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RARE Daily: the official Global Genes blog
Browse the latest news, articles, and blog posts from Global Genes.
Featured
Helping People with Undiagnosed Rare Diseases Find Answers
Michele Herndon’s son Mitchell began developing symptoms of an ultra-rare neurological condition in 2012. He went for […]
Read moreSpeeding and Scaling the Development of Genome Editing Therapies
Earlier this year the Innovative Genomics Institute and the life sciences tools conglomerate Danaher launched a collaborative […]
Read moreFebruary Roars with $3.2 Billion Raised by Public Rare Disease Therapeutics Developers
Wall Street interest in biotech boomed over the past few months as rare disease drug developers raised […]
Read moreGetting a Next-Generation Genome Editing Therapy for Sickle Cell Disease Back on Track
A serious adverse event in the first patient treated with an experimental genome editing therapy for sickle […]
Read moreDrug Development
Spruce Cuts Staff as CAH Study Misses Primary Endpoint
Rare Daily Staff Spruce Biosciences said topline results from its phase 2b study of tildacerfont in adult […]
Read moreFDA Awards Keros Fast Track Designation for Experimental Therapy to Treat MDS
Rare Daily Staff The U.S. Food and Drug Administration has granted Fast Track designation for Keros Therapeutics’ […]
Read moreFDA Expands Use of Mirum’s Livmarli to Include PFIC
Rare Daily Staff The U.S. Food and Drug Administration has expanded the approval of Mirum Pharmaceuticals’ Livmarli […]
Read moreAs Spending on Cell and Gene Therapies Grows, Challenges Remain
Rare Daily Staff Spending on cell and gene therapies grew to $5.9 billion in 2023, a 38 […]
Read moreTakeda Reports Positive Topline Results from Phase 2 Study in ITP
Rare Daily Staff Takeda reported positive topline results from a phase 2, randomized, double-blind, placebo-controlled study evaluating […]
Read moreFDA Grants Rare Pediatric Disease Designation to PepGen’s Therapeutic for DMD
Rare Daily Staff The U.S. Food and Drug Administration granted both orphan drug and rare pediatric disease […]
Read moreBioMarin to Present Long-Term Data on Achondroplasia Drug, New Data on Hypochondroplasia
Rare Daily Staff BioMarin said researchers will present positive results from an investigator-sponsored study of its drug […]
Read moreIncyte Reports Positive Data from Phase 2 Study in Rare Skin Disease
Rare Daily Staff Incyte reported results that its phase 2 study evaluating the efficacy and safety of […]
Read moreA Phase 3 Failure for Amylyx May Lead to Pulling ALS Drug from Market
Rare Daily Staff Amylyx Pharmaceuticals said it will consider removing its ALS drug Relyvrio from the market […]
Read moreFinance
Regulus Raises $100 Million in PIPE Financing to Advance Rare Kidney Disease Pipeline
Rare Daily Staff Regulus Therapeutics, a company focused on innovative microRNA therapeutics for orphan kidney diseases, entered […]
Read moreFebruary Roars with $3.2 Billion Raised by Public Rare Disease Therapeutics Developers
Wall Street interest in biotech boomed over the past few months as rare disease drug developers raised […]
Read moreLexeo Therapeutics Raises $95 Million Through Private Placement
Rare Daily Staff Lexeo, a company pursuing the development of gene therapies for rare and common diseases, […]
Read moreBanking on Positive Initial Data for DMD Gene Therapy, RegenxBio Raises $140 Million
Rare Daily Staff Two days after reporting positive initial efficacy data from its Duchenne muscular dystrophy trial […]
Read moreBridgeBio Raises $250 Million in Public Offering
Rare Daily Staff BridgeBio Pharma raised $250 million in an underwritten public offering of 8.6 million shares […]
Read moreSionna Raises $182 Million to Advance Novel Small Molecules in Cystic Fibrosis
Rare Daily Staff Sionna Therapeutics, a clinical-stage company developing differentiated treatments for cystic fibrosis, closed a $182 […]
Read moreQuoin Pharma Raises $6.5 Million to Advance Rare Disease Pipeline
Quoin Pharma Raises $6.5 Million to Advance Rare Disease Pipeline Rare Daily Staff Quoin Pharmaceuticals, a specialty […]
Read moreFirefly Fund Gifts $5 Million to Create Center for Rare Diseases at UT Austin Dell Medical School
Rare Daily Staff The Firefly Fund unveiled a gift of $5 million as the founding sponsor of […]
Read moreQuantile Health Raises $6 Million to Transform Patient Access to Gene Therapies
Rare Daily Staff Quantile Health, co-founded by MIT Professor Andrew Lo with the aim of solving the […]
Read moreMore Stories
AstraZeneca to Acquire Amolyt Pharma
AstraZeneca to Acquire Amolyt Pharma for Rare Endocrine Disease Portfolio Rare Daily Staff AstraZeneca will acquire Amolyt […]
Read moreResearchers Take Aim at DMD Therapies to Make Case for Timely Completion of Confirmatory Trials
Rare Daily Staff Spending on targeted DMD therapies reached $3.7 billion from 2016 to 2022, despite limited […]
Read moreAlmirall Licenses Eloxx Pharma’s ZKN-013 for Rare Dermatological Diseases
Rare Daily Staff Spanish biopharma Almirall entered an exclusive license agreement with Eloxx Pharmaceuticals to develop and […]
Read moreAllogene and Arbor Enter Gene Editing Licensing Deal for Allo CAR T Platform in Autoimmune Disease
Rare Daily Staff Allogene Therapeutics and Arbor Biotechnologies entered a non-exclusive, global gene editing licensing agreement for […]
Read moreBluebird Bio Enters First Outcomes-Based Agreement with Medicaid for SCD Gene Therapy
Rare Daily Staff Bluebird Bio said it has signed its first Medicaid outcomes-based agreement with the state […]
Read moreArmatus Bio Licenses Solid’s AAV Capsid to Develop an RNAi Therapy to treat FSHD
Rare Daily Staff Solid Biosciences entered into a non-exclusive worldwide license and collaboration agreement with Armatus Bio […]
Read moreWhat’s Happening
Rare, Ultra-Rare & Hyper-Rare: A Search for Paths Forward — 2024 NEXT Report
Over 40 years after the Orphan Drug Act of 1983, the definition of rare disease as it […]
Read moreRecapping Global Genes staff activities during Rare Disease Week
Mackenzie Abramson provides a recap of Rare Disease Day and Rare Disease Week activities in Washington D.C. […]
Read moreQuick and Simple Strategies to Help You and Your Family Reset and Recharge
As a rare disease caregiver or care partner, you may be feeling increasing pressure to do it […]
Read moreSigns of Depression and Anxiety in Children and Teens Living with a Rare Condition
Kids with rare diseases experience depression and anxiety at 2-3 times a higher rate than their peers. […]
Read moreFinding Support: A Compilation of Emotional and Mental Health Resources in the United States
There are a number of mental health resources available for rare disease patients, families and caregivers, but […]
Read moreGetting Help & Finding the Right Fit: Available Emotional and Mental Health Resources
Many resources are available for mental health care, but it is important to carefully research them, access […]
Read moreDepression and Anxiety: Understanding the Signs and Symptoms and Getting Help
Having a chronic and rare disease is a challenging experience, and especially difficult for children and adolescents, […]
Read moreAddressing and Managing Pain: Resources for Patients Who Have Pain
The management of chronic pain has proven to be a significant challenge for rare diseases patients and […]
Read moreCIRM Awards $56 Million to Advance Clinical Research, Including Lupus and Glioblastoma
CIRM Awards $56 Million to Advance Clinical Research, Including Lupus and Glioblastoma Rare Daily Staff The California […]
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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