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RARE Daily: the official Global Genes blog

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

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 […]

Drug Development

Sanofi’s Drug Meets Primary Endpoints in Type 3 Gaucher Disease Study

Sanofi said its experimental therapy venglustat met the primary and three of four key secondary endpoints in […]

Drug Development

Quince Stock Tumbles and Board Members Resign After Phase 3 Miss in A-T

Rare Daily Staff Quince Therapeutics said its lead experimental therapy failed to meet the primary and secondary […]

Drug Development

FDA Places Clinical Hold on Regenxbio’s MPS1 Gene Therapy

The U.S. Food and Drug Administration has placed a clinical hold on Regenxbio’s experimental gene therapy, RGX-111, […]

Drug Development

FDA Grants Immusoft RPPD for MPSII Cell Therapy

Rare Daily Staff The U.S. Food and Drug Administration granted Immusoft Rare Pediatric Disease designation to ISP-002, […]

Drug Development

Sarepta Reports Three-Year Topline Data from Phase 3 DMD Study

Rare Daily Staff Sarepta Therapeutics said data from a phase 3 study of its gene therapy Elevidys […]

Drug Development

IntraBio’s A-T Therapy Meets Phase 3 Endpoints, Will Seek FDA Approval

IntraBio reported topline results from its pivotal phase 3 clinical trial of its experimental therapy levacetylleucine for […]

Drug Development

FDA Approves Sentynl’s Menkes Disease Therapy

Rare Daily Staff The U.S. Food and Drug Administration approved Sentynl Therapeutics’ Zycubo, the first treatment in […]

Finance

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 […]

RARE Daily

AAVantgarde Closes $70.7 Million Financing

Rare Daily Staff Italy-based AAVantgarde Bio said it completed a $70.1 million (€61 million) series A financing […]

RARE Daily

Ovid Prices PIPE of up to $175 Million

Rare Daily Staff Ovid Therapeutics said it has entered into a securities purchase agreement for a PIPE […]

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 […]

Living with a RARE Disease

Seeing Perseverance through a New Lens

Rare Daily Staff The short film Ho’omao tells the story of Joey, a retired firefighter and law […]

Deals

Moderna Enters Strategic Collaboration with Recordati for Rare Metabolic Disorder

Rare Daily Staff Moderna said it entered into a strategic collaboration with Italy-based Recordati to advance Moderna’s […]

Deals

Charles River Enters Manufacturing Collaboration with Gazi University

Rare Daily Staff Charles River Laboratories International has partnered with Ankara-based Gazi University Faculty of Medicine to […]

Deals

n-Lorem Enters Collaboration with EspeRare to Expand Access to Individualized ASOs for Rare Genetic Diseases

Rare Daily Staff The n-Lorem Foundation and the Geneva-based EspeRare Foundation said they have entered a strategic […]

Strategy

Financial News Channel Launches CNBC Cures to Raise Rare Disease Awareness

Rare Daily Staff The financial news cable channel CNBC announced the launch of CNBC Cures, an 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

Rewriting Rare Disease R&D with Foundation Models

Resetting Aberrant Tregs Epigenetically to Treat Autoimmune Diseases

Finding a Ready Treatment for a Newly Discovered, Ultra-Rare Disease

One Rare Mother’s Quest to Rewrite Her Son’s Future with a Gene Therapy

Drug Development

Trump Signs Legislation to Reauthorize PRV Program

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

Sanofi’s Drug Meets Primary Endpoints in Type 3 Gaucher Disease Study

Quince Stock Tumbles and Board Members Resign After Phase 3 Miss in A-T

FDA Places Clinical Hold on Regenxbio’s MPS1 Gene Therapy

FDA Grants Immusoft RPPD for MPSII Cell Therapy

Sarepta Reports Three-Year Topline Data from Phase 3 DMD Study

IntraBio’s A-T Therapy Meets Phase 3 Endpoints, Will Seek FDA Approval

FDA Approves Sentynl’s Menkes Disease Therapy

Finance

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

AAVantgarde Closes $70.7 Million Financing

Ovid Prices PIPE of up to $175 Million

More Stories

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

Seeing Perseverance through a New Lens

Moderna Enters Strategic Collaboration with Recordati for Rare Metabolic Disorder

Charles River Enters Manufacturing Collaboration with Gazi University

n-Lorem Enters Collaboration with EspeRare to Expand Access to Individualized ASOs for Rare Genetic Diseases

Financial News Channel Launches CNBC Cures to Raise Rare Disease Awareness

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