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RARE Daily: the official Global Genes blog
Browse the latest news, articles, and blog posts from Global Genes.
Featured
A Gene Therapy Developer that Embraces Different Models for Reaching Patients
The work of gene therapy pioneer Genethon, a non-profit organization created by the patient association AFM-Telethon, has […]
Read moreRare Disease Focused Therapeutics M&A Soars in April
Two sizable acquisitions marked an otherwise slow April for rare disease focused therapeutics financings and deals, with […]
Read moreUsing Directed Evolution to Develop New Vectors for Genetic Medicines
Much of the challenge of developing genetic medicines lies in having the right vector to deliver the […]
Read moreAddressing 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 moreDrug Development
Regeneron’s Experimental Gene Therapy Restores Hearing to Normal Level in Deaf Child
Rare Daily Staff Regeneron Pharmaceuticals said its experimental gene therapy DB-OTO improved hearing to normal levels within […]
Read moreFDA Grants Breakthrough Therapy Designation for Avidity’s Treatment for Myotonic Dystrophy Type 1
Rare Daily Staff The U.S. Food and Drug Administration granted Breakthrough Therapy designation to Avidity Biosciences delpacibart […]
Read morePfizer Pauses Enrollment in DMD Gene Therapy Study After Patient Death
Rare Daily Staff Pfizer, in a May 7 letter to Duchenne muscular dystrophy patient advocacy organizations, reported that […]
Read moreStealth Biotherapeutics’ Barth Syndrome NDA Receives FDA Priority Review
Rare Daily Staff The U.S. Food and Drug Administration has granted Priority Review to Stealth Biotherapeutics’ New […]
Read moreFDA Grants Fast Track Designation to Iecure for Treatment of Neonatal OTC Deficiency
Rare Daily Staff The U.S. Food and Drug Administration granted Fast Track Designation to Iecure ECUR-506, the […]
Read moreGlycoMimetics’ Uproleselan Fails Pivotal Phase 3 Study in Relapsed/Refractory AML
Rare Daily Staff GlycoMimetics said the phase 3 global pivotal study of its experimental treatment for relapsed/refractory […]
Read moreAstraZeneca Drug Regimen Improves PFS in First-Line Rare Blood Cancer
Rare Daily Staff Positive high-level results from an interim analysis of the ECHO phase 3 trial showed […]
Read moreFDA Grants Fast Track to Amolyt’s Experimental Hypoparathyroidism Candidate
Rare Daily Staff The U.S. Food and Drug Administration granted Fast Track designation to Amolyt Pharma’s lead […]
Read moreAtsena Reports Positive Data from First Cohort of Trial Evaluating Gene Therapy for XLRS
Rare Daily Staff Atsena Therapeutics reported positive preliminary data from the first cohort of the ongoing LIGHTHOUSE […]
Read moreFinance
Aardvark Therapeutics Raises $85 Million to Advance Experimental Prader-Willi Therapeutic
Rare Daily Staff Aardvark Therapeutics said it raised $85 million in an oversubscribed series C financing to […]
Read moreEledon Raises $50 Million in Private Placement
Rare Daily Staff Eledon Pharmaceuticals entered into a securities purchase agreement with certain institutional and accredited healthcare […]
Read moreZenas Raises $200 Million to Advance Immunology-Focused Clinical Programs
Rare Daily Staff Zenas BioPharma, a company developing and commercializing inflammation and immunology-directed therapies for rare and […]
Read moreRare Disease Focused Therapeutics M&A Soars in April
Two sizable acquisitions marked an otherwise slow April for rare disease focused therapeutics financings and deals, with […]
Read moreSoleno Raises $138 Million in Public Offering to Advance Treatment for Prader-Willi
Rare Daily Staff Rare disease focused biotech Soleno Therapeutics raised $138 million in an underwritten public offering […]
Read moreLatus Bio Launches with $54 Million to Deliver Gene Therapies Directly to the Brain
Rare Daily Staff Latus Bio, a company developing improved gene therapies that target central nervous system disorders, […]
Read moreMustang Bio Raises $4 Million in Public Offering
Rare Daily Staff Mustang Bio, a company focused on cell and gene therapies for difficult-to-treat cancers and […]
Read moreEndeavor 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 moreMore Stories
PPMD Says Decision on Newborn Screening for DMD Delayed
Rare Daily Staff The Advisory Committee on Heritable Disorders in Newborns and Children unanimously voted to postpone […]
Read moreMaze Teams with Shionogi to Develop Its Investigational Treatment for Pompe Disease
Rare Daily Staff Maze Therapeutics entered into an exclusive worldwide license deal with Japanese pharmaceutical Shionigi for […]
Read moreMarinus Cuts 20 percent of Its Staff
Rare Daily Staff Marinus Pharmaceuticals said it was laying off 20 percent of its staff as part […]
Read moreArbor Acquires Gene Editing Biotech Serendipity
Rare Daily Staff Genetic medicines biotech Arbor Biotechnologies acquired gene-editing biotech Serendipity Biosciences, bolstering Arbor’s toolbox of […]
Read moreBroken String Enters Collaboration with Francis Crick Institute to Advance ALS Research
Rare Daily Staff Genomics company Broken String Biosciences said that it has entered a research collaboration with […]
Read moreResearchers Develop Global Approach for Using AI to Identify Kids with Rare Diseases
Rare Daily Staff Diagnosing children with rare, genetic diseases often involves misdiagnoses and prolonged diagnostic odysseys but […]
Read moreWhat’s Happening
Why Sharing Your Rare Disease Story Can Help
Rare disease advocates (and Global Genes staff!) talk about the importance of sharing your story, and how […]
Read moreRare Disease Focused Therapeutics M&A Soars & More — This Week in RARE Daily
This Week in RARE Daily is a feature from Global Genes where you can get a quick […]
Read moreHow Changing Sequencing Technologies Are Improving Diagnostic Success — 2024 NEXT Report
Time to diagnosis is being cut significantly in many cases due to genomic testing, but there are […]
Read more2024 RARE Mental Health Grant Recipients Announced
Global Genes is pleased to announce the rare disease patient advocacy organizations receiving 2024 RARE Mental Health […]
Read more2024 RARE Research Readiness Fellowship Recipients Announced
Global Genes is pleased to announce the rare disease patient advocacy organizations receiving 2024 RARE Research Readiness […]
Read moreZohreh Talebizadeh, Ph.D., Serves on Plenary Panel Session at Gatlinburg Conference
Zohreh Talebizadeh, Ph.D., Senior Director of the RARE-X Research Program for Global Genes, served on the Association […]
Read moreFDA Clears First Trial of Prime Medicine’s Editing Therapy & More — This Week in RARE Daily
This Week in RARE Daily is a feature from Global Genes where you can get a quick […]
Read moreShundra 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 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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