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RARE Daily: the official Global Genes blog
Browse the latest news, articles, and blog posts from Global Genes.
Featured
Bringing Precision to CRISPR-Based Genome Editing
CRISPR genome editing has the potential to revolutionize the treatment of diseases, but the imprecision of its […]
Read moreCapturing Patients’ Experience in Their Daily Lives
One of the challenges with rare diseases is how different their effects can be from one person […]
Read moreRare Diseases May Be More Common than Thought
Rare Daily Staff People who have a single inherited defective gene from one parent have often been […]
Read moreFrom One to Many
When Terry Pirovolakis’ son Michael was diagnosed with the ultra-rare neurodegenerative disease spastic paraplegia type 50, he […]
Read moreDrug Development
FDA Grants Fast Track Designation to Arcturus’ mRNA Therapeutic Candidate for OTC
Rare Daily Staff The U.S. Food and Drug Administration has granted Fast Track designation to Arcturus Therapeutics’ […]
Read moreFDA Grants Fast Track Designation for Inhibrx Treatment of Alpha-1 Antitrypsin Deficiency
Rare Daily Staff The U.S. Food and Drug Administration granted Fast Track designation to Inhibrx’ INBRX-101, an […]
Read moreEMA Grants PRIME Designation to Rocket’s Gene Therapy for Danon Disease
Rare Daily Staff The European Medicines Agency granted Priority Medicines (PRIME) designation to Rocket Pharmaceutical’s RP-A501, the […]
Read moreBringing Precision to CRISPR-Based Genome Editing
CRISPR genome editing has the potential to revolutionize the treatment of diseases, but the imprecision of its […]
Read moreFDA Grants Priority Review to BMS for Treatment for Rare Form of NSCLC
Rare Daily Staff The U.S. Food and Drug Administration granted priority review to Bristol Myers Squibb’s repotrectinib, […]
Read morePfizer Reports Positive Marstacimab Results from Pivotal Phase 3 Hemophilia A and B Trial
Rare Daily Staff Pfizer reported that the pivotal phase 3 BASIS clinical trial evaluating marstacimab has met […]
Read moreFDA Places Clinical Hold on PepGen’s Application to Initiate a Phase 1 Study in DM1
Rare Daily Staff The U.S. Food and Drug Administration notified PepGen’s that it was placing a clinical […]
Read moreVerastem Reports Positive Results in Updated Phase 2 Results of Experimental Therapy in Rare Form of Ovarian Cancer
Rare Daily Staff Verastem Oncology reported updated data from Part A of the ongoing registration-directed RAMP 201 […]
Read moreEMA Recommends Revocation of Authorization for Sickle Cell Disease Medicine Adakveo
EMA Recommends Revocation of Authorization for Sickle Cell Disease Medicine Adakveo Rare Daily Staff The European Medicines […]
Read moreFinance
Belite Bio Raises $30 Million in Public Offering
Rare Daily Staff Belite Bio, a clinical stage company focused on advancing novel therapeutics targeting retinal degenerative […]
Read moreElevateBio Raises $401 Million to Further Accelerate Growth
Rare Daily Staff ElevateBio, a technology-driven company focused on powering the creation of life-transforming cell and gene […]
Read moreRenagade Therapeutics Launches with $300 Million to Expand Reach of RNA Therapies
Rare Daily Staff Renagade Therapeutics, which is seeking to expand the utility of RNA therapeutics by delivering […]
Read moreScarlet Therapeutics Raises Seed Financing to Advance Development of Therapeutic Red Blood Cell Platform
Rare Daily Staff Scarlet Therapeutics said it has raised seed funding from Science Creates Ventures and Meltwind […]
Read moreCabaletta Bio Raises $100 Million to Advance Cell Therapy Pipeline for Autoimmune Diseases
Rare Daily Staff Cabaletta Bio, a clinical-stage biotechnology company focused on developing and launching the first curative […]
Read moreProtalix BioTherapeutics Gets $20 Million Milestone Payment from Chiesi
Rare Daily Staff Protalix BioTherapeutics said it is eligible to receive $20 million from its commercial partner […]
Read moreApril a Quiet Month for Rare Disease Therapeutics Financing and Dealmaking
April was a quiet month for rare disease focused drug developers with few financings and no significant […]
Read moreX4 Pharma Raises $65 Million Private Placement to Advance Potential WHIM Treatment
Rare Daily Staff X4 Pharmaceuticals, a company focused on the discovery and development of novel small molecule […]
Read moreEnsoma Raises $50 Million to Advance Genomic Medicine
Rare Daily Staff Ensoma, a genomic medicines company developing in vivo treatments that precisely engineer any cell […]
Read moreMore Stories
Pharming Sells Priority Review Voucher for Pre-Set Price of $22.1 Million
Rare Daily Staff Pharming Group said it has entered into a definitive agreement with Novartis Pharma to […]
Read moreBioLight Enters Agreement with Alexion to Explore Technology To Diagnose Retinal Diseases
Rare Daily Staff Israeli ocular disease focused biotech BioLight signed a research collaboration agreement with Alexion, AstraZeneca […]
Read moreAbiogen Acquires Effrx
Rare Daily Staff Italy-based Abiogen Pharma, a company focused on osteoarticular and bone metabolism diseases, has acquired […]
Read moreIdogen Shareholders to Vote on Liquidation if Company Unable to Find Agreement on Reverse Merger
Rare Daily Staff The Swedish biotechnology company Idogen, which is developing cell therapies to counter attacks by […]
Read moreNovo Nordisk and Life Edit Enter Gene Editing Collaboration in Rare and Cardiometabolic Diseases
Rare Daily Staff Novo Nordisk and Life Edit Therapeutics, an ElevateBio subsidiary, have entered into a research […]
Read moreRare Diseases May Be More Common than Thought
Rare Daily Staff People who have a single inherited defective gene from one parent have often been […]
Read moreWhat’s Happening
The RARE Research Roadmap – 2023 RARE Drug Development Symposium
Read moreMental Health in Rare Disease: Taking Care of Your Mind Is Taking Care of Your Body
May is Mental Health Awareness Month. Taking care of your mental health is key, whether you are […]
Read moreHow Can the FDA Improve Processes for Rare Diseases? – 2023 RARE Drug Development Symposium
Read moreThe RARE Research Roadmap – 2023 RARE Drug Development Symposium
Read moreThe Regulatory Landscape: Pathways, End Points, and Clinical Trials – 2023 RARE Drug Development Symposium
Read moreIntellectual Property: Balancing Stakeholders’ Rights – 2023 RARE Drug Development Symposium
Read moreNew Technologies in Early Stage Research – 2023 RARE Drug Development Symposium
Read moreUnderstanding Data for Basic Research – 2023 RARE Drug Development Symposium
Read moreCharting the Path to Treatments – 2023 RARE Drug Development Symposium
Read moreReports
Global Genes 2022 RARE Impact Grants Report
Over the past decade, Global Genes’ RARE Impact Grant Program has provided funding opportunities for rare patient […]
Read moreGlobal Genes Impact Report: Building on a Successful 2022
To our community members: The year 2022 was a significant one for the rare disease community as […]
Read moreRARE-X Insights & Recommendations for Organizational DEI Strategies Study
Ensuring diversity, equity, and inclusion in rare disease organizations requires focused programs that engage all stakeholders. A […]
Read moreMore Resources: Dictionary of Rare Diseases
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