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RARE Daily: the official Global Genes blog

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

Drug Development

Relay Reports Positive Results of Experimental Therapy for Rare Vascular Anomalies

Rare Daily Staff Relay Therapeutics said Tuesday that early clinical data suggest its experimental drug zovegalisib may […]

Drug Development

Sanofi’s AATD Experimental Drug Outperforms Standard Treatment

Rare Daily Staff Sanofi reported new data this week suggesting its experimental therapy for a rare genetic […]

Drug Development

BioMarin Reports Mixed Phase 3 Results for BMN 401 in Rare ENPP1 Deficiency

Rare Daily Staff BioMarin Pharmaceutical reported mixed results from its pivotal phase 3 ENERGY 3 trial of […]

Drug Development

FDA Places Clinical Hold on Aardvark PWS Studies

Rare Daily Staff The U.S. Food and Drug Administration has placed a full clinical hold on Ardvark […]

Drug Development

DNA Editing Targets Root Cause of Dravet in Preclinical Study

Rare Daily Staff A preclinical study in mice suggests that precision gene editing could one day correct […]

Drug Development

FDA Accepts Opus Genetics Experimental Gene Therapy into Rare Disease Evidence Principles Program

Rare Daily Staff The Food and Drug Administration has accepted Opus Genetics’ experimental gene therapy for a […]

Drug Development

Satellite Bio Secures FDA Rare Pediatric Disease Designation for UCD Therapy SB-101

Rare Daily Staff The U.S. Food and Drug Administration granted Satellite Biosciences Rare Pediatric Disease designation for […]

Drug Development

Incyte Wins FDA Approval for Once-Daily Jakafi XR for Blood Disorders

Rare Daily Staff The U.S. Food and Drug Administration approved Incyte’s Jakafi XR, a once-daily extended-release version […]

Drug Development

UK Grants Passport Designation to Orchard’s Sanfilippo Gene Therapy

Rare Daily Staff Kyowa Kirin’s Orchard Therapeutics has received an Innovation Passport designation in the United Kingdom […]

Finance

RARE Daily

RTW Foundation Issues $573,000 in Grants to Address Gaps in Rare Disease Research

Rare Daily Staff RTW Foundation has awarded $573,000 in grants to eight rare disease organizations, underscoring the […]

RARE Daily

Latus Extends Series A Round to Raise Total of $97 Million

Rare Daily Staff Latus Bio said it raised $97 million in a series A financing to advance […]

RARE Daily

Cleveland Browns Owners Donate $12.5 Million to Advance Blood Cancer Research

Rare Daily Staff Oxford-Harrington Rare Disease Centre, the international drug discovery and development organization, said Cleveland Browns […]

RARE Daily

Climb Bio to Raise $110 Million in Private Placement

Rare Daily Staff Climb Bio said it has entered into a securities purchase agreement with a select […]

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 […]

Scientists Develop New Gene Editing Approach Capable of Gene-Sized Insertions

Rare Daily Staff Scientists at UMass Chan Medical School have unveiled a new gene editing approach that […]

Research

Tiny Worms May Unlock New Treatments for Rare Childhood Disorder

Rare Daily Staff Researchers at Brown University have developed genetically engineered versions of C. elegans—tiny worms commonly used […]

Regulatory

FDA Seeks Ideas for Repurposing Drugs to Address Unmet Needs

Rare Daily Staff The U.S. Food and Drug Administration is turning to drug repurposing as a new […]

Deals

Angelini to Buy Catalyst for $4.1 Billion

Rare Daily Staff Angelini Pharma said it will acquire Catalyst Pharmaceuticals for about $4.1 billion in cash […]

Deals

BioCryst inks European licensing deal for HAE drug candidate navenibart

Rare Daily Staff BioCryst Pharmaceuticals it has granted an Irish affiliate of Neopharmed Gentili exclusive rights to […]

Deals

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, […]

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

Raising Awareness of Clinical Trials

Using AI and Longitudinal Data to Transform Rare Disease Care

Cutting through the Diagnostic Maze for Rare Diseases

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

Drug Development

Relay Reports Positive Results of Experimental Therapy for Rare Vascular Anomalies

Sanofi’s AATD Experimental Drug Outperforms Standard Treatment

BioMarin Reports Mixed Phase 3 Results for BMN 401 in Rare ENPP1 Deficiency

FDA Places Clinical Hold on Aardvark PWS Studies

DNA Editing Targets Root Cause of Dravet in Preclinical Study

FDA Accepts Opus Genetics Experimental Gene Therapy into Rare Disease Evidence Principles Program

Satellite Bio Secures FDA Rare Pediatric Disease Designation for UCD Therapy SB-101

Incyte Wins FDA Approval for Once-Daily Jakafi XR for Blood Disorders

UK Grants Passport Designation to Orchard’s Sanfilippo Gene Therapy

Finance

RTW Foundation Issues $573,000 in Grants to Address Gaps in Rare Disease Research

Latus Extends Series A Round to Raise Total of $97 Million

Cleveland Browns Owners Donate $12.5 Million to Advance Blood Cancer Research

Climb Bio to Raise $110 Million in Private Placement

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

More Stories

Scientists Develop New Gene Editing Approach Capable of Gene-Sized Insertions

Tiny Worms May Unlock New Treatments for Rare Childhood Disorder

FDA Seeks Ideas for Repurposing Drugs to Address Unmet Needs

Angelini to Buy Catalyst for $4.1 Billion

BioCryst inks European licensing deal for HAE drug candidate navenibart

Chiesi to Buy KalVista for $1.9 Billion

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