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RARE Daily: the official Global Genes blog

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

Drug Development

MDA Launches Program to Develop Gene Therapies for Ultra-Rare Neuromuscular Diseases

Rare Daily Staff The Muscular Dystrophy Association said it has launched the MDA Kickstart program for Ultra-Rare […]

Drug Development

FDA Grants Full Approval to Travere for Filspari in IgA Nephropathy

Rare Daily Staff The U.S. Food and Drug Administration granted Travere Therapeutics full approval and expanded use […]

Drug Development

NIMH Awards PsychoGenics $3 million Grant to Use AI Platform for Repurposing

Rare Daily Staff The National Institutes of Health’s National Institute of Mental Health awarded a Fast Track […]

Drug Development

FDA Grants Accelerated Approval to Gilead’s PBC Drug

Rare Daily Staff The U.S. Food and Drug Administration granted accelerated approval to Gilead Sciences’ Livdelzi for […]

Drug Development

FDA Approves Ascendis’ Hypoparathyroidism Drug After Delays

Rare Daily Staff The U.S. Food and Drug Administration approved Ascendis’ Yorvipath for the treatment of adults […]

Drug Development

FDA Approves First Cell Therapy for Solid Tumor

Rare Daily Staff The U.S. Food and Drug Administration granted accelerated approval to Adaptimmune’s Tecelra for a […]

Drug Development

James Wilson Departs Penn to Launch to Spin Out Penn Programs into Two New Gene Therapy Companies

Rare Daily Staff Gene therapy pioneer James Wilson is stepping down from his post as director of […]

Drug Development

FOXG1 Research Foundation Enters Collaboration with CRL to Advance Gene Therapy

Rare Daily Staff The FOXG1 Research Foundation has entered into a collaboration with Charles River Laboratories International […]

Drug Development

Advocates Increase Focus on PRV as Deadline to Reauthorize Approaches

Back in February, rare disease organizations applauded the introduction of legislation to reauthorize the Rare Pediatric Priority […]

Finance

RARE Daily

Tern Therapeutics Launches with $15 Million and Pipeline in CLN2 Batten Disease

Rare Daily Staff Tern Therapeutics launched with a $15 million financing and a global licensing agreement with […]

RARE Daily

Taysha Raises $75 Million in Public Offering to Advance Gene Therapy for Rett Syndrome

Rare Daily Staff One week after reporting positive data from phase 1/2 studies of its experimental gene […]

RARE Daily

Exsilio Therapeutics Launches with $82 Million to Develop Redosable Genomic Medicines

Rare Daily Staff Exsilio Therapeutics emerged from stealth with $82 million in series A financing to advance […]

RARE Daily

Entrada Raises $100 Million Registered Direct Offering

Rare Daily Staff Entrada Therapeutics entered into a securities purchase agreement with a group of investors in […]

RARE Daily

Rare Daily Staff NephroDI Therapeutics raised $2 million from Sound Bioventures that will be used to advance […]

RARE Daily

iOnctura Raises $86 Million to Advance Rare Cancer Pipeline

Rare Daily Staff Swiss biotech iOnctura closed an $86 million (€80 million) series B financing to advance […]

RARE Daily

ALS Association Gives ProJenX $1 Million Clinical Trial Award

Rare Daily Staff ProJenX, the biotech created out of a collaboration between Columbia University and Project ALS, […]

RARE Daily

Four Biotechs Raise Almost $1 Billion to Advance Rare Disease Pipelines

Rare Daily Staff Four public companies with a rare disease therapeutics focus tapped the market for almost […]

RARE Daily

Foundation for Fighting Blindness Provides Opus Genetics $1.7 Million in Funding

Rare Daily Staff The Foundation for Fighting Blindness provided $1.7 million in project-based funding to Opus Genetics […]

Chiesi Global Rare Diseases Launches Grant Initiative to Support Research into LSDs

Rare Daily Staff Chiesi Global Rare Diseases has launched Find For Rare, a new grant initiative to provide […]

Living with a RARE Disease

Study Explores Quality of Life Effects of Healthcare Access on Caregivers

Rare Daily Staff New strategies are needed to better support rare disease caregivers and improve health outcomes […]

Deals

BridgeBio Spins-Out Set of Assets into Newly Formed GondolaBio

Rare Daily Staff BridgeBio said in a filing with the U.S. Securities and Exchange Commission that it […]

Diagnosis

New Test Uses RNA to Expand Diagnostic Yield of Exome Sequencing

Rare Daily Staff Clinical genomics testing company Ambry Genetics has released ExomeReveal, a new multiomic exome sequencing […]

Regulatory

Patient and Industry Groups Warn New EU Rules Will Reduce New Rare Disease Treatments

Rare Daily Staff New guidelines set to take effect in January in the European Union threaten to […]

Living with a RARE Disease

Colin Farrell Forms Foundation to Address Needs of Adults with Intellectual Disabilities

Rare Daily Staff Actor Colin Farrell, whose 20-year-old son James has the rare neurological disorder Angelman syndrome, […]

What’s Happening

Diagnosis

Caregiver Support Program: Carrie Borrello

In 2024, Global Genes partnered with Brad Thompson, M.A., NCC, LPC-S to host a caregivers support program […]

Healthcare

First Definitive European Clinical Guidelines for BHD Diagnosis & Management

Global Genes asked Global Advocacy Alliance members to share their successes – whether it is meeting an organizational goal, kicking […]

Living with a RARE Disease

Maurya’s Rare Odyssey – a RAREly Told Stories film about Hereditary Spastic Paraplegia SPG4

In February of 2024 Global Genes together with The Disorder Channel presented a workshop, RAREly Told Stories, […]

Diagnosis

Beginner’s Guide to Rare Disease, Jessica’s Version

When we asked Jessica Patay for some resources to help guide those just beginning to face a […]

Living with a RARE Disease

The Beginner’s Guide to Rare Disease

If you were just diagnosed with a rare disease or you are feeling you may remain undiagnosed […]

Living with a RARE Disease

TBX4 Family Film – A RAREly Told Stories Film

In February of 2024 Global Genes together with The Disorder Channel presented a workshop, RAREly Told Stories, […]

Living with a RARE Disease

The yellow brick road feels a bit different for rare disease families.

There’s No Place Like Hope. There’s No Place Like Hope. You might be right to say we […]

Diagnosis

Global Genes’ Sweet 16th Birthday Success Stories

August 15th, 2024 marks Global Genes’ 16th birthday! Since 2008, our organization has made significant efforts and […]

Jansen’s Journey – A RAREly Told Stories film

In February of 2024 Global Genes together with The Disorder Channel presented a workshop, RAREly Told Stories, […]

Reports

Diagnosis

Continuing Advocacy and Expanding Research Efforts: Global Genes 2023 Impact Report

To our community members: For both the rare disease community and Global Genes, 2023 marked a year […]

DEI

Global Genes 2023 RARE Impact Grants Report

Over the past decade, Global Genes’ RARE Impact Grant Program has provided grants to rare disease patient […]

Diagnosis

NEXT Report 2024: Rewriting the Rules

Over the past year, technological advances in rare disease drug and therapy development, coupled with the tenacity of rare disease patients and advocates, have prevailed despite the challenges of financial difficulties in biopharma. Next-generation patient advocates continue to take an active role in drug development, as outlined in the 2024 NEXT Report.

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.

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RARE Daily: the official Global Genes blog

Featured

Enabling On-Demand Therapies

After Driving an N-of-1 Therapy for Her Son, an Advocate Turns to Helping Others

Putting Rare Disease Organization in the Business of Drug Development

Understanding the Many Questions Gene Therapies Raise

Drug Development

MDA Launches Program to Develop Gene Therapies for Ultra-Rare Neuromuscular Diseases

FDA Grants Full Approval to Travere for Filspari in IgA Nephropathy

NIMH Awards PsychoGenics $3 million Grant to Use AI Platform for Repurposing

FDA Grants Accelerated Approval to Gilead’s PBC Drug

FDA Approves Ascendis’ Hypoparathyroidism Drug After Delays

FDA Approves First Cell Therapy for Solid Tumor

James Wilson Departs Penn to Launch to Spin Out Penn Programs into Two New Gene Therapy Companies

FOXG1 Research Foundation Enters Collaboration with CRL to Advance Gene Therapy

Advocates Increase Focus on PRV as Deadline to Reauthorize Approaches

Finance

Tern Therapeutics Launches with $15 Million and Pipeline in CLN2 Batten Disease

Taysha Raises $75 Million in Public Offering to Advance Gene Therapy for Rett Syndrome

Exsilio Therapeutics Launches with $82 Million to Develop Redosable Genomic Medicines

Entrada Raises $100 Million Registered Direct Offering

iOnctura Raises $86 Million to Advance Rare Cancer Pipeline

ALS Association Gives ProJenX $1 Million Clinical Trial Award

Four Biotechs Raise Almost $1 Billion to Advance Rare Disease Pipelines

Foundation for Fighting Blindness Provides Opus Genetics $1.7 Million in Funding

More Stories

Chiesi Global Rare Diseases Launches Grant Initiative to Support Research into LSDs

Study Explores Quality of Life Effects of Healthcare Access on Caregivers

BridgeBio Spins-Out Set of Assets into Newly Formed GondolaBio

New Test Uses RNA to Expand Diagnostic Yield of Exome Sequencing

Patient and Industry Groups Warn New EU Rules Will Reduce New Rare Disease Treatments

Colin Farrell Forms Foundation to Address Needs of Adults with Intellectual Disabilities

What’s Happening

Caregiver Support Program: Carrie Borrello

First Definitive European Clinical Guidelines for BHD Diagnosis & Management

Maurya’s Rare Odyssey – a RAREly Told Stories film about Hereditary Spastic Paraplegia SPG4

Beginner’s Guide to Rare Disease, Jessica’s Version

The Beginner’s Guide to Rare Disease

TBX4 Family Film – A RAREly Told Stories Film

The yellow brick road feels a bit different for rare disease families.

Global Genes’ Sweet 16th Birthday Success Stories

Jansen’s Journey – A RAREly Told Stories film

Reports

Continuing Advocacy and Expanding Research Efforts: Global Genes 2023 Impact Report

Global Genes 2023 RARE Impact Grants Report

NEXT Report 2024: Rewriting the Rules

More Resources: Dictionary of Rare Diseases