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RARE Daily: the official Global Genes blog

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

Drug Development

BioCryst to Scrap Internal Discovery

Rare Daily Staff BioCryst Pharmaceuticals is shutting down its internal drug discovery operations and closing its Birmingham, […]

Drug Development

Gene Editing Therapy for Rare Immune Disorder Moves Closer to Clinic

Rare Daily Staff A first-in-class gene therapy targeting CTLA-4 insufficiency, a rare and debilitating immune disorder, is […]

Drug Development

FDA Approves Viridian’s TED Therapy

Rare Daily Staff The U.S. Food and Drug Administration has approved Viridian Therapeutics’ Lumvoa for thyroid eye […]

Drug Development

FDA Reverses Course on Regenxbio’s MPS II Gene Therapy, Clearing Path to Possible Accelerated Approval

Rare Daily Staff Regenxbio said it has reached agreement with the U.S. Food and Drug Administration on […]

Drug Development

FDA Reverses on UniQure’s Huntington’s Disease Gene Therapy as Company Plans to Seek Accelerated Approval

Rare Daily Staff The U.S. Food and Drug Administration in a reversal has agreed that mid-stage clinical […]

Drug Development

Jazz’s Xywav Shows Promise in Improving Heart Health Markers for People with Narcolepsy and Idiopathic Hypersomnia

Rare Daily Staff Jazz Pharmaceuticals reported new data suggesting its low-sodium sleep medicine Xywav may offer benefits […]

Drug Development

Rhythm Reports Durable Weight Loss with MC4R Drugs in Rare Obesity Disorders

Rare Daily Staff Rhythm Pharmaceuticals reported new data showing that its melanocortin‑4 receptor (MC4R) drugs can drive […]

Drug Development

Alexion’s Ultomiris wins FDA Priority Review in IgA nephropathy

Rare Daily Staff The U.S. Food and Drug Administration granted Alexion, AstraZeneca’s rare disease unit, Priority Review […]

Drug Development

FDA Grants Gen1E Orphan and Rare Pediatric Disease Designations for DMD Therapy

Rare Daily Staff The U.S. Food and Drug Administration (FDA) has granted Gen1E Life Sciences both Orphan […]

Finance

RARE Daily

CIRM Issues $60 Million in Grants Including Funding for FOXG1 Gene Therapy

Rare Daily Staff California Institute of Regenerative Medicine, the state’s stem cell agency, is issuing $60 million […]

RARE Daily

UniQure Raises $225 Million to Back Accelerated Approval Bid for Huntington’s Gene Therapy

Rare Daily Staff UniQure is capitalizing on an improved regulatory outlook for its experimental Huntington’s disease gene […]

RARE Daily

Sangamo Files for Bankruptcy, Lilly and Astellas Line up as Lead Bidders for Assets

Rare Daily Staff Sangamo Therapeutics has filed for Chapter 11 bankruptcy protection and entered into asset sale […]

RARE Daily

Nura Raises $73.8 Million to Advance Novel ALS Strategy

Rare Daily Staff Nura Bio said it raised $73.8 million in series B financing and began clinical […]

RARE Daily

Beren Raises $300 Million to Prepare for Potential Commercial Launch of NPC Therapy

Rare Daily Staff Beren Therapeutics said it raised $300 million to support the potential U.S. commercial launch […]

RARE Daily

Novellia Raises $18 Million to Expand RWD Platform

Rare Daily Staff Novellia said it raised $18 million in a series A financing round to expand […]

RARE Daily

Oak Hill Bio Raises $32.5 Million to Advance ASO in Angelman Syndrome

Rare Daily Staff Oak Hill Bio announced it has closed a $32.5 million series A financing to […]

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 […]

Ionis Enters Global Licensing Deal with Recordati for Alexander disease ASO

Rare Daily Staff Ionis Pharmaceuticals has struck a global licensing deal with Italy-based Recordati to bring its […]

Deals

Incyte to Acquire Vega for up to $2 Billion

Rare Daily Staff Incyte is expanding its hematology portfolio into bleeding disorders through a deal to acquire […]

Deals

Cartesian Deal Advances Development of In vivo CAR-T for Autoimmune Diseases

Rare Daily Staff Cartesian Therapeutics has entered a strategic licensing agreement with WestGene Biopharma aimed at accelerating […]

Regulatory

BrainStorm Call for Updated FDA Approaches for Rare Disease Therapies in Journal Article

Rare Daily Staff BrainStorm Cell Therapeutics’ CEO has escalated the company’s long‑running dispute with the U.S. Food […]

Deals

Rallybio Agrees to Reverse Merger

Rare Daily Staff Rare disease drug developer Rallybio said it agreed to a reverse merger with Avenzo […]

Deals

Servier to Acquire Edgewise Muscular Dystrophy Business for up to $2.65 Billion

Servier to Acquire Edgewise Muscular Dystrophy Business for up to $2.65 Billion Rare Daily Staff Servier has […]

What’s Happening

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 […]

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. […]

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

Emerging from a Life in the Shadows

Fitting Big Genes into Small Vectors

Targeting Iron Dysregulation in the Neurodegenerative Condition MSA

Long Reads, Shorter Journeys

Drug Development

BioCryst to Scrap Internal Discovery

Gene Editing Therapy for Rare Immune Disorder Moves Closer to Clinic

FDA Approves Viridian’s TED Therapy

FDA Reverses Course on Regenxbio’s MPS II Gene Therapy, Clearing Path to Possible Accelerated Approval

FDA Reverses on UniQure’s Huntington’s Disease Gene Therapy as Company Plans to Seek Accelerated Approval

Jazz’s Xywav Shows Promise in Improving Heart Health Markers for People with Narcolepsy and Idiopathic Hypersomnia

Rhythm Reports Durable Weight Loss with MC4R Drugs in Rare Obesity Disorders

Alexion’s Ultomiris wins FDA Priority Review in IgA nephropathy

FDA Grants Gen1E Orphan and Rare Pediatric Disease Designations for DMD Therapy

Finance

CIRM Issues $60 Million in Grants Including Funding for FOXG1 Gene Therapy

UniQure Raises $225 Million to Back Accelerated Approval Bid for Huntington’s Gene Therapy

Sangamo Files for Bankruptcy, Lilly and Astellas Line up as Lead Bidders for Assets

Nura Raises $73.8 Million to Advance Novel ALS Strategy

Beren Raises $300 Million to Prepare for Potential Commercial Launch of NPC Therapy

Novellia Raises $18 Million to Expand RWD Platform

Oak Hill Bio Raises $32.5 Million to Advance ASO in Angelman Syndrome

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

More Stories

Ionis Enters Global Licensing Deal with Recordati for Alexander disease ASO

Incyte to Acquire Vega for up to $2 Billion

Cartesian Deal Advances Development of In vivo CAR-T for Autoimmune Diseases

BrainStorm Call for Updated FDA Approaches for Rare Disease Therapies in Journal Article

Rallybio Agrees to Reverse Merger

Servier to Acquire Edgewise Muscular Dystrophy Business for up to $2.65 Billion

What’s Happening

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

RARE Advocacy Exchange Session 6 : Grieving a RARE 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