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RARE Daily: the official Global Genes blog

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

Drug Development

New Biotech Launches as Part of a Father’s Quest to Get Treatment for Son

Rare Daily Staff A father’s effort to find a treatment for his infant son has led to […]

Longeveron Heart Defect Study Missed Main Endpoint, but Analysis Found Fewer Serious Cardiac Events in Infants

Rare Daily Staff Longeveron said an updated analysis of its experimental cell therapy laromestrocel found fewer major […]

Drug Development

Vertex Reports Published Results for IgA Nephropathy Drug Ahead of FDA Decision

Rare Daily Staff Vertex Pharmaceuticals said its experimental drug povetacicept substantially reduced protein in the urine in […]

Drug Development

Alnylam’s Huntington’s Drug Lowers Disease-Linked Protein in Early Trial

Rare Daily Staff Alnylam Pharmaceuticals said its experimental Huntington’s disease treatment brezosiran substantially reduced levels of a […]

Drug Development

Priovant Will Seek Expanded Approval of Lisraya After Positive Results in Inflammatory Eye Disease

Rare Daily Staff Roivant and its subsidiary Priovant Therapeutics said their drug Lisraya significantly delayed treatment failure […]

Drug Development

FDA approves BioCryst’s Pellet Formulation of Orladeyo for HAE

Rare Daily Staff The U.S. Food and Drug Administration has approved a pellet formulation of BioCryst Pharmaceuticals’ […]

Drug Development

Disc Medicine’s Bitopertin Meets Main Goals in Late-Stage Trial for EPP

Rare Daily Staff Disc Medicine said its experimental drug bitopertin met all primary and secondary goals in […]

Drug Development

FDA approves Roche’s Enspryng as First at-Home Injection for TED

Rare Daily Staff The U.S. Food and Drug Administration approved Roche’s Enspryng for adults with thyroid eye […]

Drug Development

Takeda’s Experimental Immune Therapy Cuts Infusion Time in Late-Stage Study

Rare Daily Staff Takeda said its experimental treatment TAK-881 delivered the same antibody dose as its approved […]

Finance

RARE Daily

Nouscom Raises $135 Million to Advance Cancer-Prevention Immunotherapy for Lynch Syndrome

Rare Daily Staff Nouscom raised about $135 million (€120 million) to advance an experimental immunotherapy designed to […]

RARE Daily

Ultragenyx Agrees to Sell Rare Pediatric Disease Voucher for $203 Million

Rare Daily Staff Ultragenyx Pharmaceutical has agreed to sell a rare pediatric disease priority review voucher for […]

RARE Daily

OMass Raises $310 Million to Advance Endocrine and Immunology Drugs

Rare Daily Staff OMass Therapeutics said it has raised $310 million (£230 million) in a series C […]

RARE Daily

Altay Raises $37 Million to Advance Oral Drug for Inherited Muscle Disease

Rare Daily Staff Altay Therapeutics said it raised $37 million in series A financing to advance its […]

RARE Daily

X4 Secures up to $150 Million in Debt Financing as Chronic Neutropenia Trial Advances

Rare Daily Staff X4 Pharmaceuticals said it has entered into a loan agreement with K2 HealthVentures worth […]

RARE Daily

Ultragenyx Agrees to Sell Priority Review Voucher for $210 Million

Rare Daily Staff Ultragenyx Pharmaceutical has agreed to sell a rare pediatric disease priority review voucher for […]

RARE Daily

AlphaRose Secures NIH SBIR Fast Track Grant to Advance Rosiphersen for Bain Syndrome

AlphaRose Therapeutics said it has received a multi-million-dollar Small Business Innovation Research Fast Track grant from the […]

RARE Daily

ARPA-H Awards $35 Million to UNC, Emory to Build Rare-Disease AI Data Resource

Rare Daily Staff The Advanced Research Projects Agency for Health’s Rare Disease AI/ML for Precision Integrated Diagnostics […]

RARE Daily

Quoin Pharmaceuticals Prices Private Placement with up to $50 Million in Potential Proceeds

Rare Daily Staff Quoin Pharmaceuticals said it has entered into a private-placement financing expected to generate approximately […]

Anthropic Acquires Mithril Medicine to Test AI-Supported Model for Rare Disease Drug Development

Rare Daily Staff Anthropic, the artificial intelligence company behind Claude, has acquired Mithril Medicine, bringing two early-stage […]

Regulatory

FDA Issues Draft Guidance on Personalized Gene-Editing Treatments for Rare Diseases

Rare Daily Staff The U.S. Food and Drug Administration has proposed a framework that could make it […]

Deals

Merck KGaA to Acquire Silence Therapeutics for $1.3 Billion

Rare Daily Staff Germany’s Merck KGaA has agreed to acquire U.K. biotechnology company Silence Therapeutics in a […]

Regulatory

FDA Launches Effort to Reshape Rare Disease Drug Development

Rare Daily Staff The U.S. Food and Drug Administration is developing a new framework for evaluating rare […]

Diagnosis

Ambry Launches Genome Sequencing Test to Help Diagnose Rare Diseases

Rare Daily Staff Tempus AI subsidiary Ambry Genetics said it launched GenomeNext, a whole-genome sequencing test that […]

Support

I AM ALS, Protected Tomorrows Launch Financial Planning Program for ALS Families

Rare Daily Staff I AM ALS and financial advisory organization Protected Tomorrows have launched a nationwide virtual […]

What’s Happening

Big News for RARE-X and Rare Disease Families: Citizen Health Partnership

Dear Global Genes Community, Today, I’m thrilled to share an exciting new chapter for RARE-X and for […]

Drug Development

Acting FDA commissioner pledges to prevent political interference in rare disease meeting

Global Genes was honored to be invited by the FDA to participate in a closed roundtable discussion […]

Drug Development

CURE SYNGAP1 announces $111,960 investment in RARE-X partnership to advance ProMMIS patient-reported outcome measure data collection – PR46

CURE SYNGAP1 is proud to announce a strategic investment of $111,960 in RARE-X to accelerate the collection of high-quality […]

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 […]

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.

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RARE Daily: the official Global Genes blog

Featured

Yearning for Normal

Building Better Rare Disease Data

Using AI to Help Patients Get the Care They Need

Using Exosomes to Tackle the Delivery Challenge of Genetic Medicines for the Brain

Drug Development

New Biotech Launches as Part of a Father’s Quest to Get Treatment for Son

Longeveron Heart Defect Study Missed Main Endpoint, but Analysis Found Fewer Serious Cardiac Events in Infants

Vertex Reports Published Results for IgA Nephropathy Drug Ahead of FDA Decision

Alnylam’s Huntington’s Drug Lowers Disease-Linked Protein in Early Trial

Priovant Will Seek Expanded Approval of Lisraya After Positive Results in Inflammatory Eye Disease

FDA approves BioCryst’s Pellet Formulation of Orladeyo for HAE

Disc Medicine’s Bitopertin Meets Main Goals in Late-Stage Trial for EPP

FDA approves Roche’s Enspryng as First at-Home Injection for TED

Takeda’s Experimental Immune Therapy Cuts Infusion Time in Late-Stage Study

Finance

Nouscom Raises $135 Million to Advance Cancer-Prevention Immunotherapy for Lynch Syndrome

Ultragenyx Agrees to Sell Rare Pediatric Disease Voucher for $203 Million

OMass Raises $310 Million to Advance Endocrine and Immunology Drugs

Altay Raises $37 Million to Advance Oral Drug for Inherited Muscle Disease

X4 Secures up to $150 Million in Debt Financing as Chronic Neutropenia Trial Advances

Ultragenyx Agrees to Sell Priority Review Voucher for $210 Million

AlphaRose Secures NIH SBIR Fast Track Grant to Advance Rosiphersen for Bain Syndrome

ARPA-H Awards $35 Million to UNC, Emory to Build Rare-Disease AI Data Resource

Quoin Pharmaceuticals Prices Private Placement with up to $50 Million in Potential Proceeds

More Stories

Anthropic Acquires Mithril Medicine to Test AI-Supported Model for Rare Disease Drug Development

FDA Issues Draft Guidance on Personalized Gene-Editing Treatments for Rare Diseases

Merck KGaA to Acquire Silence Therapeutics for $1.3 Billion

FDA Launches Effort to Reshape Rare Disease Drug Development

Ambry Launches Genome Sequencing Test to Help Diagnose Rare Diseases

I AM ALS, Protected Tomorrows Launch Financial Planning Program for ALS Families

What’s Happening

Big News for RARE-X and Rare Disease Families: Citizen Health Partnership

Acting FDA commissioner pledges to prevent political interference in rare disease meeting

CURE SYNGAP1 announces $111,960 investment in RARE-X partnership to advance ProMMIS patient-reported outcome measure data collection – PR46

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

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