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RARE Daily: the official Global Genes blog

Browse the latest news, articles, and blog posts from Global Genes.

Drug Development

FDA Grants Expression Fast Track and RPPD for Hemophilia A Stem Cell Therapy

Rare Daily Staff The U.S. Food and Drug Administration has granted Fast Track and Rare Pediatric Disease […]

Drug Development

Decades-Old Drug Show Promise as Treatment for Ultra-Rare Disorder

Rare Daily Staff A decades-old drug originally developed to treat a tropical disease is offering new hope […]

Drug Development

FDA Proposes Pulling Amgen Rare Disease Drug from Market

Rare Daily Staff The U.S. Food and Drug Administration has taken the unusual step of proposing to […]

Drug Development

Intellia Reports Positive Phase 3 Results in HAE, a First for an In Vivo Gene Editor

Rare Daily Staff Intellia Therapeutics said its experimental gene-editing treatment for hereditary angioedema met the main goals […]

Drug Development

FDA Approves Regeneron’s Gene Therapy for Hearing Loss

Rare Daily Staff The U.S. Food and Drug Administration has granted accelerated approval to Regeneron’s Otarmeni, the […]

Drug Development

Ionis Reports Positive New Data on Experimental Alexander Disease Therapy

Rare Daily Staff Ionis Pharmaceuticals released new data showing its experimental drug zilganersen for Alexander disease helped […]

Drug Development

FDA Grants Star Two Key Designations for VWD Therapy

Rare Daily Staff The U.S. Food and Drug Administration granted Star Therapeutics Rare Pediatric Disease and Breakthrough […]

Drug Development

FDA Grants Full Approval to Travere’s Filspari for FSGS

Rare Daily Staff The U.S. Food and Drug Administration has granted Travere Therapeutics full approval for Filspari, […]

Drug Development

FDA Grants RMAT Designation to Grace Science’s Gene Therapy for NGLY1 Deficiency

Rare Daily Staff The U.S. Food and Drug Administration has granted Regenerative Medicine Advanced Therapy (RMAT) designation […]

Finance

RARE Daily

Rocket Sells PRV for $180 Million

Rare Daily Staff Rocket Pharmaceuticals said it signed a definitive agreement to sell for $180 million the […]

RARE Daily

Immutrin Raises $87 Million to Advance ATTR-CM Therapy

Rare Daily Staff U.K.-based Immutrin raised $87 million (£65 million) in an oversubscribed series A financing to […]

RARE Daily

Prothena Receives $50 Million Milestone Payment from Novo for ATTR-CM Progress

Rare Daily Staff Prothena said it received a $50 million milestone payment from Novo Nordisk after reaching […]

RARE Daily

Atavistik Bio Adds $40 Million to Series B to Advance HHT and MPN Programs

Rare Daily Staff Atavistik Bio said it raised an additional $40 million in an extension of its […]

RARE Daily

Antiverse Raises $9.3 Million, Enters Research Agreement with Cystic Fibrosis Foundation

Rare Daily Staff Antiverse, a biotech using artificial intelligence to design antibodies for traditionally “undruggable” targets, has […]

RARE Daily

ARPA-H to Provide Every Cure up to $76 Million for AI-Drive Drug Repurposing

Rare Daily Staff The Advanced Research Projects Agency for Health (ARPA-H) will provide up to $76 million […]

RARE Daily

Cyprium to Sell PRV for $205 Million

Rare Daily Staff Cyprium Therapeutics, a majority-owned subsidiary of Fortress Biotech, agreed to sell its Rare Pediatric […]

RARE Daily

CIRM Approves $100 Million Plan to Accelerate Genetic Therapies for Rare Diseases

Rare Daily Staff The California Institute for Regenerative Medicine has approved a new funding program to accelerate […]

RARE Daily

Ocugen Closes $22.5 Million Offering

Rare Daily Staff Ocugen, which is developing gene therapies for blinding diseases, said it raised $22.5 million […]

Chiesi to Buy KalVista for $1.9 Billion

Rare Daily Staff Chiesi Group said it will buy KalVista Pharmaceuticals for $27 a share in cash, […]

Research

Gene Therapy Innovators Share 2026 Breakthrough Prize

Rare Daily Staff The Breakthrough Prize Foundation awarded the 2026 Breakthrough Prizes in Life Sciences to a […]

Diagnosis

CHOP Using RNA Sequencing to Diagnose Rare Disease

Rare Daily Staff Researchers at Children’s Hospital of Philadelphia have developed a powerful new technology that could […]

Deals

Lunai Bioworks and Geneial to Develop Rare Disease Patient Cohorts

Rare Daily Staff Lunai Bioworks and Geneial announced plans to collaborate on building trial-ready patient cohorts in […]

Research

Illumina Partners with D3b to Advance Genomic Research in Pediatric Rare Diseases

Rare Daily Staff Illumina and the Center for Data-Driven Discovery in Biomedicine (D3b) have entered a partnership […]

Diagnosis

Labcorp and CHOP Collaborate to Improve Diagnosis of Pediatric Patients

Rare Daily Staff Labcorp and Children’s Hospital of Philadelphia are teaming up on a new effort to […]

What’s Happening

Savara’s Early Access Program

Savara’s Early Access Program (EAP) for molgramostim inhalation solution (molgramostim) in Autoimmune Pulmonary Alveolar Proteinosis (Autoimmune […]

Publication Announcement — RARE-X: Advancing Rare Disease Research Through Patient-Driven Data

Global Genes is excited to announce “RARE-X: A patient-driven approach for collecting symptom and patient-reported outcome data […]

Accelerating Rare Disease Research Through Collaboration: Global Genes and Notre Dame Launch the Research Acceleration Program

Global Genes and University of Notre Dame have partnered to launch the Research Acceleration Program, a collaborative […]

Equity

RARE Advocacy Exchange Session 8, Knowing Your Rare Rights

This session recording is from Oct. 16, 2025 Rare Advocacy Exchange Session 8: Knowing the Rights for […]

Living with a RARE Disease

Global Genes Guide to Starting a Nonprofit Patient Advocacy Organization

This Global Genes Quick Guide is a resource for advocates focused on pressing topics causing challenges in […]

Diagnosis

RARE Advocacy Exchange Session 7, Getting a Precise Genetic Diagnosis

This session recording is from Sept. 18, 2025 Rare Advocacy Exchange Session 7: Getting A Precise Diagnosis […]

Living with a RARE Disease

RARE Advocacy Exchange Session 6 : Grieving a RARE Diagnosis

Rare disease grief differs from the grief we commonly associate with losing a loved one to death. […]

A Student’s Perspective: Lessons from the 2025 RARE Drug Development Symposium

During my first week as a Harvard College junior, I was delighted to be given the opportunity […]

Diagnosis

Global Genes Guide to Genetic Diagnosis

This Global Genes Quick Guide is a resource for advocates focused on pressing topics causing challenges in […]

Reports

Diagnosis

More Resources: Dictionary of Rare Diseases

Search for a specific disease in our extensive list of rare diseases for disease-specific information, news, and resources.

Search Our Full Disease Dictionary

RARE Daily: the official Global Genes blog

Featured

How Parents Took Development of a Gene Therapy into Their Own Hands

Matching the Right Therapy to the Right Child with a Rare Cancer

From Treating Symptoms to Addressing Causes in Rare Autoimmune Diseases

A Mother’s Journey to Rewrite a Neurodevelopmental Disorder

Drug Development

FDA Grants Expression Fast Track and RPPD for Hemophilia A Stem Cell Therapy

Decades-Old Drug Show Promise as Treatment for Ultra-Rare Disorder

FDA Proposes Pulling Amgen Rare Disease Drug from Market

Intellia Reports Positive Phase 3 Results in HAE, a First for an In Vivo Gene Editor

FDA Approves Regeneron’s Gene Therapy for Hearing Loss

Ionis Reports Positive New Data on Experimental Alexander Disease Therapy

FDA Grants Star Two Key Designations for VWD Therapy

FDA Grants Full Approval to Travere’s Filspari for FSGS

FDA Grants RMAT Designation to Grace Science’s Gene Therapy for NGLY1 Deficiency

Finance

Rocket Sells PRV for $180 Million

Immutrin Raises $87 Million to Advance ATTR-CM Therapy

Prothena Receives $50 Million Milestone Payment from Novo for ATTR-CM Progress

Atavistik Bio Adds $40 Million to Series B to Advance HHT and MPN Programs

Antiverse Raises $9.3 Million, Enters Research Agreement with Cystic Fibrosis Foundation

ARPA-H to Provide Every Cure up to $76 Million for AI-Drive Drug Repurposing

Cyprium to Sell PRV for $205 Million

CIRM Approves $100 Million Plan to Accelerate Genetic Therapies for Rare Diseases

Ocugen Closes $22.5 Million Offering

More Stories

Chiesi to Buy KalVista for $1.9 Billion

Gene Therapy Innovators Share 2026 Breakthrough Prize

CHOP Using RNA Sequencing to Diagnose Rare Disease

Lunai Bioworks and Geneial to Develop Rare Disease Patient Cohorts

Illumina Partners with D3b to Advance Genomic Research in Pediatric Rare Diseases

Labcorp and CHOP Collaborate to Improve Diagnosis of Pediatric Patients

What’s Happening

Savara’s Early Access Program

Publication Announcement — RARE-X: Advancing Rare Disease Research Through Patient-Driven Data

Accelerating Rare Disease Research Through Collaboration: Global Genes and Notre Dame Launch the Research Acceleration Program

RARE Advocacy Exchange Session 8, Knowing Your Rare Rights

Global Genes Guide to Starting a Nonprofit Patient Advocacy Organization

RARE Advocacy Exchange Session 7, Getting a Precise Genetic Diagnosis

RARE Advocacy Exchange Session 6 : Grieving a RARE Diagnosis

A Student’s Perspective: Lessons from the 2025 RARE Drug Development Symposium

Global Genes Guide to Genetic Diagnosis

Reports

Early and Often: Reimagining patient community engagement to improve clinical trials feasibility

Continuing Advocacy and Expanding Research Efforts: Global Genes 2023 Impact Report

Global Genes 2023 RARE Impact Grants Report

More Resources: Dictionary of Rare Diseases