Patient registries are a hot topic of rare disease research and many organizations are taking advantage of this resource by signing up their patient communities and connecting with researchers. Eric Sid, MD, program officer for the Office of Rare Diseases Research (ORDR), said it is difficult to estimate how…
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Problems in the metabolism of low-density lipoprotein (LDL) — also known as “bad cholesterol” — and its receptor contribute to changes in lung cells that ultimately lead to pulmonary fibrosis (PF), an analysis of fibrotic lung tissue and cell and animal models suggests. Notably, a combination of two approved…
Eperisone — an oral muscle relaxant used to treat muscle spasms and rigidity — significantly eased lung scarring and improved lung function in a mouse model of idiopathic pulmonary fibrosis (IPF), a study shows. Comparisons between eperisone and the approved IPF therapies Ofev (nintedanib) and Esbriet (pirfenidone) in…
OncoArendi Therapeutics and the University of Michigan (U-M) have signed an option-to-license agreement, allowing the Poland-based biotech company to develop new therapies to treat fibrotic diseases, including idiopathic pulmonary fibrosis (IPF). “We are thrilled to be partnering with the University of Michigan, one of the world’s leading research…
RXC007, an experimental oral treatment of idiopathic pulmonary fibrosis (IPF) being developed by Redx Pharma, continues to be safe and well-tolerated in a Phase 1 study in healthy volunteers, the company reported. “Clinical data from our Phase 1 study suggests that RXC007 is safe and well-tolerated at the drug doses…
Bridge Biotherapeutics has entered into an exclusive option-to-license agreement with Cellion BioMed to develop BBT-301, an experimental ion modulator therapy, as a potential treatment of idiopathic pulmonary fibrosis (IPF) and other fibrotic diseases. “We are highly excited to in-license BBT-301, a novel drug candidate for the treatment of fibrotic…
Horizon Therapeutics has launched its #RAREis Representation program aimed at increasing diversity, equity, and inclusion among patients with rare diseases. There are about 400 million people worldwide living with a rare disease; for many of them, access to diagnosis, care, and treatments can be challenging. Accessing better care depends on…
The China National Intellectual Property Administration has granted AdAlta a patent covering AD-214, the biotech company’s experimental therapy for idiopathic pulmonary fibrosis (IPF). The patent — number CN 107427574 B — entitled “CXCR4 binding molecules,” will expire in early January 2036. “China is now the second largest (and…
Dosing has begun in healthy volunteers in a Phase 1 clinical trial of ISM001-055, a small molecule potential treatment of idiopathic pulmonary fibrosis (IPF) developed using an artificial intelligence (AI) platform, Insilico Medicine announced. Up to 80 adults in this study (NCT05154240), currently enrolling at its one…
Nearly half of the people with pulmonary fibrosis (PF) responding to a survey were dissatisfied with their quality of life, while more than a third felt pessimist about their future. The U.S.-based survey was conducted by Pulmonary Fibrosis News. Nevertheless, most respondents said they were satisfied with their health…
