RARE Daily landing - Global Genes

Learn

RARE Daily: the official Global Genes blog

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

Drug Development

FDA Grants Fast Track Designation to Arcturus’ mRNA Therapeutic Candidate for OTC

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

Drug Development

FDA Grants Fast Track Designation for Inhibrx Treatment of Alpha-1 Antitrypsin Deficiency

Rare Daily Staff The U.S. Food and Drug Administration granted Fast Track designation to Inhibrx’ INBRX-101, an […]

Drug Development

EMA Grants PRIME Designation to Rocket’s Gene Therapy for Danon Disease

Rare Daily Staff The European Medicines Agency granted Priority Medicines (PRIME) designation to Rocket Pharmaceutical’s RP-A501, the […]

Drug Development

Bringing Precision to CRISPR-Based Genome Editing

CRISPR genome editing has the potential to revolutionize the treatment of diseases, but the imprecision of its […]

Drug Development

FDA Grants Priority Review to BMS for Treatment for Rare Form of NSCLC

Rare Daily Staff The U.S. Food and Drug Administration granted priority review to Bristol Myers Squibb’s repotrectinib, […]

Drug Development

Pfizer Reports Positive Marstacimab Results from Pivotal Phase 3 Hemophilia A and B Trial

Rare Daily Staff Pfizer reported that the pivotal phase 3 BASIS clinical trial evaluating marstacimab has met […]

Drug Development

FDA Places Clinical Hold on PepGen’s Application to Initiate a Phase 1 Study in DM1

Rare Daily Staff The U.S. Food and Drug Administration notified PepGen’s that it was placing a clinical […]

Drug Development

Verastem Reports Positive Results in Updated Phase 2 Results of Experimental Therapy in Rare Form of Ovarian Cancer

Rare Daily Staff Verastem Oncology reported updated data from Part A of the ongoing registration-directed RAMP 201 […]

Drug Development

EMA Recommends Revocation of Authorization for Sickle Cell Disease Medicine Adakveo

EMA Recommends Revocation of Authorization for Sickle Cell Disease Medicine Adakveo Rare Daily Staff The European Medicines […]

Finance

RARE Daily

Belite Bio Raises $30 Million in Public Offering

Rare Daily Staff Belite Bio, a clinical stage company focused on advancing novel therapeutics targeting retinal degenerative […]

RARE Daily

ElevateBio Raises $401 Million to Further Accelerate Growth

Rare Daily Staff ElevateBio, a technology-driven company focused on powering the creation of life-transforming cell and gene […]

RARE Daily

Renagade Therapeutics Launches with $300 Million to Expand Reach of RNA Therapies

Rare Daily Staff Renagade Therapeutics, which is seeking to expand the utility of RNA therapeutics by delivering […]

RARE Daily

Scarlet Therapeutics Raises Seed Financing to Advance Development of Therapeutic Red Blood Cell Platform

Rare Daily Staff Scarlet Therapeutics said it has raised seed funding from Science Creates Ventures and Meltwind […]

RARE Daily

Cabaletta Bio Raises $100 Million to Advance Cell Therapy Pipeline for Autoimmune Diseases

Rare Daily Staff Cabaletta Bio, a clinical-stage biotechnology company focused on developing and launching the first curative […]

RARE Daily

Protalix BioTherapeutics Gets $20 Million Milestone Payment from Chiesi

Rare Daily Staff Protalix BioTherapeutics said it is eligible to receive $20 million from its commercial partner […]

RARE Daily

April a Quiet Month for Rare Disease Therapeutics Financing and Dealmaking

April was a quiet month for rare disease focused drug developers with few financings and no significant […]

RARE Daily

X4 Pharma Raises $65 Million Private Placement to Advance Potential WHIM Treatment

Rare Daily Staff X4 Pharmaceuticals, a company focused on the discovery and development of novel small molecule […]

RARE Daily

Ensoma Raises $50 Million to Advance Genomic Medicine

Rare Daily Staff Ensoma, a genomic medicines company developing in vivo treatments that precisely engineer any cell […]

Pharming Sells Priority Review Voucher for Pre-Set Price of $22.1 Million

Rare Daily Staff Pharming Group said it has entered into a definitive agreement with Novartis Pharma to […]

Deals

BioLight Enters Agreement with Alexion to Explore Technology To Diagnose Retinal Diseases

Rare Daily Staff Israeli ocular disease focused biotech BioLight signed a research collaboration agreement with Alexion, AstraZeneca […]

Deals

Abiogen Acquires Effrx

Rare Daily Staff Italy-based Abiogen Pharma, a company focused on osteoarticular and bone metabolism diseases, has acquired […]

Strategy

Idogen Shareholders to Vote on Liquidation if Company Unable to Find Agreement on Reverse Merger

Rare Daily Staff The Swedish biotechnology company Idogen, which is developing cell therapies to counter attacks by […]

Deals

Novo Nordisk and Life Edit Enter Gene Editing Collaboration in Rare and Cardiometabolic Diseases

Rare Daily Staff Novo Nordisk and Life Edit Therapeutics, an ElevateBio subsidiary, have entered into a research […]

Diagnosis

Rare Diseases May Be More Common than Thought

Rare Daily Staff People who have a single inherited defective gene from one parent have often been […]

What’s Happening

Drug Development

The RARE Research Roadmap – 2023 RARE Drug Development Symposium

Diagnosis

Mental Health in Rare Disease: Taking Care of Your Mind Is Taking Care of Your Body

May is Mental Health Awareness Month. Taking care of your mental health is key, whether you are […]

Drug Development

How Can the FDA Improve Processes for Rare Diseases? – 2023 RARE Drug Development Symposium

Drug Development

The RARE Research Roadmap – 2023 RARE Drug Development Symposium

Drug Development

The Regulatory Landscape: Pathways, End Points, and Clinical Trials – 2023 RARE Drug Development Symposium

Drug Development

Intellectual Property: Balancing Stakeholders’ Rights – 2023 RARE Drug Development Symposium

Drug Development

New Technologies in Early Stage Research – 2023 RARE Drug Development Symposium

Drug Development

Understanding Data for Basic Research – 2023 RARE Drug Development Symposium

Drug Development

Charting the Path to Treatments – 2023 RARE Drug Development Symposium

Reports

DEI

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

Bringing Precision to CRISPR-Based Genome Editing

Capturing Patients’ Experience in Their Daily Lives

Rare Diseases May Be More Common than Thought

From One to Many

Drug Development

FDA Grants Fast Track Designation to Arcturus’ mRNA Therapeutic Candidate for OTC

FDA Grants Fast Track Designation for Inhibrx Treatment of Alpha-1 Antitrypsin Deficiency

EMA Grants PRIME Designation to Rocket’s Gene Therapy for Danon Disease

Bringing Precision to CRISPR-Based Genome Editing

FDA Grants Priority Review to BMS for Treatment for Rare Form of NSCLC

Pfizer Reports Positive Marstacimab Results from Pivotal Phase 3 Hemophilia A and B Trial

FDA Places Clinical Hold on PepGen’s Application to Initiate a Phase 1 Study in DM1

Verastem Reports Positive Results in Updated Phase 2 Results of Experimental Therapy in Rare Form of Ovarian Cancer

EMA Recommends Revocation of Authorization for Sickle Cell Disease Medicine Adakveo

Finance

Belite Bio Raises $30 Million in Public Offering

ElevateBio Raises $401 Million to Further Accelerate Growth

Renagade Therapeutics Launches with $300 Million to Expand Reach of RNA Therapies

Scarlet Therapeutics Raises Seed Financing to Advance Development of Therapeutic Red Blood Cell Platform

Cabaletta Bio Raises $100 Million to Advance Cell Therapy Pipeline for Autoimmune Diseases

Protalix BioTherapeutics Gets $20 Million Milestone Payment from Chiesi

April a Quiet Month for Rare Disease Therapeutics Financing and Dealmaking

X4 Pharma Raises $65 Million Private Placement to Advance Potential WHIM Treatment

Ensoma Raises $50 Million to Advance Genomic Medicine

More Stories

Pharming Sells Priority Review Voucher for Pre-Set Price of $22.1 Million

BioLight Enters Agreement with Alexion to Explore Technology To Diagnose Retinal Diseases

Abiogen Acquires Effrx

Idogen Shareholders to Vote on Liquidation if Company Unable to Find Agreement on Reverse Merger

Novo Nordisk and Life Edit Enter Gene Editing Collaboration in Rare and Cardiometabolic Diseases

Rare Diseases May Be More Common than Thought

What’s Happening

The RARE Research Roadmap – 2023 RARE Drug Development Symposium

Mental Health in Rare Disease: Taking Care of Your Mind Is Taking Care of Your Body

How Can the FDA Improve Processes for Rare Diseases? – 2023 RARE Drug Development Symposium

The RARE Research Roadmap – 2023 RARE Drug Development Symposium

The Regulatory Landscape: Pathways, End Points, and Clinical Trials – 2023 RARE Drug Development Symposium

Intellectual Property: Balancing Stakeholders’ Rights – 2023 RARE Drug Development Symposium

New Technologies in Early Stage Research – 2023 RARE Drug Development Symposium

Understanding Data for Basic Research – 2023 RARE Drug Development Symposium

Charting the Path to Treatments – 2023 RARE Drug Development Symposium

Reports

Global Genes 2022 RARE Impact Grants Report

Global Genes Impact Report: Building on a Successful 2022

RARE-X Insights & Recommendations for Organizational DEI Strategies Study

More Resources: Dictionary of Rare Diseases