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RARE Daily: the official Global Genes blog

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

Drug Development

Rinascera Launches with Focus on Rare Genetic Skin Diseases

Rare Daily Staff Boston-based Rinascera Therapeutics, a company with two experimental treatments aimed at addressing rare genetic […]

Drug Development

Q32 Reports Positive Mid-Stage Results for Alopecia Areata Therapy

Rare Daily Staff Q32 Bio reported positive mid-stage clinical trial results for its experimental drug bempikibart in […]

Drug Development

FDA Grants Niagen Rare Pediatric Disease Designation for Experimental A-T Therapy

Rare Daily Staff The U.S. Food and Drug Administration has granted Niagen Bioscience Rare Pediatric Disease (RPD) […]

Drug Development

Ipsen Reports Positive Late-Stage Results for PBC Therapy

Rare Daily Staff Ipsen reported that its liver disease drug Iqirvo significantly improved a key marker of […]

Drug Development

FDA Grants Vanda Rare Pediatric Disease Designation for CMT Therapy

Rare Daily Staff The U.S. Food and Drug Administration has granted Vanda Pharmaceuticals Rare Pediatric Disease designation […]

Drug Development

FDA Approves Vera’s IgAN Therapy

Rare Daily Staff The U.S. Food and Drug Administration has granted accelerated approval to Vera Therapeutics’ Trutakna […]

Drug Development

FDA Green Lights Human Clinical Trial of BGTC Developed Gene Therapy for MSD

Rare Daily Staff The U.S. Food and Drug Administration has cleared the way for human clinical trials […]

Drug Development

FDA Approves Expaneded Use of Vertex’s SCD Gene Therapy

Rare Daily Staff The U.S. Food and Drug Administration has approved an expanded label for Vertex Pharmaceuticals […]

Drug Development

BioCryst to Scrap Internal Discovery

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

Finance

RARE Daily

Regenxbio Prices $100 Million Public Offering to Bolster Capital Position

Rare Daily Staff Regenxbio has priced an underwritten public offering expected to generate approximately $100 million in […]

RARE Daily

Mighty Therapeutics Secures Financing for Up to $150 Million

Rare Daily Staff Mighty Therapeutics, formerly known as Stealth BioTherapeutics, said it has secured up to $150 […]

RARE Daily

ARPA-H Awards up to $160 Million to Advance Personalized Curative Medicines for Rare Genetic Diseases

Rare Daily Staff The Advanced Research Projects Agency for Health has awarded up to $160 million in […]

RARE Daily

Developer Makes $10 Million Gift to Penn for Preclinical Development of RVCL Therapy

Rare Daily Staff The Clayco Foundation, the philanthropic arm of the Chicago-based real estate development and construction […]

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 […]

Newly Launched Raremap Helps Rare Disease Community Navigate UK Ecosystem

Rare Daily Staff LifeArc, the self-funded UK organization focused on addressing high-risk and complex medical challenges, has […]

Deals

Vertex Expands Rare Disease Portfolio with Deal to Acquire Crinetics for $10 Billion

Rare Daily Staff Vertex Pharmaceuticals said it will acquire Crinetics Pharmaceuticals in a deal valued at about […]

Deals

United Therapeutics Expands Regen Med Platform with $300 Million Acquisition of Thymus Therapy

Rare Daily Staff United Therapeutics is deepening its push into regenerative medicine and immune system engineering with […]

Deals

Ipsen Expands Rare Disease Portfolio with $797 Million Acquisition of Memo

Rare Daily Staff Paris-based Ipsen said it will acquire the Swiss biotech Memo Therapeutics in a deal […]

Deals

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 […]

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

A One-Time Cell Therapy to Reset the Immune System in Autoimmune Diseases

Building the Infrastructure for Made to Order Gene Therapies

Rewiring the Rare Disease Diagnostic Odyssey

Emerging from a Life in the Shadows

Drug Development

Rinascera Launches with Focus on Rare Genetic Skin Diseases

Q32 Reports Positive Mid-Stage Results for Alopecia Areata Therapy

FDA Grants Niagen Rare Pediatric Disease Designation for Experimental A-T Therapy

Ipsen Reports Positive Late-Stage Results for PBC Therapy

FDA Grants Vanda Rare Pediatric Disease Designation for CMT Therapy

FDA Approves Vera’s IgAN Therapy

FDA Green Lights Human Clinical Trial of BGTC Developed Gene Therapy for MSD

FDA Approves Expaneded Use of Vertex’s SCD Gene Therapy

BioCryst to Scrap Internal Discovery

Finance

Regenxbio Prices $100 Million Public Offering to Bolster Capital Position

Mighty Therapeutics Secures Financing for Up to $150 Million

ARPA-H Awards up to $160 Million to Advance Personalized Curative Medicines for Rare Genetic Diseases

Developer Makes $10 Million Gift to Penn for Preclinical Development of RVCL Therapy

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

More Stories

Newly Launched Raremap Helps Rare Disease Community Navigate UK Ecosystem

Vertex Expands Rare Disease Portfolio with Deal to Acquire Crinetics for $10 Billion

United Therapeutics Expands Regen Med Platform with $300 Million Acquisition of Thymus Therapy

Ipsen Expands Rare Disease Portfolio with $797 Million Acquisition of Memo

Ionis Enters Global Licensing Deal with Recordati for Alexander disease ASO

Incyte to Acquire Vega for up to $2 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