RARE Daily - Global Genes

Articles

Drug Development

July 26, 2021

European Commission Approves Rhythm’s Drug for Rare Obesity Disorders

Drug Development

July 26, 2021

Denali Reports Positive Interim Data from Phase 1/2 Hunter Syndrome Trial

RARECast Podcasts

July 23, 2021

Using Model Systems to Find Drugs to Repurpose for Rare Diseases

Rare Advocate

July 22, 2021

Rare Leader: Cyndi Frank, Co-President and Co-Founder, Gaucher Community Alliance

Finance

July 22, 2021

Hemab Raises $55 Million to Advance Therapeutics for Rare Bleeding Disorders

Healthcare

July 22, 2021

NHS England Unveils New Innovative Medicines Fund to Fast-Track Promising New Drugs

Drug Development

July 22, 2021

Spark Says Ongoing Phase 1/2 Hemophilia A Gene Therapy Data Suggests Durability

Research

July 22, 2021

Researchers Find Immune Component to Rare Neurodegenerative Disease

Drug Development

July 22, 2021

FDA Approves Octapharma’s Therapy for Adult Dermatomyositis

Deals

July 22, 2021

Parexel Partners with Rare Disease Foundation in China

Finance

July 21, 2021

Amylyx Raises $135 Million to Support Development and Potential Launch of ALS Treatment

Drug Development

July 21, 2021

European Commission Approves Bluebird Bio’s Gene Therapy for CALD

Drug Development

July 21, 2021

FDA Approves Albireo’s Bylvay as First Drug for PFIC

Deals

July 21, 2021

Lexeo Acquires Stelios to Expand Pipeline into Rare Cardiac Gene Therapies

Finance

July 21, 2021

Horama Raises $39 Million to Develop its Gene Therapy Pipeline and Changes Name to Coave Therapeutics

Drug Development

July 21, 2021

FDA Grants Breakthrough Therapy Designation to Genentech for Therapy to Treat Myelodysplastic Syndromes

Diagnosis

July 20, 2021

UW Opens Clinic for Patients with Undiagnosed Genetic Diseases

Policy

July 20, 2021

Netherland Fines Rare Disease Drugmaker for Excessive Price

Drug Development

July 19, 2021

Cytokinetics Reports Positive Results from Mid-State Trial of Hypertrophic Cardiomyopathy

Deals

July 19, 2021

GentiBio and Forge Biologics Form GMP Manufacturing Partnership

RARECast Podcasts

July 16, 2021

How a Small and Young Foundation Catalyzed Research into a Rare Disease

Research

July 16, 2021

NIH Funds New Effort to Discover Genetic Causes of Single-Gene Disorders T

Rare Advocate

July 15, 2021

Rare Leader: Chelsey McCarthy, Executive Director, DDX3X Foundation

Finance

July 15, 2021

Shape Therapeutics Raises $112 Million to Advance RNA Technology Platforms

Deals

July 15, 2021

Rady Children’s Institute for Genomic Medicine Enters Research Collaboration with Takeda

Deals

July 14, 2021

AstraZeneca Clears Final Hurdle for Acquisition of Alexion

Drug Development

July 14, 2021

FDA Grants Breakthrough Therapy Designation to Alkeus Stargardt Disease Therapy

Finance

July 13, 2021

Prime Medicine Launches with $315 Million to Deliver on the Promise of Prime Editing

Data Center

July 13, 2021

Rare Disease Drug Developers Bring in $13.7 Billion in New Capital in First Half of 2021

Research

July 13, 2021

Researchers Use Prenatal Editing in Animal Model to Correct Lysosomal Storage Disease

Deals

July 13, 2021

Novo Nordisk Acquires Prothena’s Antibody Targeting ATTR Amyloidosis

Strategy

July 13, 2021

Poxel Shifts Focus to Rare Metabolic Diseases

Drug Development

July 13, 2021

Gene Therapy Reverses Neurological Deficiencies in AADC

RARECast Podcasts

July 9, 2021

Overcoming the Limitations of Conventional Cell Engineering

Research

July 9, 2021

Browsable Resource Linking Rare Protein-Coding Genetic Variants to Human Health and Disease

Drug Development

July 9, 2021

FDA Places Clinical Hold on Sigilon’s Phase 1/2 Study in Hemophilia A

Drug Development

July 9, 2021

BlueBird Bio Resumes Marketing TDT Gene Therapy in Europe

Rare Advocate

July 8, 2021

Rare Leader: Deanna Fournier, Executive Director, Histiocytosis Association

Deals

July 8, 2021

BridgeBio Enters Collaborations with Three Academic Research Institutions

Deals

July 8, 2021

Lilly Enters Collaboration with Verge to Use Its AI to Discover and Develop New Treatments for...

Drug Development

July 8, 2021

FDA Grants Lysogene Fast Track Designation for GM1 Gangliosidosis Gene Therapy

Research

July 8, 2021

CMT Research Foundation Enters Partnership with University of Illinois Chicago

Research

July 7, 2021

Patient Groups Begin Using RARE-X’s Data Collection Platform

Drug Development

July 7, 2021

ChemoCentryx Addresses FDA Advisory Panel Questions in Amendment to Filing on Vasculitis Drug

Deals

July 6, 2021

EC Clears AstraZeneca’s Acquisition of Alexion

Deals

July 6, 2021

Orchard and Pharming Collaborate on Gene Therapy for HAE

Deals

July 6, 2021

Eureka and MSK License Novel Multiple Myeloma Target Domain to Sanofi

Drug Development

July 6, 2021

FDA Grants Priority Review to Levo for Prader-Willi Syndrome Therapy

Deals

July 6, 2021

Abbisko and Sperogenix Partner to Develop ABSK021 for ALS, Other Rare CNS Diseases in Greater China

RARECast Podcasts

July 2, 2021

Experimental Therapy for Rare Endocrine Disorder Offers Hope of Improved Care

Drug Development

July 2, 2021

Arrowhead Pauses RNAi Study in Cystic Fibrosis After Tox Study Signals

Deals

July 2, 2021

GSK Enters into Global Collaboration with Alector to Develop Antibodies for Neurodegenerative Diseases

Rare Advocate

July 1, 2021

Rare Leader: Diane Powell, CEO, The Hypersomnia Foundation

Deals

July 1, 2021

Apellis and Beam Collaborate to Apply Base Editing for Complement-Driven Diseases Drug Discovery

Drug Development

July 1, 2021

FDA Approves Jazz’s Rylaze as Component of Treatment Regimen for Most Common Childhood Cancer

Finance

June 30, 2021

Intellia Raises $600 Million to Advance Gene Editing Therapeutic Pipeline

Finance

June 30, 2021

Sofinnova Invests in Three New Rare Disease Gene Therapy Startups

Healthcare

June 30, 2021

NHS Creates First-of-Its-Kind Rare Disease Network in UK

Drug Development

June 30, 2021

FDA Grants Allogene Fast Track Designation for Multiple Myeloma Therapy

Drug Development

June 30, 2021

GenSight Reports Phase 3 Study Misses Endpoint, but Improves Vision in Patients with Degenerative Eye Disease

Finance

June 30, 2021

Aerovate Raises $121.5 Million in Upsized IPO to Advance PAH Therapy

Drug Development

June 30, 2021

FDA Grants Lexeo Therapeutics Rare Pediatric Disease Designation

Strategy

June 29, 2021

Orphazyme Lays Off Two-Thirds of Staff After FDA Setback

Research

June 29, 2021

Natural History Study Informs Potent Lifesaving Update to Standard of Care for PMM2-CDG Patients

Research

June 29, 2021

Pediatricians Find Cause of Muscle Breakdown in Rare Disease

Drug Development

June 28, 2021

Intellia and Regeneron Report First-Ever Data Supporting In Vivo CRISPR Editing in ATTR Patients

Drug Development

June 28, 2021

EU Approves Roche’s Enspryng as First At-home Subcutaneous Treatment for NMOSD

Drug Development

June 28, 2021

FDA Grants Fast Track Designation to Three Experimental Rare Disease Therapies

RARECast Podcasts

June 25, 2021

Forging Gene Therapy Capacity and a Pipeline at the Same Time

Finance

June 25, 2021

Graphite Bio Raises $238 Million in IPO to Advance Precision Gene Editing Pipeline

Legislation

June 25, 2021

Senators Introduce Legislation to Help Diagnose Children with Rare Diseases

Research

June 25, 2021

International Study of Rare Childhood Cancer Finds Genetic Clues, Potential for Tailored Therapy

Rare Advocate

June 24, 2021

Rare Leader: Kevin Mead, Executive Director of the International Pemphigus & Pemphigoid Foundation

Deals

June 24, 2021

AbbVie Exercises Right to Acquire TeneoOne and Lead Asset Targeting Multiple Myeloma

Deals

June 24, 2021

4D Molecular Reports Trial Data for Rare Eye Disorders and Termination of Roche Agreement

Research

June 24, 2021

Pacific Biosciences and Rady Children’s Collaborate on WGS Study of Rare Diseases

Drug Development

June 24, 2021

Corbus Reports Lenabasum Fails to Meet Primary Endpoint in Late-Stage Trial for Dermatomyositis

Drug Development

June 24, 2021

FDA Approves Santen’s Verkazia to Treat Vernal Keratoconjunctivitis in Children and Adults

Finance

June 23, 2021

Glycomine Raises $35 Million to Advance Novel Treatment for Rare Glycosylation Disease

Drug Development

June 23, 2021

UniQure Reports Positive 52-Week Data from Pivotal Trial of Hemophilia B Gene Therapy

Research

June 22, 2021

MIND Institute Gets $1.25 Million Grant for SYNGAP1 Syndrome

Finance

June 22, 2021

Protagonist Raises $132 Million to Advance Rare Blood Cancer Therapy

Finance

June 22, 2021

Neurona Raises $41.5 Million to Advance Cell Therapies for Epilepsy and Neurological Disorders

Deals

June 22, 2021

Neurelis Acquires Rights to Portfolio of Compounds Targeting Rare CNS Disorder

Deals

June 22, 2021

PerkinElmer to Acquire Viral Vector Gene Delivery Specialist Sirion Biotech

Deals

June 22, 2021

Jasper and Aruvant Collaborate on Treatment for Sickle Cell Disease

Research

June 22, 2021

New UAE Center will Diagnose and Treat Patients with Genetic Diseases

Drug Development

June 21, 2021

EC Expands Approval of Sanofi’s Oral MS Therapy to Include Children and Adolescents

Drug Development

June 21, 2021

Agios Seek Approval for Mitapivat for Rare Inherited Blood Disorder

RARECast Podcasts

June 18, 2021

Using A Natural DNA Repair Process to Improve Genetic Medicines

Drug Development

June 18, 2021

FDA Says It Won’t Approve Orphazyme’s NPC Therapy without Additional Data

Drug Development

June 18, 2021

FDA Grant Applied Therapeutics Fast Track Designation for Experimental Galactosemia Therapy

Drug Development

June 18, 2021

FDA Grants United Therapeutics Priority Review for PAH Drug

Deals

June 18, 2021

Perlara Enters Collaboration with Vivan for Multiple Rare Diseases

Rare Advocate

June 17, 2021

Rare Leader: Fallon Schultz, President and Founder, International FPIES Association

Finance

June 17, 2021

Gene Editing Therapeutics Developer Verve Completes $266.7 Million IPO

Deals

June 17, 2021

Horizon Therapeutics Buy Manufacturing Facility to Support Its Growth

Drug Development

June 16, 2021

FDA Expands Approval of Blueprint Medicines’ Ayvakit to Include Rare Blood Disorder

Uncategorized

June 16, 2021

CAMP4 Raises $45 Million for Programmable Therapeutics to Upregulate Genes

Deals

June 16, 2021

n-Lorem Foundation Forms Partnership with Manufacturer Oligonucleotide Nitto Avecia

Research

June 15, 2021

Researchers Use Base Editing to Turn Pathogenic Gene Benign in Mice with SCD

Drug Development

June 15, 2021

Biogen Choroideremia Gene Therapy Fails in Phase 3 Study

Deals

June 15, 2021

CRISPR Therapeutics and Capsida Enter Strategic Collaboration to Develop Gene-Edited Therapies for ALS, FA

Drug Development

June 15, 2021

Novartis Reports Positive Results for PNH Therapy

Research

June 15, 2021

Nanoparticle Therapy Shows Early Promise at Preventing a Rare, Fatal Newborn Lung Disease

Deals

June 14, 2021

Cure Rare Disease Enters Partnership with Curi Bio for DMD Gene Therapy

Drug Development

June 14, 2021

Study Shows Evrysdi Improved Motor Function in Pre-Symptomatic Babies

Strategy

June 14, 2021

CDMO Yposkesi Launches Construction of Advanced Therapy Facility Near Paris

Drug Development

June 14, 2021

FDA Grants Jasper Therapeutics Rare Pediatric Disease Designation for Conditioning Treatment Prior to Stem Cell Transplant

RARECast Podcasts

June 11, 2021

Empowering Rare Disease Patients with their Own Health Records

In Rare Form

June 11, 2021

A Mother’s Prosecution Alarms Rare Patient Advocates

Drug Development

June 11, 2021

Sanofi Presents Positive New Data for Sutimlimab for the Treatment of CAD

Drug Development

June 11, 2021

Vertex Achieves Primary Endpoint in AATD Study but Drops Program Due to Insufficient Clinical Benefit

Rare Advocate

June 10, 2021

Rare Leader: Kim Hollander, Executive Director, Oxalosis & Hyperoxaluria Foundation

Deals

June 10, 2021

Codexis and Takeda Expand Collaboration to Additional Gene Therapy for a Rare Genetic Disorder

Finance

June 10, 2021

Kurome Closes $15 Million Financing to Develop Targeted Kinase Inhibitors to Treat Rare Blood Cancers

Drug Development

June 10, 2021

New Data Point to Potential Long-Term Efficacy of Biogen’s Spinraza for SMA

Data Center

June 9, 2021

Rare Disease Therapeutics Developers Continued to Rake in Cash in May

Finance

June 9, 2021

Alcyone Therapeutics Launches to Advance Next-Generation Gene Therapies for CNS Disorders

Drug Development

June 9, 2021

FDA Approves Trikafta in Children with Cystic Fibrosis Ages 6 through 11 with Certain Mutations

Finance

June 8, 2021

CyGenica Secures Seed Funding to Accelerate Cancer and Rare Genetic Disease Drug Delivery

Drug Development

June 8, 2021

FDA Approves Alexion’s Ultomiris for Children and Adolescents with PNH

Policy

June 8, 2021

Rare Disease Company Coalition Names Executive Committee

Drug Development

June 7, 2021

FDA Lifts Clinical Hold on Bluebird Bio’s Sickle Cell Disease and β-Thalassemia Studies

Drug Development

June 7, 2021

FDA Approves First Treatment for Patients with Plasminogen Deficiency, a Rare Genetic Disorder

Drug Development

June 7, 2021

Novartis Reports Positive Interim Analysis from Phase 2 Study in Rare Kidney Disease

RARECast Podcasts

June 4, 2021

Targeting CNS Diseases with Gene Therapies

Drug Development

June 4, 2021

Nanoscope Gene Therapy Restores Vision in 11 Patients Blinded by Retinitis Pigmentosa

Rare Advocate

June 3, 2021

Rare Leader: Krista Vasi, Executive Director, Usher Syndrome Coalition

Strategy

June 3, 2021

Auris Medical Reboots to RNA Therapeutics with Acquisition of Trasir Therapeutics

Finance

June 3, 2021

Amyl Raises $22 million to Develop Novel Therapies for Amyloidosis

Finance

June 3, 2021

Stablix Therapeutics Launches with $63 Million to Target Protein Stabilization

Drug Development

June 3, 2021

AavantiBio and Resilience Form Strategic Collaboration for Gene Therapy Development and Manufacturing

Drug Development

June 3, 2021

FDA Grants Protagonist Breakthrough Therapy Designation for Rusfertide in PV

Drug Development

June 3, 2021

FDA Grants Priority Review to Amryt’s EB Drug

Deals

June 2, 2021

MorphoSys to Acquire Rare Cancer Drug Developer Constellation Pharma for $1.7 Billion

Finance

June 2, 2021

Transine $12.8 Million Seed Funding to Advance Novel Class of Therapeutic RNAs

Drug Development

June 2, 2021

Santhera and ReveraGen Report Positive Topline Results in Pivotal DMD Study

Research

June 2, 2021

Barth Syndrome Foundation and American Heart Association Collaborate to Advance ResearchRare Daily Staff

Research

June 2, 2021

Treatabolome Project Seeks to Shorten Diagnosis-to-Treatment Time for Rare Disease Patients

Drug Development

June 2, 2021

FDA Grants Breakthrough Therapy Designation to Janssen Off-the-Shelf Cell Therapy for Multiple Myeloma

Drug Development

June 1, 2021

FDA Approves QED and Helsinn’s Truseltiq to Treat Patients with Cholangiocarcinoma

Research

June 1, 2021

Scientists Discover a New Genetic Form of ALS in Children

Diagnosis

June 1, 2021

Solve-RD Collaboration Makes Case for Data Sharing and Periodic Reanalysis

Drug Development

June 1, 2021

FDA Grants Fast Track Designation to BridgeBio’s Encaleret for the Treatment of ADH1

Deals

June 1, 2021

Genomenon and Nostos Genomics Partner to Accelerate Rare Genetic Disease Diagnoses

Drug Development

May 27, 2021

A New Therapy Offers A Different Approach to Inhibiting the Complement System

Rare Advocate

May 27, 2021

Rare Leader: Kyle Dempsey, President, Superficial Siderosis Research Alliance

Drug Development

May 27, 2021

Eloxx and Metagenomi Receive Funding Awards from Cystic Fibrosis Foundation

Finance

May 27, 2021

Day One Raises $160 Million in IPO to Advance Genetically Defined Cancer Therapies

Drug Development

May 26, 2021

Larimar Says $95 Million Placement Cancelled After FDA Places Clinical Hold on Lead Program

Business

May 26, 2021

Penn’s Gene Therapy Program Becomes Anchor Tenant of Discovery Labs

Finance

May 26, 2021

Rgenta Therapeutics Completes Seed Financing, Raising $38 Million for RNA-Targeted Pipeline

Drug Development

May 26, 2021

FDA Asks Travere for Additional Data Ahead of Marketing Application for FSGS Therapy

Deals

May 26, 2021

BlueRock and Senti Bio Collaborate to Develop Engineered Cell Therapies for Regenerative Medicine

Drug Development

May 25, 2021

Apellis and Sobi Report Positive Results from Empaveli Study in Treatment Naïve Patients with PNH

Drug Development

May 25, 2021

FDA Grants Scholar Rock Fast Track Designation for SMA Therapy

Regulatory

May 25, 2021

FDA Approves First Therapy for Rare Form of NSCLC with Companion Diagnostic

Regulatory

May 24, 2021

Xeris to Acquire Strongbridge

Deals

May 24, 2021

Xeris to Acquire Strongbridge

Regulatory

May 24, 2021

Bipartisan Legislation Introduced to Advance Biomedical Research Stalled by Pandemic

Regulatory

May 24, 2021

CHMP Recommends Approval of Therapies to Treat the Rare Disorders PFIC and POMC

Drug Development

May 21, 2021

Bringing Aberrant Proteins Back into the Fold

Finance

May 21, 2021

Haya Therapeutics Raises $20 Million Seed Financing for RNA-based Fibrosis Therapies

Deals

May 21, 2021

Mallinckrodt Reaches Agreement to Sell Experimental NPC-1 Drug Adrabetadex to Mandos

Drug Development

May 21, 2021

European Regulators Give Bluebird Bio Positive Opinion for CALD Gene Therapy

Drug Development

May 21, 2021

Biogen and Ginkgo Bioworks Enter Collaboration to Create Novel Gene Therapy Manufacturing Platform

Drug Development

May 21, 2021

European Medicines Agency Grants PRIME Designation to Larimar’s Treatment for Friedreich’s Ataxia

Rare Advocate

May 20, 2021

Rare Leader: Trish Flanagan, co-founder and president, YBRP

Drug Development

May 20, 2021

Adaptimmune Reports Positive Initial Data from Mid-Stage Trial in Rare Cancer

Data Center

May 19, 2021

Investors Pile $2.2 Billion into Rare Disease Therapeutics Developers in April

Finance

May 19, 2021

Eloxx Raises $52 Million in Public Offering to Advance Rare Disease Pipeline

Deals

May 19, 2021

Ciitizen and RARE-X Form Collaboration

Finance

May 18, 2021

G2 Bio Companies Launch with $200 Million to Develop Transformative Genetic-Based Therapies

Finance

May 18, 2021

Gamma Biosciences Acquires Controlling Stake in Mirus Bio

Finance

May 18, 2021

Vedere Bio II Launches with $77 Million to Develop Next Generation Ocular Gene Therapies

Finance

May 18, 2021

Stealth Bio Raises $30 Million to Advance Elamipretide Clinical Trials

Finance

May 18, 2021

Interius Raises $76 Million for Cell and Gene Therapy Platform

Deals

May 17, 2021

Charles River to Acquire Gene Therapy CDMO Vigene Biosciences

Drug Development

May 17, 2021

FDA Approves Apellis’ Empaveli for Adults with Paroxysmal Nocturnal Hemoglobinuria

RARECast Podcasts

May 14, 2021

A Cat who Curates Rare Mice

Finance

May 14, 2021

Praxis Precision Medicines Raises $91.3 Million in Public Offering

Deals

May 14, 2021

PerkinElmer to Expand Cell and Gene Therapy and Biologics Manufacturing with $260 Million Nexcelom Acquisition

Finance

May 14, 2021

Vera Therapeutics Raises $48 Million in IPO

Drug Development

May 14, 2021

Biogen XLRP Gene Therapy Fails Late-Stage Study

Drug Development

May 14, 2021

FDA Grants BridgeBio Fast Track Designation for CAH Gene Therapy

Rare Advocate

May 13, 2021

Rare Leader: Julie Breneiser, Executive Director, Gorlin Syndrome Alliance

Finance

May 13, 2021

SpliSense Secures $28.5 Million in Series B Financing Including Backing from CFF

Finance

May 13, 2021

Flare Launches with $82 Million to Advance Novel Drug Discovery Approach for Transcription Factors

Finance

May 13, 2021

Magenta Raises $86.4 Million to Advance Novel Antibody-Drug Conjugate Conditioning Platform

Policy

May 13, 2021

Rare Disease Drug Developers Form Coalition to Address Challenges

Deals

May 13, 2021

CRO Emmes Acquires UK-Based Orphan Reach

Deals

May 12, 2021

MD Anderson and Broad Institute Launch Translational Research Platform Focused on Rare Cancers

Finance

May 12, 2021

Huma Raises $200 Million to Scale Digital Health Platform for Better Care and Research

Finance

May 12, 2021

Gennao Bio Closes $40 Million in Financing to Develop Pipeline of Targeted Nucleic Acid Therapeutics

Drug Development

May 12, 2021

FDA Grants Fulcrum Fast Track for FSHD Drug

Finance

May 12, 2021

Xontogeny Seeds NephroDI to Develop a Treatment for Nephrogenic Diabetes Insipidus

Research

May 11, 2021

Gene Therapy Restores Immune Function in Children with Rare Immunodeficiency

Deals

May 11, 2021

Biogen Enters Collaboration with Capsigen for AAV Capsids for CNS and Neuromuscular Disorders

Research

May 11, 2021

Mutations Cause Rare Genetic Disease in Children

Uncategorized

May 11, 2021

FDA Halts Rocket Pharma Trial of Experimental Gene Therapy for Danon Disease

Drug Development

May 10, 2021

Laronde Unveiled to Advance New Class of Programmable Medicines

Drug Development

May 10, 2021

Ionis Stops Development of Inhaled Antisense Therapy for CF

Drug Development

May 10, 2021

FDA Grants Fast-Track Designation to Smart Immune’s T Cell Immunity Enhancement Product

RARECast Podcasts

May 7, 2021

Putting the Patient at the Center of Rare Disease Clinical Trials

Finance

May 7, 2021

Talaris Raises $150 Million in IPO to Advance Cell Therapy Pipeline

Finance

May 7, 2021

Science 37 to Become Publicly Listed via Merger with SPAC

Drug Development

May 7, 2021

ChemoCentryx Continues Slide on Divided FDA Advisory Committee Votes

Drug Development

May 7, 2021

Orphazyme Drug Fails in Pivotal Study

Rare Advocate

May 6, 2021

Rare Leader: Tiffany House, President Acid Maltase Deficiency Association

Finance

May 6, 2021

Dyno Therapeutics Raises $100 Million to Accelerate AI‑powered Gene Therapy Platform

Finance

May 6, 2021

TwinStrand Raises $50 Million to Grow Market for Duplex Sequencing Technology

Deals

May 5, 2021

Amryt Acquires Chiasma, Strengthening Its Rare and Orphan Disease Portfolio

Research

May 5, 2021

Centogene Discovers Six New Rare Diseases

Drug Development

May 5, 2021

ChemoCentryx Shares Crash After FDA Questions Data Supporting ANCA Vasculitis Therapy

Research

May 5, 2021

Study Finds People with Rare Diseases Often Feel Stigmatized

Drug Development

May 4, 2021

Sarepta Reports High-Dose SRP-5051 Increases Dystrophin in Certain DMD Patients

Finance

May 4, 2021

Mogrify Raises $17 Million to Advance Reprogrammed Cell Therapies

Deals

May 4, 2021

Passage Bio Partners with InformedDNA to Offer Genetic Counseling and Testing for FTD

Regulatory

May 4, 2021

Acella Issues Voluntary Nationwide Recall of Certain Lots of Thyroid Tablets Due to Sub Potency

Finance

May 3, 2021

Affinia Raises $110 Million to Advance Engineered AAV Gene Therapy Vector Platform

Finance

May 3, 2021

Aceragen Launches with $35 Million and Experimental Therapy for Farber Disease

Drug Development

May 3, 2021

With Accelerated Approval Off the Table, Avrobio Seeks Registration Trial for Fabry Disease Gene Therapy

Drug Development

May 3, 2021

Alnylam Reports Positive Early Results of Phase 3 PH1 Study

Drug Development

May 3, 2021

FDA Expands Approval of Chiesi’s Ferriprox for Transfusional Iron Overload due to Sickle Cell Disease

RARECast Podcasts

April 30, 2021

Creating a Toolkit to Accelerate the Development of Gene Editing Therapies

Rare Advocate

April 29, 2021

Rare Leader: Nancy Lazarus, Founder, Achalasia Awareness Organization

Finance

April 29, 2021

Capsida Debuts with $140 Million to Advance Next Generation Gene Therapies

Deals

April 29, 2021

Mirum Grants CANbridge Exclusive License to Maralixibat in Greater China for Rare Liver Diseases

Drug Development

April 29, 2021

FDA Grants Fast Track to Akari Bullous Pemphigoid Therapy

Drug Development

April 29, 2021

Astellas Takes $532 Million Charge Over Delay of Gene Therapy for XLMTM

Deals

April 28, 2021

Allen Institute and BioMarin Collaborate to Develop Gene Therapies for Rare Brain Diseases

Drug Development

April 28, 2021

FDA Tell Protalix and Chiesi It won’t Grant Accelerated Approval for Fabry Therapy

Deals

April 27, 2021

LogicBio Enters Gene-Editing Collaborations with CANBridge and Daiichi Sankyo

Deals

April 27, 2021

AllStripes Collaborates with NIH’s NCATS to Launch Effort in Rare Disease Diagnosis

Drug Development

April 27, 2021

FDA Removes Clinical Hold on Voyager, Clears IND for Huntington’s Gene Therapy

Drug Development

April 27, 2021

Biogen Will Provide Early Access to Experimental ALS Drug

Drug Development

April 27, 2021

EMA Grants PRIME Designation to Vertex and CRISPR’s Therapy for Transfusion-Dependent Beta Thalassemia

Drug Development

April 27, 2021

AavantiBio Partners with Catalent to Advance Gene Therapies for Rare Genetic Diseases

Regulatory

April 27, 2021

FDA Issues Guidance on Individualized ASOs for Ultra-Rare Conditions

Drug Development

April 26, 2021

FDA Removes Clinical Hold on Hemophilia B Gene Therapy Program

Drug Development

April 26, 2021

FDA Approves ADC Therapeutics’ Zynlonta to Treat Rare Cancer

RARECast Podcasts

April 23, 2021

PTC Looks to Advanced Therapies

Deals

April 23, 2021

CDISC and NORD to Develop Data Standards for Rare Disease

Rare Advocate

April 22, 2021

Rare Leader: Heidi Edwards, President and Founder, Sister’s Hope Foundation

Deals

April 22, 2021

Vertex and Obsidian Enter into Collaboration to Discover Novel Therapies that Regulate Gene Editing

Finance

April 22, 2021

ITM raises $109 Million to Advance Radiopharmaceutical Precision Oncology Pipeline

Finance

April 21, 2021

Code Biotherapeutics Launches with $10 Million to Develop Therapies for Debilitating Genetic Diseases

Finance

April 21, 2021

EdiGene Raises $62 Million to Advance and Scale Up Clinical Translation of Gene Editing Technologies

Drug Development

April 21, 2021

Beam Publishes Data Demonstrating Ability to Rationally Design Base Editors for Precise Editing

Pricing

April 21, 2021

Bluebird Withdraws Zynteglo from German Market, Cuts Workforce

Drug Development

April 21, 2021

EC Grants Approval to GW Pharmaceuticals’ Epidyolex for TSC-Associated Seizures

Uncategorized

April 21, 2021

FDA Grants RMAT Designation to Allogene’s Cell Therapy for Multiple Myeloma

Research

April 20, 2021

The Making of a Gene Therapy for an Ultra-Rare Disease

Drug Development

April 20, 2021

FDA Puts Clinical Hold on KalVista’s HAE Therapy

Deals

April 20, 2021

Citrine and Sinopharm Enter Strategic Partnership to Broaden Access to Rare Disease Drugs in China

Deals

April 20, 2021

Vertex and CRISPR Amend Sickle Cell Disease and Beta Thalassemia Collaboration

Research

April 20, 2021

IndoUSrare and Rare-X Conduct Feasibility Study for a Patient-Owned Health Data Collection Program for India

Business

April 20, 2021

Castle Creek Biosciences Appoints Matthew Gantz as President and CEO

Finance

April 19, 2021

Rezolute Enters $30 Million Debt Agreement to Advance Rare and Metabolic Disease Pipeline

Drug Development

April 19, 2021

Study Finds Positive Impact of Fintepla on Dravet Patients and Caregivers

Drug Development

April 19, 2021

Biogen Reports New Spinraza Data in SMA

RARECast Podcasts

April 16, 2021

Going to Extreme Lengths

Research

April 16, 2021

Study Identifies Key Target in Treatment-Resistant Hemophilia A

Finance

April 16, 2021

Recursion Raises $436 Million in Upsized IPO to Advance Rare Disease Pipeline

Rare Advocate

April 15, 2021

Lauren Dunlap, Co-Founder and Executive Director, AAIDA

Finance

April 15, 2021

Medable Raises $78 Million to Decentralize Clinical Trials

Finance

April 15, 2021

Alchemab Raises $82 Million to Advance Antibody Therapeutics in Neurodegeneration and Cancer

Deals

April 15, 2021

Ciitizen Partners with STXBP1 Foundation to Accelerate Patient Outcomes

Business

April 15, 2021

DOJ Subpoenas Documents from Alnylam Over Marketing of Onpattro

Drug Development

April 15, 2021

FDA Grants Rare Pediatric Disease Designation to FibroGen Treatment for DMD

Drug Development

April 15, 2021

New Evrysdi Data Shows Improvement in Motor Function Over Time for SMA Patients

Finance

April 14, 2021

Investment in Rare Disease Drug Development Continues to Surge

Research

April 14, 2021

Gene Therapy Shows Promise in Treating Rare Eye Disease in Mice

Deals

April 14, 2021

Huma Enters Partnership with RARE-X to Accelerate Rare Disease Research

Drug Development

April 14, 2021

FDA Grants Rare Pediatric Disease Designation to Therapies from Moleculin and Akuous

Deals

April 13, 2021

Senti Bio and Spark Collaborate to Develop Next-Generation Precision Gene Therapies

Deals

April 13, 2021

BridgeBio Pharma Enters Seven Collaborations with University Partners

Finance

April 13, 2021

Theseus Debuts with $100 Million Series B to for Targeted Treatment-Resistant Cancer Mutations

Finance

April 13, 2021

Arcellx Raises $115 Million to Advance Pipeline of Adaptive and Controllable Cell Therapies

Finance

April 13, 2021

Jaguar Gene Therapy Closes $139 Million Funding to Advance Preclinical Rare Disease Pipeline

Deals

April 13, 2021

ChemGenes to Provide n-Lorem Support for ASO Manufacturing

Deals

April 12, 2021

Taysha Acquires Global Rights to Experimental Gene Therapy for Rare Neurodegenerative Disease

Deals

April 12, 2021

Aytu Licenses Late-Stage Pediatric Onset Rare Disease Asset from Rumpus Therapeutics

Deals

April 12, 2021

Mirum Signs Option to License Vivet’s Experimental Gene Therapy Programs for PFIC

Deals

April 12, 2021

Sanofi Acquires Tidal Therapeutics with Its Innovative mRNA-based Research Platform

Drug Development

April 12, 2021

FDA Grants FibroGen Fast Track Designation for DMD Therapy

RARECast Podcasts

April 9, 2021

Everything’s up to Date in Kansas City, at Least When It Comes to Genomics

Finance

April 9, 2021

Two Rare Disease Biotechs Go Public, Raising Combined $222 Million in IPOs

Finance

April 8, 2021

ValenzaBio Raises $70 million in Venture Round

Finance

April 8, 2021

Three Rare Disease Biotechs Raise Combined $675 Million in Public and Private Offerings

Finance

April 8, 2021

Dicerna Sells Oxlumo Royalty Interest to Royalty Pharma for up to $240 Million

Rare Advocate

April 8, 2021

Rare Leader: Melody Burchett, President, A Cure In Sight

Strategy

April 7, 2021

Ionis Signs Distribution Deal with Sobi and Cuts 70 Percent of Akcea Workforce

Science & Technology

April 7, 2021

U.K.’s University of Sheffield Establishes New Gene Therapy Innovation Center

Finance

April 7, 2021

Metagenomi Extends Series A Financing to Advance Next-Gen Gene Editing Technology

Research

April 7, 2021

NIH Scientists Develop Breath Test for Rare Metabolic Condition

Research

April 7, 2021

Harvard Awards First Round of Rare Cancer Research Grants

Drug Development

April 7, 2021

FDA Grants Breakthrough Therapy to Taiho’s Treatment for Advanced Cholangiocarcinoma

Deals

April 6, 2021

Invitae Acquires Personalized Oncology Testing Company Generosity, Scores $1.2 B Financing

Drug Development

April 6, 2021

Progenity and Ionis Pharmaceuticals Enter into Agreement for Oral Delivery of Antisense Therapies

Drug Development

April 6, 2021

Scholar Rock Reports Positive Topline Results from Therapy for SMA Types 2 and 3

Research

April 5, 2021

Foundation for USP7 Related Diseases Makes Grant to Neurolentech

Drug Development

April 5, 2021

n-Lorem Enters Partnership with Ultragenyx for Personalized Treatments for Patients with Ultra-Rare Conditions

Drug Development

April 5, 2021

FDA Approves Praluent as Add-on Therapy for Patients with HoFH

Drug Development

April 5, 2021

Takeda’s Rare Disease Drug Natpara Won’t Be Available for Another Year

RARECast Podcasts

April 2, 2021

Choosing the Right Viral Vector for a Gene Therapy

Research

April 2, 2021

Penn Researchers Restore Vision in Blind Patient with Rare Genetic Condition with RNA Therapy

Rare Advocate

April 1, 2021

Rare Leader: Marissa Penrod, Founder and CEO, Team Joseph

Strategy

April 1, 2021

Nitto to Invest $226 Million to Expand Oligonucleotide Manufacturing in Massachusetts

Deals

April 1, 2021

Eloxx Acquires Zikani to Focus on Ribosomal RNA-Targeted Gene Therapy

Drug Development

April 1, 2021

FDA Approves New Orphan Indications for Three Marketed Drugs

Research

April 1, 2021

Uplifting Athletes Issues $140,000 in Rare Disease Research Grants

Deals

March 31, 2021

BridgeBio’s QED Therapeutics Enters Blockbuster Partnership with Helsinn for Infigratinib

Finance

March 31, 2021

Scribe Raises $100 Million to Further Develop Its “CRISPR by Design” Platform

Finance

March 31, 2021

Entrada Raises $116 Million to Advance Intracellular Biologics Pipeline into the Clinic

Drug Development

March 31, 2021

Forma Reports Positive Data in SCD Trial

Drug Development

March 30, 2021

Europe Approves Incyte’s Pemazyre for Rare Cancer

Finance

March 30, 2021

Omega Raises $126 Million to Advance Epigenomic Programming Pipeline and Platform

Drug Development

March 30, 2021

Wave Dumps Two Huntington’s Treatments after Failure to Show Benefit in Studies

Finance

March 30, 2021

CZI Begins New Round of Funding for Rare Disease Organizations

Finance

March 30, 2021

Imara Opens Applications for Real Impact Grants

Policy

March 29, 2021

Report Finds Most Rare Disease Drugs Protected by Patents Rather than Exclusivity

Drug Development

March 29, 2021

Ionis Reports Positive Topline Phase 2 Data of Antisense Treatment for HAE

Drug Development

March 29, 2021

Cyclo NPC1 Phase 1/2 Trial Hits Primary Endpoint

Drug Development

March 29, 2021

FDA Approves First Cell-Based Gene Therapy for Adult Patients with Multiple Myeloma

Drug Development

March 29, 2021

Orphazyme’s Treatment for Rare Muscle Wasting Disease Fails in Late Stage Trial

RARECast Podcasts

March 26, 2021

A Software Platform to Give Patients the Tools to Build Treatments for Rare Genetic Diseases

Finance

March 26, 2021

Two Rare Disease Drug Developers Raise Combined $416 Million in IPOs

Finance

March 26, 2021

Gyroscope Raises $148 Million to Advance AMD Gene Therapy

Drug Development

March 26, 2021

FDA Expands Use of Myrbetriq to Include Rare Pediatric Indication

Drug Development

March 26, 2021

FDA Approves Kiniksa’s Pericarditis Therapy Arcalyst

Rare Advocate

March 25, 2021

Rare Leader: Lisa Bonebrake, Executive Director, Alport Syndrome Foundation

Diagnosis

March 25, 2021

Sanford Health Enters Partnership with Congenica to Provide Answers for Undiagnosed Rare Disease Patients

Finance

March 24, 2021

Rare Disease Drug Developers’ Deals and IPOs Boost February Numbers

Finance

March 24, 2021

Step Pharma Raises $41.3 Million to Advance First-in-Class Therapy to Clinic in T Cell Malignancies

Innovation

March 24, 2021

Horizon Therapeutics Launches Prize with MIT to Advance Solutions for the Rare Disease Community

Drug Development

March 24, 2021

FDA Grants Fast Track Designation to Reneo’s PMM Therapy

Drug Development

March 24, 2021

ProQR Reports Positive Results of QR-421a in Usher Syndrome

Research

March 24, 2021

Researchers Find New Genetic Links to Rare Eye Disease

Uncategorized

March 23, 2021

PhoreMost Raises $46 Million to Advance Brain Cancer Treatment into Clinic

Drug Development

March 23, 2021

Maze Therapeutics Reveals First Three Programs Targeting Genetic Drivers of Disease

Drug Development

March 23, 2021

Genentech Halting Late-Stage Huntington’s Disease Study

Drug Development

March 23, 2021

Recordati Reports Positive Results from Late-Stage Cushing’s Disease Study

Drug Development

March 22, 2021

Study Shows Proof-of-Concept of Gene Editing in DMD

Diagnosis

March 22, 2021

Whole Genome Sequencing Enables Rare Disease Diagnoses

Drug Development

March 22, 2021

China Grants Citrine Clinical Trial Waiver for Narcolepsy Therapy Wakix

Finance

March 22, 2021

Aura Raises $80 Million to Advance Pipeline Targeting Rare Ocular Cancers

Strategy

March 22, 2021

VeriSIM Launches PulmoSIM to Develop Treatments for Rare Respiratory Diseases

Drug Development

March 22, 2021

BridgeBio’s Calcilytix Reports Proof-Of-Concept for ADH1 Therapy

RARECast Podcasts

March 19, 2021

Side Effects: The Toll a Rare Disease Can Take on a Family in Pursuit of a...

Finance

March 18, 2021

Two Rare Disease Biotechs Rake in $170 Million in Public Equity Offerings

Drug Development

March 18, 2021

Sarepta Reports Positive 2-Year Results from LGMD2E Gene Therapy Trial

Business

March 18, 2021

Chris Austin Will Leave NCATS to Join VC Firm Flagship Pioneering

Finance

March 18, 2021

Gain Therapeutics Raises $40M in IPO to Advance Rare Disease Pipeline

Rare Advocate

March 18, 2021

Rare Leader: Melanie McKay, Founder, Ryker’s Foundation for Pompe Disease

Deals

March 18, 2021

Anima and Takeda to Collaborate on Neurological Disease Therapeutics

Drug Development

March 18, 2021

Translate Bio Reports Results from Trial of mRNA Therapy for Cystic Fibrosis

Deals

March 17, 2021

Amathus and Merck Enter into Neurodegenerative Disease Pact

Deals

March 16, 2021

GBT In-Licenses Two Sickle Cell Small Molecule Programs from Sanofi

Finance

March 16, 2021

StrideBio Raises $81.5M to Advance AAV-Based Gene Therapies into Clinic

Research

March 16, 2021

Scientist Develop Novel Gene Editor to Correct CF and Other Mutations

Drug Development

March 16, 2021

Genentech Reports Positive New Data for Evrysdi in SMA Type 2 or Type 3

Drug Development

March 16, 2021

Nurix Enters Pediatric Cancer Collaboration Sponsored by ALSF

Drug Development

March 16, 2021

Solid Reports New Data from DMD Clinical Trial, Resumes Patient Dosing

Drug Development

March 15, 2021

Bluebird bio Reports Positive Long-Term Data for CALD Gene Therapy

Finance

March 15, 2021

Graphite Raises $150 Million to Advance Gene Editing Therapies for Rare Diseases

Deals

March 15, 2021

Genevant and Takeda Partner to Develop RNA Therapies for Liver Fibrosis

Finance

March 15, 2021

ElevateBio Raises $525M to Advance Cell/ Gene Therapy Business Model

Finance

March 15, 2021

Savara Raises $130 Million to Advance Development of aPAP Therapy

RARECast Podcasts

March 12, 2021

Realizing the Potential of CRISPR Gene Editing

Legislation

March 12, 2021

Congress Introduces Legislation Intended to Speed Development of Rare Disease Therapies

Finance

March 12, 2021

Longboard Raises $80 Million in IPO to Advance Rare Neurological Pipeline

Rare Advocate

March 11, 2021

Rare Leader: Blynda Killian, President, Cyclic Vomiting Syndrome Association

Drug Development

March 11, 2021

Intellia Presents Preclinical Proof of Concept for CRISPR-Based in Vivo Editing

Deals

March 11, 2021

Science 37 and Xperiome Partner to Speed Rare Disease Studies for Patients and Providers

Rare Patient

March 11, 2021

FDA Approves Compassionate Use of Longeveron Therapy in Child with Rare Heart Defect

Drug Development

March 11, 2021

Inability to Enroll Patients Terminates More Than a Quarter of Rare Disease Drug Trials

Regulatory

March 10, 2021

FDA Approves Second Sight Medical Products’ Retinal Prosthesis System

Drug Development

March 10, 2021

Bluebird Says Case of AML Unlikely Related to Gene Therapy Vector

Drug Development

March 10, 2021

FDA Tells Soleno It Will Need to Conduct Additional Study for Prader-Willi Therapy

Drug Development

March 9, 2021

Spinal Muscular Atrophy Foundation and PTC Therapeutics Expand Partnership

Drug Development

March 9, 2021

FDA Grants RMAT Designation to Rocket’s Gene Therapy for Rare Pediatric Disease LAD-I

Drug Development

March 8, 2021

FDA Approves Kite’s Yescarta for Relapsed or Refractory Follicular Lymphoma as Third Line Therapy

Policy

March 8, 2021

NICE Recommends Novartis’ Gene Therapy for SMA

Drug Development

March 8, 2021

AnaptysBio’s Experimental Therapy for Palmoplantar Pustulosis Fails in Phase 2 Trial

Drug Development

March 8, 2021

FDA Grants Fast Track Designation to Three Passage Bio Gene Therapy Candidates for Rare CNS Disorders

Drug Development

March 8, 2021

FDA Grants BioMarin RMAT Designation for Experimental Hemophilia A Gene Therapy

RARECast Podcasts

March 5, 2021

Understanding the Economic Toll of Rare Disease in the United States

Drug Development

March 5, 2021

FDA Approves Genentech’s Actemra to Slow Decline in Pulmonary Function in Adults with SSc

Policy

March 5, 2021

Rare Diseases Account for 11 percent of U.S. Drug Spending, Report Finds

In Rare Form

March 4, 2021

When Blockbusters Garner the Benefits of Orphan Drugs

Rare Advocate

March 4, 2021

Rare Leader: Danae’ Bartke, executive director and co-founder, HCU Network America

Policy

March 4, 2021

Advocacy Groups Call on Governors to Prioritize Vaccination of Rare Disease Patients and Caregivers

Strategy

March 4, 2021

Biogen Will Construct $200 Million Gene Therapy Manufacturing Facility in North Carolina

Deals

March 3, 2021

Takeda Secures Global Rights to Rare Epilepsy Treatment from Ovid for $860 Million

Finance

March 3, 2021

Caribou Raises $115 Million to Advance Next-Generation CRISPR Technologies

Research

March 3, 2021

Study Shows Potential for Lipid Nanonparticles to Deliver Gene Editing Therapy to Liver

Drug Development

March 2, 2021

FDA approves Oncopeptides’ Multiple Myeloma Therapy

Finance

March 2, 2021

eGenesis Completes $125 Million Financing

Finance

March 2, 2021

Mesoblast Raises $110 Million

Healthcare

March 2, 2021

Children’s National Hospital Rare Disease Institute Partners with Takeda to Standardize Care for Patients with Rare...

Healthcare

March 2, 2021

GBT Grant Program to Support to the Sickle Cell Disease Community

Finance

March 1, 2021

DTx Pharma Raises $100 Million to Advance its Platform and Pipeline of RNA Therapeutics

Drug Development

March 1, 2021

FDA Grants BridgeBio and Its Affiliate Origin Biosciences Approval for MoCD Therapy

Finance

March 1, 2021

SalioGen Therapeutics Emerges from Stealth, Closes $20 Million Series A

Research

March 1, 2021

Ionis Establishes New Grant Program to Advance Research and Understanding of ATTR

RARECast Podcasts

February 26, 2021

The Promise of Gene-Based Therapies for Neurodegenerative Conditions

Policy

February 25, 2021

When a Dollar Sign Becomes an Exclamation Point

Drug Development

February 25, 2021

Former AveXis Executives Launch Jaguar Gene Therapy to Target Rare Diseases

Rare Advocate

February 25, 2021

Rare Leader: Mark Stone, President and CEO, FSHD Society

Drug Development

February 25, 2021

FDA Grants Rare Pediatric Disease Designation to Retrotope’s Therapy for Two Neurodegenerative Indications

Finance

February 24, 2021

Orna Raises $80 Million to Develop a New Class of Engineered RNA Therapies

Deals

February 24, 2021

Scailyte and Volv Global Enter Strategic Partnership

Deals

February 24, 2021

Children’s Mercy Kansas City Expands Sequencing Capabilities

Deals

February 24, 2021

Cyprium and Sentynl Sign Development and Purchase Agreement for Menkes Disease Therapy

Uncategorized

February 23, 2021

Shepherd Enters Collaboration with NCATS to Improve Treatments for Rare Head and Neck Cancer

Deals

February 23, 2021

Soleno Collaborates with Vanderbilt on KATP Channel Activators for the Treatment of Rare Diseases

Deals

February 23, 2021

Day One Licenses Merck KGaA’s MEK Inhibitor Pimasertib, Expands Pipeline

Deals

February 23, 2021

BC Platforms Partners with Genomenon to Boost Rare Disease Diagnostics and Research

Deals

February 23, 2021

Beam Completes Acquisition of GuideTx

Drug Development

February 23, 2021

Protalix and Chiesi Report Positive Results from Open-Label, Switch Over Trial of Therapy for Fabry Disease

Drug Development

February 22, 2021

FDA Tells BrainStorm Evidence Appears Lacking to Support Seeking Approval for ALS Therapy

RARECast Podcasts

February 19, 2021

Moving Beyond Viral Vectors for Gene Therapies

Rare Advocate

February 18, 2021

Rare Leader: Paula Evans, founder and chairperson, Foundation for Angelman Syndrome Therapeutics (FAST)

Research

February 18, 2021

NCI Awards $16 Million Grant to Advance Research of Two Rare Cancers

Deals

February 18, 2021

Charles River to Acquire Cell and Gene Therapy Manufacturer Cognate BioServices for $875 million

Innovation

February 18, 2021

FDA Approves First-of-Its-Kind Implant to Treat Rare Bone Disease as a Humanitarian Use Device

Diagnosis

February 18, 2021

FDNA Launches Telehealth AI Telegenetics Platform

Finance

February 18, 2021

Evox Therapeutics Raises $95.4 Million To Advance Exosome-Based Pipeline

Deals

February 18, 2021

Otsuka and Eikonizo to Collaborate on Treatments for ALS and Other Rare Diseases

Deals

February 17, 2021

Novartis Teams with Gates Foundation to Develop an Accessible in Vivo SCD Gene Therapy

Data Center

February 17, 2021

Rare Disease Drug Developers Busy Raising Money in January

Drug Development

February 17, 2021

Health Canada Approves Ultragenyx’ Drug for Group of Rare Metabolic Disorders

Healthcare

February 17, 2021

Eurordis Survey Finds People with Rare Diseases Have Concerns with Quality of Healthcare Experience

Research

February 16, 2021

Study Finds Commercial Genetic Testing Technology Falsely Detects Very Rare Variants

Drug Development

February 16, 2021

Bluebird Bio Temporarily Suspends SCD Gene Therapy Trials after Case of AML

Drug Development

February 16, 2021

FDA Approves Pfizer’s Panzyga to Treatment of Adults with CIDP

Deals

February 16, 2021

Shepherd and Oncoheroes Partner to on Medicines for Rare Cancers

Drug Development

February 16, 2021

FDA Grants Rare Pediatric Disease Designation to Forge’s Gene Therapy for Krabbe Disease

Rare Community

February 12, 2021

Mila Makovec, Recipient of Bespoke ASO for Ultra-Rare Condition, Dies at 10

RARECast Podcasts

February 12, 2021

Acquisitions Help Jazz Build Toward Commercial Crescendo

Research

February 12, 2021

Patients’ Cells Offer Insight into New Gene Therapy Approach to Rare Eye Disease

Finance

February 12, 2021

Travere Raises $175 Million in Public Offering to Advance Rare Kidney Disease Pipeline

Finance

February 12, 2021