News

Ionis, AstraZeneca Team Up to Further Develop Eplontersen

Ionis Pharmaceuticals has entered into a collaboration agreement with AstraZeneca to develop and commercialize eplontersen, Ionis’ investigational therapy to treat transthyretin amyloidosis (ATTR). ATTR amyloidosis is a group of conditions characterized by the formation of toxic aggregates or clumps of the TTR protein that build up in…

Heart Disease Can Be Prevalent in Men With ATTRv Amyloidosis

Heart involvement is more prevalent among men with hereditary transthyretin (ATTRv) amyloidosis, a group of rare disorders that includes familial amyloid polyneuropathy (FAP), a study reports. Findings suggest that female sex may be protective against heart disease in ATTRv, and that being male may also contribute to disease worsening. The…

NTLA-2001 Gene Editing Trial to Include Patients With Heart Disease

A clinical trial of the gene-editing therapy NTLA-2001 in people with familial amyloid polyneuropathy (FAP) is expanding to include those with a related condition, called ATTR amyloidosis with cardiomyopathy (ATTR-CM), Intellia Therapeutics, which is developing the therapy, reported. FAP is a form of ATTR amyloidosis — a group of disorders…

Protego Raises $51M to Support Its Protein-misfolding Therapies

Protego Biopharma has raised $51 million to support the development of therapies for diseases caused by protein misfolding, such as familial amyloid polyneuropathy (FAP). The series A financing was co-led by Lightspeed Venture Partners, Vida Ventures, and MPM Capital. As part of the financing, representatives from these three…

Gene-editing Therapy NTLA-2001 Given Orphan Drug Status by FDA

Note: This story was updated Nov. 24, 2021 to clarify that NTLA-2001 works by disrupting the TTR gene to reduce transthyretin levels.   The U.S. Food and Drug Administration (FDA) has given orphan drug designation to NTLA-2001, an investigational gene-editing therapy for familial amyloid polyneuropathy (FAP) and other forms of…

Global Genes, Diversity Coalition Team Up to Advance Health Equity

Global Genes has partnered with the Rare Disease Diversity Coalition (RDDC) to advance health equity for rare disease patients and caregivers in underrepresented communities of color. “For rare disease patients, there are many challenges — and for people of color with a rare disease, these challenges are compounded…

Tegsedi Gets Innovative Medication Category Pricing in Brazil

Tegsedi (inotersen), an approved treatment for familial amyloid polyneuropathy (FAP), has received a Category 1 classification by the legal body tasked with economic regulation of medications in Brazil. Category 1 is given by Câmara de Regulação do Mercado de Medicamentos to innovative treatments that are more effective than…