rare disease
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Another Intellia CRISPR therapy flashes potential for one-time, in vivo treatment
Intellia Therapeutics has encouraging early clinical data for a gene-editing therapy addressing the rare swelling disorder hereditary angioedema. This therapy, the biotech’s second CRISPR-editing based treatment that performs in vivo edits, offers potential for a one-time treatment of a disease that is currently addressed by chronic oral and injectable drugs.
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After RNA therapy falls short in the eye, ProQR Therapeutics pivots to liver, CNS
RNA therapies developer ProQR Therapeutics is eying a future without ophthalmology. After receiving European Medicines Agency feedback for its lead program for a rare eye disorder, the biotech has decided to find a strategic partner for all of its eye drug assets and apply its RNA technology to other therapeutic areas.
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Payer’s Place: Dawn Maroney
Dawn Maroney, President, Markets of Alignment Health and CEO of Alignment Health Plan, to discuss how they are using technology to provide better service and care to consumers.
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Disappointing data lead Pfizer to drop heart drug acquired as part of $11B deal
Pfizer is stopping work on a drug for a rare heart disease after an interim look at Phase 3 data indicated that the study was unlikely to succeed. The small molecule came to Pfizer’s drug pipeline as part of the $11.4 billion acquisition of Array Biopharma in 2019.
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Alnylam drug succeeds in heart study, setting stage for FDA filing and showdown with Pfizer
Alnylam Pharmaceuticals drug Onpattro, an FDA-approved treatment for nerve pain caused by hereditary transthyretin amyloidosis, now has Phase 3 data showing it can also help the much larger group of patients suffering heart problems from the rare protein disease. An FDA submission is planned, which would pit the Alnylam drug in competition with a blockbuster Pfizer product.
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Aiming to catch Alnylam, AstraZeneca & Ionis plan FDA filing for rare disease drug
The planned FDA submission follows the report from AstraZeneca and Ionis Pharmaceuticals that their partnered drug, eplontersen, met the main goals of a pivotal study in treating nerve pain caused by hereditary transthyretin-mediated amyloidosis. The data come one week after rival Alnylam Pharmaceuticals won FDA approval for its second drug for this rare disease.
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BrightInsight launches new digital disease management solution for biopharma and medtech
BrightInsight developed the Disease Management Solution to help biopharma and medtech companies bring their Software as a Medical Device (SaMD) to market faster, according to BrightInsight CEO and Co-founder Dr. Kal Patel.
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AllStripes raises $50 million to support rare disease research
The startup, which is focused on letting patients share their health records for rare disease research, plans to use the funds to launch 100 rare disease programs.
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Hospitals, Events, Health Tech
As children’s hospitals build up precision medicine programs, families still face challenges in reaching a diagnosis
Children’s hospitals are launching big efforts to identify more rare diseases through genome sequencing. Even as new treatments become available, many families struggle to reach a diagnosis.
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INVEST Precision Medicine conference kicks off today. Register now to be part of the conversation.
Register for the INVEST Precision Medicine conference, which kicks off today. Over the next three days industry experts will talk about precision medicine developments across pediatrics, bioinformatics, startups and more.
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Applying Remote Patient Monitoring to Surgery Prep and Recovery, Oncology and Women’s Health
Join us to learn about the latest trends in remote monitoring and how to extend its benefits beyond chronic conditions to more patients – all while using fewer staff resources.
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How biotech startup Ensoma aims to take gene therapy beyond rare diseases
Ensoma contends its technology avoids the complexities of other approaches to gene therapy, which could expand genomic medicine’s reach to more patients and more diseases. Takeda Pharmaceutical is collaborating with the startup on up to five gene therapies.
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Amicus’s path to unseat Sanofi in rare disease gets steeper after Phase 3 miss
Amicus Therapeutics CEO John Crowley said the totality of data support the Pompe disease therapy’s submission to the FDA. That rolling application is on track for completion in the second quarter.
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FDA approves Regeneron drug for rare, inherited form of high cholesterol
The FDA approval clears Regeneron’s drug, Evkeeza, as a treatment for the rarer of two forms of an inherited disease that causes high cholesterol levels. The company already markets Praluent, which treats the more common form of familial hypercholesterolemia.
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KalVista Pharma plans pivotal test after HAE drug posts positive Phase 2 data
One year after losing Merck as a partner, KalVista reports mid-stage clinical data for a pill that could offer an alternative to injectable drugs for a rare, potentially life-threatening disease. KalVista plans to meet with the FDA about proceeding to a Phase 3 study.
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MedCity Influencers, BioPharma, Diagnostics
How augmented intelligence and NLP can help clinicians, researchers identify rare diseases
To help clinicians diagnose rare disease more quickly and accurately, many healthcare organizations are embracing technology solutions like natural language processing (NLP) tools that can create augmented intelligence workflows that facilitate the rapid search of unstructured clinical data from multiple data sources.
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Regeneron scores Phase III win with drug for rare cholesterol disorder
An analyst wrote that while the data are positive, the bigger question is how the drug will be priced, based on another drug already on the market for the same disease, HoFH, which affects about 1,300 people in the U.S.
Promoted
Payer’s Place: Dr. Anil Singh
Dr. Anil Singh shares his insights into the strategies employed by the organization to identify the most effective digital solutions for their members.
Promoted
Discover the Next-Gen Platform for Integrated Collaborative Care
Beyond EHRs and digital front doors, reducing the gaps in patient care journeys.
