RARE Daily landing

Learn

RARE Daily: the official Global Genes blog

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

Drug Development

FDA Issues Safety Alert on Amgen Vasculitis Drug

Rare Daily Staff The Food and Drug Administration is warning that Amgen’s rare‑disease drug Tavneos can cause […]

Drug Development

Viridian Drug Meets Main Goals in TED Trial, but Shares Sink on Results

Rare Daily Staff Viridian Therapeutics said its experimental drug elegrobart met the main goals of a late-stage […]

Drug Development

FDA Grants Accelerated Approval to Rocket’s Gene Therapy for LAD-1

Rare Daily Staff The U.S. Food and Drug Administration has granted accelerated approval to Rocket Pharmaceuticals’ gene […]

Drug Development

Karyopharm Reports Mixed Study Result, Raises $30 Million

Rare Daily Staff Karyopharm Therapeutics reported mixed but promising results from a clinical trial in myelofibrosis, a […]

Drug Development

FDA Grants Accelerated Approval to Denali’s Hunter Syndrome ERT

Rare Daily Staff The U.S. Food and Drug Administration granted accelerated approval to Denali Therapeutics’ Avlayah, a […]

Drug Development

CIRM Approves $100 Million for Platform-Based Gene Therapies for Rare Diseases

Rare Daily Staff The California Institute for Regenerative Medicine is launching a new funding initiative aimed at […]

Drug Development

Alfasigma wins FDA approval for liver disease itch treatment

Rare Daily Staff The U.S. Food and Drug Administration has approved Alfasigma’s linerixibat to treat severe itching […]

Drug Development

FDA Expands Approval of Rhythm’s Imcivree

Rare Daily Staff The U.S. Food and Drug Administration has granted expanded approval to Rhythm Pharmaceuticals’ obesity […]

Drug Development

FDA Grants Breakthrough Therapy for Sanofi’s Type 3 Gaucher Disease Drug

Rare Daily Staff The U.S. Food and Drug Administration granted Breakthrough Therapy designation to Sanofi’s experimental drug […]

Finance

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 […]

RARE Daily

ARPA-H to Provide Every Cure up to $76 Million for AI-Drive Drug Repurposing

Rare Daily Staff The Advanced Research Projects Agency for Health (ARPA-H) will provide up to $76 million […]

RARE Daily

Cyprium to Sell PRV for $205 Million

Rare Daily Staff Cyprium Therapeutics, a majority-owned subsidiary of Fortress Biotech, agreed to sell its Rare Pediatric […]

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 […]

Eli Lilly to acquire Centessa in $6.3 Billion to Expand Sleep Disorder Pipeline

Rare Daily Staff Eli Lilly agreed to acquire Centessa Pharmaceuticals in a deal valued at about $6.3 […]

Deals

Biogen to Acquire Apellis is $5.6 Billion Deal

Rare Daily Staff Biogen has agreed to acquire Apellis Pharmaceuticals in a $5.6 billion cash deal that will add […]

Diagnosis

AI System Has Potential to Shorten Rare Disease Diagnostic Odyssey

Rare Daily Staff A new artificial intelligence system could help shorten the diagnostic odyssey families face for […]

Deals

Sentynl Therapeutics to License Experimental Progeria Drug

Rare Daily Staff Sentynl Therapeutics has entered into an agreement to license an experimental drug for Hutchinson-Gilford […]

Diagnosis

Natera Launches New WGS Test

Natera said it has launched a new genomic testing platform designed to help diagnose rare diseases faster. […]

Regulatory

Prasad to Step Down at FDA’s CBER

Rare Daily Staff Vinay Prasad, the controversial head of the U.S. Food and Drug Administration’s Center for […]

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

A Mother’s Journey to Rewrite a Neurodevelopmental Disorder

How a Foundation Built Its Own Drug Program for an Ultra-Rare Disease

Reopening the Developmental Window in Rett Syndrome with a Gene Therapy

From Bloodletting to Breakthroughs in PV

Drug Development

FDA Issues Safety Alert on Amgen Vasculitis Drug

Viridian Drug Meets Main Goals in TED Trial, but Shares Sink on Results

FDA Grants Accelerated Approval to Rocket’s Gene Therapy for LAD-1

Karyopharm Reports Mixed Study Result, Raises $30 Million

FDA Grants Accelerated Approval to Denali’s Hunter Syndrome ERT

CIRM Approves $100 Million for Platform-Based Gene Therapies for Rare Diseases

Alfasigma wins FDA approval for liver disease itch treatment

FDA Expands Approval of Rhythm’s Imcivree

FDA Grants Breakthrough Therapy for Sanofi’s Type 3 Gaucher Disease Drug

Finance

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

ARPA-H to Provide Every Cure up to $76 Million for AI-Drive Drug Repurposing

Cyprium to Sell PRV for $205 Million

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

More Stories

Eli Lilly to acquire Centessa in $6.3 Billion to Expand Sleep Disorder Pipeline

Biogen to Acquire Apellis is $5.6 Billion Deal

AI System Has Potential to Shorten Rare Disease Diagnostic Odyssey

Sentynl Therapeutics to License Experimental Progeria Drug

Natera Launches New WGS Test

Prasad to Step Down at FDA’s CBER

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