RARE Daily landing - Global Genes

Learn

RARE Daily: the official Global Genes blog

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

Latest

Drug Development

Genentech’s C5 Inhibitor Meets Co-Primary Endpoints in Phase 3 Study in PNH

Rare Daily Staff Roche’s Genentech said its C5 complement inhibitor crovalimab met its co-primary endpoints in a […]

Drug Development

Janssen Reports Positive Phase 2 Results for Nipocalimab in Pregnant Women at High Risk for Severe Hemolytic Disease of the Fetus and Newborn

Rare Daily Staff The Janssen Pharmaceutical Companies of Johnson & Johnson reported positive topline results from the […]

Drug Development

FDA Grants Fast Track Designation to Endogena’s EA-2353 for the Treatment of Retinitis Pigmentosa

Rare Daily Staff The U.S. Food and Drug Administration granted Fast Track designation to Endogena Therapeutics’ EA-2353 […]

Drug Development

FDA Approves Takeda’s Takhzyro to Prevent Hereditary Angioedema Attacks in Children 2 Years of Age and Older

Rare Daily Staff The U.S. Food and Drug Administration has approved Takeda’s supplemental Biologics License Application for […]

Diagnosis

Use of AI Platform Improves Diagnostic Rate of Rare Diseases in Japan

Rare Daily Staff The National Center for Child Health and Development in Japan and the U.S.-based OmniTier […]

Finance

CIRM Awards $1.5 Million to Rubedo Life Sciences for IPF Program

Rare Daily Staff The California Institute for Regenerative Medicine awarded Rubedo Life Sciences a $1.5 million grant […]

Drug Development

Certa Reports Positive Phase 2 Results in Scleroderma

Rare Daily Staff Certa Therapeutics reported positive results from a phase 2 study of FT011, its experimental […]

Vyant Bio Begins Cash Preservation Plan, Potentially Winding Down Operations

Rare Daily Staff Vyant Bio, a company focused on neurodevelopmental and neurodegenerative disorders, said its board of […]

Drug Development

FDA Places Hold on 4D Molecular Gene Therapy for Fabry Disease

Rare Daily Staff 4D Molecular said the U.S. Food and Drug Administration placed a clinical hold on […]

Drug Development

RARE Daily

FDA Grants Fast Track Designation to Reneo’s Mavodelpar in a Genotype of LC-FAOD

Rare Daily Staff The U.S. Food and Drug Administration has granted Fast Track designation to Reneo Pharmaceuticals’ […]

RARE Daily

FDA Grants Breakthrough Therapy Designation to Abbisko’s Pimicotinib

Rare Daily Staff The U.S. Food and Drug Administration granted Abbisko Therapeutics Breakthrough Therapy designation for pimicotinib, […]

RARE Daily

FDA Grants Fast Track Designation to Syros’ Tamibarotene for Treatment Group of Rare Cancers

Rare Daily Staff The U.S. Food and Drug Administration granted Fast Track designation to Syros Pharmaceuticals’ tamibarotene […]

RARE Daily

Magenta Pauses Dose Escalation Trial of Conditioning Agent Following Patient Death

Rare Daily Staff Magenta Therapeutics said it voluntarily paused a dose escalation trial of its conditioning agent […]

RARE Daily

Capricor Reports Positive 18-Month Results from Ongoing DMD Study

Rare Daily Staff Capricor Therapeutics said 18-month results from its ongoing HOPE-2 open label extension study in […]

RARE Daily

Cadrenal Granted FDA Fast Track Designation for Tecarfarin for Prevention of Blood Clots

Rare Daily Staff The U.S. Food and Drug Administration granted a Fast Track designation to Cadrenal Therapeutics’ […]

RARE Daily

Pliant Reports Positive Data from Phase 2a Trial of Bexotegrast in Patients with IPF

Rare Daily Staff Pliant Therapeutics reported 12-week interim data from a phase 2a clinical trial of bexotegrast […]

RARE Daily

FDA Grants Blueprint Medicines Priority Review for Ayvakit in Expanded Indication

Rare Daily Staff The U.S. Food and Drug Administration granted priority review to Blueprint Medicines’ Ayvakit for […]

RARE Daily

FDA Lifts Hold on Astellas’ Pompe Gene Therapy

Rare Daily Staff

The U.S. Food and Drug Administration lifted the clinical hold it placed on Astellas Pharma’s FORTIS phase […]

Finance

RARE Daily

Minoryx Raises $55 Million to Support MAA and Launch for X-ALD Therapy

Rare Daily Staff Spanish rare CNS disease focused biotech Minoryx Therapeutics raised $55 million (€51 million) to […]

RARE Daily

Dutch Biotech Xinvento Raises Seed Round to Develop Treatment for Congenital Hyperinsulinism

Rare Daily Staff Dutch biotech Xinvento said it completed a seed funding round, which it will use […]

RARE Daily

Heidelberg Pharma Secures Financing Commitment from its Main Shareholder, Doses First Patient in Study

Rare Daily Staff Heidelberg Pharma said it has secured a commitment of $40.9 million (€36 million) in […]

RARE Daily

Star Emerges from Stealth with Plans to Be “Innovation Engine” for Rare Disease Therapies, Spins out Electra

Rare Daily Staff Star Therapeutics has emerged from stealth mode with $100 million in funding today to […]

RARE Daily

SpliceBio Raises $57 Million to Advance Protein Splicing Platform and Expand Gene Therapy Pipeline

Rare Daily Staff Spanish biotech SpliceBio completed an oversubscribed $57 million (€50 million) series A financing to […]

RARE Daily

NMD Pharma Raises $39.7 Million to Advance Pipeline Targeting Rare Neuromuscular Disorders

Rare Daily Staff Danish biotech NMD Pharma, a clinical stage company developing first in class, small molecule […]

RARE Daily

Albireo Study Highlighting the Caregiver Impact of Rare Children’s Liver Disease

Rare Daily Staff Albireo Pharma said its PICTURE study evaluating and quantifying the impact that progressive familial […]

Magenta Therapeutics to Explore Strategic Alternatives

Rare Daily Staff Magenta Therapeutics said it has halted further development of its programs and will conduct […]

FDA

FDA Tells Taysha It Should Increase the Number of Patients in GAN Gene Therapy Study

Rare Daily Staff Taysha Gene Therapies, in providing an update on its program for giant axonal neuropathy, […]

Strategy

Quince Therapeutics Prioritizes Pipeline, Slashes Workforce

Rare Daily Staff Less than a year after ditching a failed Alzheimer’s program and focusing on a […]

Regulatory

EMA Amends Policy of Orphan Designations of Therapies for Inherited Retinal Dystrophies

Rare Daily Staff The European Medicines Agency’s Committee on Orphan Medicinal Products said it has amended its […]

Diagnosis

Novo Nordisk to Acquire Forma, Expanding Presence in Sickle Cell Disease and Rare Blood Disorders

Rare Daily Staff Novo Nordisk and Forma Therapeutics said that they have entered into a definitive agreement […]

Drug Development

FDA Approves Imbruvica for Treatment of Children with Chronic Graft Versus Host Disease

Rare Daily Staff The U.S. Food and Drug Administration approved the use of AbbVie’s Imbruvica for the […]

What’s Happening

Diagnosis

Rare Diseases: Reconciling Uncertainty and Family; Leah Sarah Peer, Medical Student and third place winner of 2022 Cox Scholarship

Talking Can Be Excellent Medicine; Jenna Kwiecinski, Medical Student and second place winner of 2022 Cox Scholarship

Medical Dignity at Every Turn: Insights Learned from Rare Disease; Raymond So, Medical Student and first place winner of 2022 Cox Scholarship

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

Why a Topical Cannabidiol Gel May Help Treat the Behavioral Symptoms of Fragile X

An Ultra-Rare Disease Drug Developer Tries to Navigate Regulatory Uncertainty

A Down Year for Drug Approvals, But Notable for Its Standouts

FDA Won’t Change Approach to Orphan Exclusivity after Court Ruling

Latest

Genentech’s C5 Inhibitor Meets Co-Primary Endpoints in Phase 3 Study in PNH

Janssen Reports Positive Phase 2 Results for Nipocalimab in Pregnant Women at High Risk for Severe Hemolytic Disease of the Fetus and Newborn

FDA Grants Fast Track Designation to Endogena’s EA-2353 for the Treatment of Retinitis Pigmentosa

FDA Approves Takeda’s Takhzyro to Prevent Hereditary Angioedema Attacks in Children 2 Years of Age and Older

Use of AI Platform Improves Diagnostic Rate of Rare Diseases in Japan

CIRM Awards $1.5 Million to Rubedo Life Sciences for IPF Program

Certa Reports Positive Phase 2 Results in Scleroderma

Vyant Bio Begins Cash Preservation Plan, Potentially Winding Down Operations

FDA Places Hold on 4D Molecular Gene Therapy for Fabry Disease

Drug Development

FDA Grants Fast Track Designation to Reneo’s Mavodelpar in a Genotype of LC-FAOD

FDA Grants Breakthrough Therapy Designation to Abbisko’s Pimicotinib

FDA Grants Fast Track Designation to Syros’ Tamibarotene for Treatment Group of Rare Cancers

Magenta Pauses Dose Escalation Trial of Conditioning Agent Following Patient Death

Capricor Reports Positive 18-Month Results from Ongoing DMD Study

Cadrenal Granted FDA Fast Track Designation for Tecarfarin for Prevention of Blood Clots

Pliant Reports Positive Data from Phase 2a Trial of Bexotegrast in Patients with IPF

FDA Grants Blueprint Medicines Priority Review for Ayvakit in Expanded Indication

FDA Lifts Hold on Astellas’ Pompe Gene TherapyRare Daily Staff

Finance

Minoryx Raises $55 Million to Support MAA and Launch for X-ALD Therapy

Dutch Biotech Xinvento Raises Seed Round to Develop Treatment for Congenital Hyperinsulinism

Heidelberg Pharma Secures Financing Commitment from its Main Shareholder, Doses First Patient in Study

Star Emerges from Stealth with Plans to Be “Innovation Engine” for Rare Disease Therapies, Spins out Electra

SpliceBio Raises $57 Million to Advance Protein Splicing Platform and Expand Gene Therapy Pipeline

NMD Pharma Raises $39.7 Million to Advance Pipeline Targeting Rare Neuromuscular Disorders

Albireo Study Highlighting the Caregiver Impact of Rare Children’s Liver Disease

More Stories

Magenta Therapeutics to Explore Strategic Alternatives

FDA Tells Taysha It Should Increase the Number of Patients in GAN Gene Therapy Study

Quince Therapeutics Prioritizes Pipeline, Slashes Workforce

EMA Amends Policy of Orphan Designations of Therapies for Inherited Retinal Dystrophies

Novo Nordisk to Acquire Forma, Expanding Presence in Sickle Cell Disease and Rare Blood Disorders

FDA Approves Imbruvica for Treatment of Children with Chronic Graft Versus Host Disease

What’s Happening

What I Learned About Intrahepatic Cholestasis With Both of My Pregnancies

A New, More Inclusive Membership Model for Global Advocacy Alliance Members

RARE Global Advocacy Alliance Member Spotlight: Sonny Mullen

Rare Diseases: Reconciling Uncertainty and Family; Leah Sarah Peer, Medical Student and third place winner of 2022 Cox Scholarship

Talking Can Be Excellent Medicine; Jenna Kwiecinski, Medical Student and second place winner of 2022 Cox Scholarship

Medical Dignity at Every Turn: Insights Learned from Rare Disease; Raymond So, Medical Student and first place winner of 2022 Cox Scholarship

RARE Global Advocacy Alliance Member Spotlight: Giovi Moschoudis

RARE Global Advocacy Alliance Member Spotlight: David Simon Ross

What We Learned at RARE Health Equity Summit

More Resources: Dictionary of Rare Diseases

FDA Lifts Hold on Astellas’ Pompe Gene Therapy

Rare Daily Staff