RARE Daily landing

Learn

RARE Daily: the official Global Genes blog

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

Drug Development

FDA Grants Tessera Fast Track, Orphan Drug Designations for AATD Gene Writing Therapy

Rare Daily Staff The U.S. Food and Drug Administration granted Tessera Therapeutics Fast Track and Orphan Drug […]

CervoMed’s Experimental Drug Selected for UK Platform Trial

Rare Daily Staff CervoMed said its lead experimental candidate, neflamapimod, has been selected for inclusion in the […]

Drug Development

Roche Reports Positive Phase 3 Results for Gazyva in Rare Kidney Disease

Rare Daily Staff Roche reported that its drug Gazyva met the main goal of a global phase […]

Drug Development

FDA Rejects Disc Medicine’s EPP Drug

Rare Daily Staff The U.S. Food and Drug Administration told Disc Medicine it would not approve its […]

Drug Development

PTC Therapeutics Withdraws FDA Resubmission for DMD

Rare Daily Staff PTC Therapeutics said it has withdrawn the new drug application resubmission for Translarna (ataluren) […]

Drug Development

BridgeBio Reports Positive Phase 3 Results for Oral Achondroplasia Therapy

Rare Daily Staff BridgeBio Pharma said its experimental oral drug, infigratinib, helped children with achondroplasia grow faster […]

Drug Development

FDA Rejects Regenxbio’s MPS II Gene Therapy

Rare Daily Staff The U.S. Food and Drug Administration rejected Regenxbio’s application for accelerated approval of RGX-121, […]

Drug Development

Trump Signs Legislation to Reauthorize PRV Program

Rare Daily Staff The Rare Pediatric Disease Priority Review Voucher program has been reauthorized through fiscal 2029 […]

Drug Development

Ultragenyx Reports Positive Longer-Term Data for MPS IIIA Gene Therapy as It Resubmits BLA to FDA

Rare Daily Staff Ultragenyx Pharmaceutical said new long-term data from clinical studies of its experimental gene therapy […]

Finance

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 […]

RARE Daily

Mendra Launches with $82 Million to Focus on Unmet Needs in Rare Disease

Rare Daily Staff Mendra, a biopharmaceutical company seeking to advance rare disease therapies utilizing artificial intelligence, announced […]

RARE Daily

Doudna’s Aurora Launches with $16 Million to Industrialize Personalized Genetic Therapies

Rare Daily Staff Aurora Therapeutics, a company seeking to transform personalized gene editing from a one-patient breakthrough […]

RARE Daily

CIRM Awards Cure Rare Disease $7.4 Million to Advance LGMD Gene Therapy

Rare Daily Staff The California Institute for Regenerative Medicine has awarded Cure Rare Disease a $7.4 million […]

RARE Daily

Denali Enters $275 Million Funding Agreement with Royalty Pharma

Rare Daily Staff Denali Therapeutics said it entered into a $275 million synthetic royalty funding agreement with […]

RARE Daily

Protego Raises $130 Million to Fund AL Amyloidosis Pivotal Study

Rare Daily Staff Protego Biopharma, which is pioneering first-in-class small-molecule therapeutics that reprogram protein folding to address […]

Avidity Spinout Launches with $270 Million, Focus on Rare Cardiomyopathies

Rare Daily Staff Atrium Therapeutics, a spinout from Avidity Biosciences, has launched as a newly independent, publicly […]

Regulatory

FDA Unveils Framework to Speed Up Personalized Therapies for Ultra-Rare Diseases

Rare Daily Staff The U.S. Food and Drug Administration on Monday released draft guidance outlining a new regulatory framework […]

Research

UC Berkeley Partners with BioMarin to Accelerate Rare Disease Research

The UC Berkeley Molecular Therapeutics Initiative (MTI) has entered into a new research collaboration with BioMarin Pharmaceutical […]

Strategy

Ultragenyx Cuts 10 Percent of Staff as Part of Restructuring

Rare Daily Staff Ultragenyx said it has initiated a strategic restructuring plan that will eliminate 10 percent […]

Research

Oxford-Harrington Awards Nearly $900K in Friedreich’s Ataxia Grants

Rare Daily Staff The Oxford-Harrington Rare Disease Centre’s FA Alliance Innovation Fund has awarded funding to five […]

Research

Kingsmore Stepping Down from Rady Children’s Institute for Genomic Medicine

Rare Daily Staff Stephen Kingsmore, a pioneer in the clinical use of rapid whole genome sequencing, will […]

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

A Data Strategy to Capitalize on a Multi‑Trillion Dollar Opportunity

When a Gene Echoes Through a Family

Changing the Autoimmune Disease Playbook with RNA-Engineered CAR T Cells

Rewriting Rare Disease R&D with Foundation Models

Drug Development

FDA Grants Tessera Fast Track, Orphan Drug Designations for AATD Gene Writing Therapy

CervoMed’s Experimental Drug Selected for UK Platform Trial

Roche Reports Positive Phase 3 Results for Gazyva in Rare Kidney Disease

FDA Rejects Disc Medicine’s EPP Drug

PTC Therapeutics Withdraws FDA Resubmission for DMD

BridgeBio Reports Positive Phase 3 Results for Oral Achondroplasia Therapy

FDA Rejects Regenxbio’s MPS II Gene Therapy

Trump Signs Legislation to Reauthorize PRV Program

Ultragenyx Reports Positive Longer-Term Data for MPS IIIA Gene Therapy as It Resubmits BLA to FDA

Finance

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

Mendra Launches with $82 Million to Focus on Unmet Needs in Rare Disease

Doudna’s Aurora Launches with $16 Million to Industrialize Personalized Genetic Therapies

CIRM Awards Cure Rare Disease $7.4 Million to Advance LGMD Gene Therapy

Denali Enters $275 Million Funding Agreement with Royalty Pharma

Protego Raises $130 Million to Fund AL Amyloidosis Pivotal Study

More Stories

Avidity Spinout Launches with $270 Million, Focus on Rare Cardiomyopathies

FDA Unveils Framework to Speed Up Personalized Therapies for Ultra-Rare Diseases

UC Berkeley Partners with BioMarin to Accelerate Rare Disease Research

Ultragenyx Cuts 10 Percent of Staff as Part of Restructuring

Oxford-Harrington Awards Nearly $900K in Friedreich’s Ataxia Grants

Kingsmore Stepping Down from Rady Children’s Institute for Genomic Medicine

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