfenidone: Overcoming the efficacy paradigm in IPF
PureTech’s Camilla Graham talks about the IPF landscape, barriers to antifibrotic use, and the development of deupirfenidone.
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Chiesi scores FDA priority review for rare vision disorder
Raxone is approved for Leber Hereditary Optic Neuropathy (LHON), a rapid vision-loss disease, in multiple countries, including the EU.
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UK NICE recommends Chiesi’s rare eye therapy
The final draft guidance supports Chiesi’s Raxone for patients aged 12 and above with Leber Hereditary Optic Neuropathy (LHON).
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Fresh off merger, Minovia scores twin FDA designations for lead therapy
Minovia’s MNV-201 has scored US FDA Fast Track and Rare Pediatric Disease designations for treating Pearson Syndrome.
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Rare Disease Day: Importance of using ‘authentic’ imagery in disease materials
The Chiesi UK team discusses the importance of authentic and accurate representation in media and imagery for people with rare diseases.
Latest posts
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ASH25: Gallop’s LYT-200 shows unprecedented survival in AML
The company’s execs talk about the striking efficacy and safety signals seen in Phase I data, the science behind the molecule, and their vision for transforming AML therapy.
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Novo Nordisk’s denecimig cuts bleeding in hemophilia A regardless of inhibitors
Phase III trial shows once‑monthly and once‑weekly denecimig (Mim8) significantly reduces annualized bleeding rate versus standard care, with up to 95% of patients bleed‑free.
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Beyond the Snapshot: Continuous physiologic monitoring in clinical trials
Vivalink’s CEO discusses how continuous physiologic monitoring is transitioning from a niche tool for early-stage feasibility studies to a core component of late-stage drug development.