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RARE Daily: the official Global Genes blog
Browse the latest news, articles, and blog posts from Global Genes.
Featured
A Small Molecule Therapy to Regenerate Muscle in People with DMD
Duchenne muscular dystrophy is an inherited disease caused by genetic mutations that no longer allow the dystrophin […]
Read moreForging a Faster Path for Gene Therapies
The Bespoke Gene Therapy Consortium, a public-private partnership backed by the Foundation for the National Institutes of […]
Read moreDetermining the Value of Rare Disease Therapies
The small patient populations of rare diseases, the limited natural history of these conditions, and the lack […]
Read moreFinancings of Public Rare Disease Drug Developers Soar in First Quarter 2024
Companies developing drugs for rare diseases raised substantial new capital so far this year, as rare disease […]
Read moreDrug Development
FDA Tells Abeona It Won’t Approve RDEB Cell Therapy without Additional CMC Information
Rare Daily Staff The U.S. Food and Drug Administration told Abeona Therapeutics that it would not approve […]
Read moreEU Approves AstraZeneca’s Voydeya for PNH as Add-on
Rare Daily Staff The European Union approved AstraZeneca’s Voydeya as an add-on to Ultomiris and Soliris for […]
Read moreSanofi Reports Experimental Therapy for Blood Disorder Met Phase 3 Endpoint
Rare Daily Staff Sanofi said its experimental therapy rilzabrutinib met its primary endpoint of durable platelet response […]
Read moreFDA Grants Breakthrough Therapy Designation for Kura’s Treatment for NPM1-Mutant AML
Rare Daily Staff The U.S. Food and Drug Administration granted Breakthrough Therapy designation for Kura Oncology’s ziftomenib […]
Read moreRNA-Targeted Therapy Shows Promise for a Rare Childhood Dementia
Rare Daily Staff A study in mice suggests a new RNA-targeted therapy can halt the progression of […]
Read moreArgenx Highlights gMG Drug Data in CIDP
Rare Daily Staff Argenx said that positive data from its phase 3 ADHERE trial evaluating Vyvgart Hytrulo […]
Read moreFDA Grants Fast Track Designation for Lexeo’s Gene Therapy for Friedreich’s Ataxia Cardiomyopathy
Rare Daily Staff The U.S. Food and Drug Administration has granted Fast Track designation to Lexeo Therapeutics’ […]
Read moreA 37-Year Path to a Diagnose, but Now a Drug May Be Two Weeks Away
It took Courtney Ampezzan 37 years to get a correct diagnosis for her condition, an unusual length […]
Read moreUltragenyx Reports Positive Interim Phase 1/2 Data in Patients with Angelman Syndrome
Rare Daily Staff Ultragenyx Pharmaceutical reported positive new data from the phase 1/2 study of GTX-102 for […]
Read moreFinance
Seamless Raises $25 Million in Seed Financing, Expands to U.S. to Advance Programmable Gene Editing Platform
Rare Daily Staff German biotech Seamless Therapeutics closed a $25 million in seed financing to support the […]
Read moreSynOx Raises $75 Million to Advance Development of Potential Treatment for TGCT
Rare Daily Staff Irish biotech SynOx Therapeutics raised $75 million in a series B financing to advance […]
Read moreKorro Bio Raises $70 Million to Advance RNA Edited Therapies
Rare Daily Staff Korro Bio, which is developing a new class of genetic medicines based on editing […]
Read moreTwo Rare Disease Drug Developers Raise Combined $152.5 Million in PIPE Offerings
Rare Daily Staff Two companies developing therapeutics for rare diseases, Zura Bio and Benitec Biopharma, raised $152.5 […]
Read moreJ&J Enter Collaborations with Rallybio, Makes Equity Investment
Rallybio said it has entered into a collaboration with Johnson & Johnson to support the development of […]
Read moreZevra Refinances Existing Debt with Up to $100 Million Under New Credit Facility
Rare Daily Staff Rare diseases therapeutics company Zevra Therapeutics said it has entered into a new credit […]
Read moreNvelop Launches with Dual Platforms for In Vivo Delivery of NextGen Genetic Medicines
Rare Daily Staff Nvelop Therapeutics formally announced its launch with $100 million in funding to develop programmable, […]
Read moreFinancings of Public Rare Disease Drug Developers Soar in First Quarter 2024
Companies developing drugs for rare diseases raised substantial new capital so far this year, as rare disease […]
Read moreAeovian Raises $50 Million, Doses First Cohort in Phase 1 TSC Study
Rare Daily Staff Aeovian Pharmaceuticals, a clinical-stage company developing therapeutics that potently inhibit the mTORC1 pathway, completed […]
Read moreMore Stories
LifeArc Invests $50 Million to Launch Rare Disease Research Centers
Rare Daily Staff The UK charity LifeArc is investing $50 million (£40 million) to launch four new […]
Read moreGeneDx Partners with Komodo Health to Expand Access to World’s Largest Rare Disease Dataset
Rare Daily Staff GeneDx and Komodo Health have entered into a strategic partnership to increase access to […]
Read moreIpsen Enters Collaboration with Skyhawk for RNA-Targeted Therapies for Neurological Diseases
Rare Daily Staff Ipsen said it entered into an exclusive, worldwide collaboration to discover and develop small […]
Read moreLexeo Enters Data Collaboration with Cornell to Accelerate Development of Gene Therapy for FA Cardiomyopathy
Rare Daily Staff Lexeo Therapeutics entered an in-license agreement with Cornell University to expedite development of the […]
Read moreExpanding Access to Genome Sequencing in Rural Populations
Rare Daily Staff Patients in rural area often face challenges accessing cutting-edge medical technologies, such as state-of-the-art […]
Read moreGroup Calls for Use of Genome Sequencing as First Line Test for Patients Suspected of Genetic Disease
Rare Daily Staff The Medical Genome Initiative, a group made up of sequencing giant Illumina and leading […]
Read moreWhat’s Happening
National DNA Day: Cracking the Code on Rare Diseases and Unlocking Hope
National DNA Day is celebrated each year on April 25th to commemorate the discovery of DNA’s double […]
Read moreExpanding Access to Genome Sequencing in Rural Populations & More — This Week in RARE Daily
This Week in RARE Daily is a feature from Global Genes where you can get a quick […]
Read moreFinancings of Public Rare Disease Drug Developers Soar & More — This Week in RARE Daily
This Week in RARE Daily is a feature from Global Genes where you can get a quick […]
Read morePromise and Challenges of Gene-Editing and Other Genomic Medicines — 2024 NEXT Report
When scientists completed the Human Genome Project in 2003, it provided, among other things, a reference genome […]
Read moreNCATS Seeks Applicants for Rare Disease Clinical Research Funding & More — This Week in RARE Daily
This Week in RARE Daily is a feature from Global Genes where you can get a quick […]
Read moreThe Impact of Grants Provided For Ukraine Relief
Healthcare Education Institute was one of six organizations providing aid and support to those affected by the […]
Read moreMental Health Resources for the Rare Disease Community
Those in the rare disease community may experience a variety of mental health issues, ranging from anxiety […]
Read moreCelebrating the Most Expensive Drug for the Disease You Never Heard Of & More — This Week in RARE Daily
This Week in RARE Daily is a feature from Global Genes where you can get a quick […]
Read moreThe Vital Role of Education in Patient Advocacy: A Gene Therapy Perspective
Gene therapy promises to revolutionize medicine, particularly for rare diseases, approximately 80% of which are genetic in […]
Read moreReports
Global Genes 2023 RARE Impact Grants Report
Over the past decade, Global Genes’ RARE Impact Grant Program has provided grants to rare disease patient […]
Read moreNEXT Report 2024: Rewriting the Rules
Over the past year, technological advances in rare disease drug and therapy development, coupled with the tenacity of rare disease patients and advocates, have prevailed despite the challenges of financial difficulties in biopharma. Next-generation patient advocates continue to take an active role in drug development, as outlined in the 2024 NEXT Report.
Read moreGlobal Genes 2022 RARE Impact Grants Report
Over the past decade, Global Genes’ RARE Impact Grant Program has provided funding opportunities for rare patient […]
Read moreMore Resources: Dictionary of Rare Diseases
Search for a specific disease in our extensive list of rare diseases for disease-specific information, news, and resources.
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