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RARE Daily: the official Global Genes blog
Browse the latest news, articles, and blog posts from Global Genes.
Featured
Addressing a Shortage of Genetic Counselors with AI
As genetic testing continues to expand, it is bringing into focus a shortage of genetic counselors who […]
Read moreA 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 moreDrug Development
New BioMarin CEO Prioritizes Three Programs, Axes Four Others
Rare Daily Staff BioMarin Pharmaceutical said that after a strategic R&D asset review to maximize profitability, it […]
Read moreFDA Approves Pfizer’s Hemophilia B Gene Therapy
Rare Daily Staff The U.S. Food and Drug Administration approved Pfizer’s Beqvez, a gene therapy for the […]
Read moreModerna and OpenAI Collaborate to Advance mRNA Medicine
Rare Daily Staff Moderna and OpenAI announced an ongoing collaboration, which will integrate generative AI throughout the […]
Read moreFDA Grants Accelerated Approval to Day One’s Ojemda for Childhood Brain Tumor
Rare Daily Staff The U.S. Food and Drug Administration has granted accelerated approval to Day One Biopharmaceuticals’ […]
Read moreProfluent Releases AI-Created and Open-Source Gene Editor
Rare Daily Staff Profluent said it demonstrated the first successful precision editing of the human genome with […]
Read moreEC Grants Accelerated Approval to Travere and CSL Vifor’s Filspari for Rare Kidney Disease
Rare Daily Staff The European Commission granted accelerated approval to Travere Therapeutics and CSL Vifor’s Filspari for […]
Read moreFDA Approves Novartis’ Lutathera for Children with Rare Neuroendocrine Tumors
Rare Daily Staff The U.S. Food and Drug Administration approved Novartis’ radioligand therapy Lutathera for the treatment […]
Read moreFDA 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 moreFinance
Endeavor Bio Raises $132.5 Million to Advance Pipeline
Rare Daily Staff Endeavor BioMedicines raised $132.5 million in a series C financing, including the conversion of […]
Read moreSeamless 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 moreMore Stories
Regeneron and Mammoth Collaborate on Next-Gen CRISPR Gene Editing for Multiple Diseases
Rare Daily Staff Regeneron Pharmaceuticals and Mammoth Biosciences entered a collaboration to research, develop and commercialize in […]
Read moreRett Syndrome Research Trust Partners with Vivalink to Accelerate Research
Rare Daily Staff Digital health technology provider Vivalink entered into a collaboration with the Rett Syndrome Research […]
Read moreLabcorp to Acquire Invitae Assets
Rare Daily Staff Laboratory services company Labcorp said it’s $239 million bid for assets of the medical […]
Read morePerrigo Receives Offer to Divest its Rare Diseases Business for up to $295 Million
Rare Daily Staff Perrigo Company, a provider of consumer self-care products, said that Spanish pharmaceutical company Esteve […]
Read moreLifeArc 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 moreWhat’s Happening
Shundra Wooten on her short film “Keep It Moving”
Shundra Wooten was diagnosed with Spinocerebellar Ataxia 3, and made a short film “Keep It Moving” to […]
Read moreProfluent Releases AI-Created and Open-Source Gene Editor & More — This Week in RARE Daily
This Week in RARE Daily is a feature from Global Genes where you can get a quick […]
Read moreShantel Sonier on “Glitching with Nick”
Shantel Sonier, a rare mom and caregiver to Nick, who was diagnosed with GRIN2A, created the short […]
Read moreLesley Holroyd on her short film “Born to be Heard”
Lesley Holroyd, who lives with congenital adrenal hyperplasia, discusses what she learned about filmmaking and telling her […]
Read more“Miss Diagnosis” breaking the rules with Tara Rule
Daniel DeFabio, Director of Community Engagement for Global Genes, spoke to Tara Rule about what rules should […]
Read moreNational DNA Day: Cracking the Code on Rare Diseases and Unlocking Hope
by Shruti Mitkus, Ph.D. National DNA Day is celebrated each year on April 25th to commemorate the […]
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 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
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