pre-IPO PHARMA

TAG: Gene Therapy

Sep 28, 2023

Nanoscope Therapeutics to Present at the 4th Annual Gene Therapy for Ophthalmic Disorders Summit


Sep 19, 2023

ATA-200, Atamyo Therapeutics’ Gene Therapy to Treat Limb-Girdle Muscular Dystrophy Type 2C/R5, Reaches Key Milestones with the Filing of a Clinical Trial Application in Europe and a Non-Dilutive Financing from France 2030 Program


Sep 6, 2023

Neurophth Secures Australian Approval for Ophthalmic Gene Therapy


Aug 17, 2023

Neurophth Announces First Patient Dosed in Phase I/II Clinical Trial of Second Gene Therapy


Aug 11, 2023

Neurophth Secures Nearly 95 Million USD in Series C+ Financing for Gene Therapy Clinical Trials



Aug 3, 2023

Results from XyloCor Therapeutics’ Phase 1 Portion of EXACT Trial of XC001 for Cardiovascular Disease Published in Circulation: Cardiovascular Interventions


Aug 1, 2023

LEXEO Therapeutics Announces FDA Clearance of IND for LX2020, an AAV-based Gene Therapy Candidate for PKP2 Arrhythmogenic Cardiomyopathy


Jul 24, 2023

Gene Therapy Pioneer Roger Hajjar, MD, Joins Atamyo Therapeutics Board of Directors


Jul 20, 2023

Excision BioTherapeutics Receives FDA Fast Track Designation for EBT-101, a First-in-Class CRISPR-Based Gene Therapy Candidate to Functionally Cure HIV-1


Jul 18, 2023

XyloCor Therapeutics Reports Sustained Results in 12-Month Extension of Phase 2 EXACT Clinical Trial of XC001 Novel Gene Therapy for Refractory Angina



Jun 28, 2023

New Hope Research Foundation and Forge Biologics Announce cGMP Manufacturing Partnership to Accelerate Gene Therapy for Patients with Tay-Sachs Disease


Jun 27, 2023

Intergalactic Therapeutics Announces Positive Results for Its Non-Viral Gene Therapy Platform in ABCA4 Retinopathies in Non-Human Primates


Jun 24, 2023

Fractyl Health Demonstrates Significant Improvement in Glucose Control and Obesity in Preclinical Studies of its Rejuva GLP-1 Based Pancreatic Gene Therapy (GLP1 PGTx) Compared to Chronic Semaglutide at ADA's 83rd Scientific Sessions


Jun 5, 2023

Vycellix Presents Proof-of-Concept Data for VY-UC Allogeneic Cell Therapy Platform at the International Society for Cell and Gene Therapy (ISCT) Annual Meeting, Paris


May 31, 2023

SNIPR Biome reports positive clinical interim results for groundbreaking, first-in-human, CRISPR-based microbial gene therapy USA - English USA - English APAC - English



May 23, 2023

Forge Biologics Joins Bespoke Gene Therapy Consortium (BGTC) as Partner to Accelerate the Development and Manufacturing of AAV Gene Therapies for Patients with Rare Diseases


May 18, 2023

Fractyl Health Unveils Groundbreaking Pre-Clinical Proof of Concept Results for its Rejuva Pancreatic Gene Therapy Platform for Metabolic Diseases at the American Society of Gene and Cell Therapy Congress


May 17, 2023

Kelonia Therapeutics Presents Research Highlighting Potential of iGPS™ in vivo Gene Therapy to Bring Safe, Effective CAR T Therapies to Common Medical Practice for Treatment of All Multiple Myeloma Patients


May 11, 2023

Intergalactic Therapeutics to Present Preclinical Data Highlighting Potential of Its Non-Viral Gene Therapy Platform at the American Society of Gene and Cell Therapy 26th Annual Meeting


May 9, 2023

Fractyl Health to Present Preclinical Proof-of-Concept Data for Rejuva Pancreatic Gene Therapy Platform Program at the American Society of Gene and Cell Therapy Congress



May 3, 2023

Forge Biologics and Labcorp Announce Strategic AAV Gene Therapy Development and Manufacturing Collaboration


May 3, 2023

HuidaGene Therapeutics Announces 19 Presentations Highlighting Advances in its Gene Therapy Technology Platforms at the 2023 ASGCT Annual Meeting


Apr 20, 2023

Intergalactic Therapeutics to Present Updated Data From Its Non-Viral Gene Therapy Platform at the 2023 Retinal Cell and Gene Therapy Innovation Summit


Apr 20, 2023

Former Pfizer Legal Counsel Discusses Gene Therapy with CancerVAX CEO Ryan Davies


Apr 18, 2023

Ascidian Therapeutics to Present Data From Its Lead Program Targeting ABCA4 Retinopathies at the Retinal Cell and Gene Therapy Innovation Summit and ARVO 2023 Annual Meeting



Apr 5, 2023

Ginkgo Bioworks Strengthens End-to-End RandD Capabilities in Gene Therapy through Acquisition of StrideBio's AAV Capsid Discovery and Engineering Platform Assets


Apr 3, 2023

HuidaGene Receives Orphan Drug Designation for Gene Therapy of Blindness USA - English France - Français Deutschland - Deutsch


Mar 15, 2023

Intergalactic Therapeutics Announces Positive Preclinical Results of Its Non-Viral Gene Therapy Platform in ABCA4 Retinopathies


Mar 7, 2023

Maltese Patient Received World's First ND1-LHON Gene Therapy Injection Donated by Neurophth in China USA - English USA - español USA - Deutsch USA - Français


Mar 7, 2023

Maltese Patient Received World's First ND1-LHON Gene Therapy Injection Donated by Neurophth in China



Feb 27, 2023

SonoThera™ Receives Exclusive Licensing of GE HealthCare’s Optison and Sonazoid Microbubble Products for use with its Ultrasound-guided Gene Therapy Platform


Feb 23, 2023

Genascence Receives $11.6 Million Award From California Institute for Regenerative Medicine to Help Advance its Lead Gene Therapy Program in Knee Osteoarthritis


Feb 23, 2023

AviadoBio Signs Exclusive License Agreement with Neurgain Technologies for Novel Gene Therapy Spinal Delivery Technology


Feb 21, 2023

Commercialization of Gene Therapy is coming soon: Neurophth Completes Patient Enrollment for Phase III clinical trial for the gene therapy treatment of LHON


Jan 26, 2023

XyloCor Therapeutics Reports Positive Topline Safety and Efficacy Results from Phase 2 EXACT Clinical Trial of XC001 Novel Gene Therapy for Refractory Angina



Jan 17, 2023

Forge Biologics Receives Priority Medicines (PRIME) Designation from the European Medicines Agency (EMA) for Novel Gene Therapy FBX-101 for the Treatment of Patients with Krabbe Disease


Jan 17, 2023

Indapta Therapeutics Names Moya Daniels, Senior Vice President of Regulatory, Quality and Clinical Operations


Jan 10, 2023

Forge Biologics and Oculogenex Announce AAV Contract Development and Manufacturing Partnership


Dec 19, 2022

Neurophth Receives IND Clearance from FDA for AAV-ND1 Gene Therapy of LHON


Dec 15, 2022

SwanBio Therapeutics Initiates First-in-Human Study of AAV Gene Therapy for Adrenomyeloneuropathy



Dec 8, 2022

Anjarium Biosciences appoints ex-Novartis gene therapy leader Otmane Boussif, DSc as Chief Technology Officer


Dec 5, 2022

Fractyl Health Announced Positive Proof-of-Concept Data of Pancreatic GLP-1 Gene Therapy in a Type 2 Diabetes Animal Model at the 20th World Congress on Insulin Resistance, Diabetes and Cardiovascular Disease


Dec 5, 2022

SonoThera™ Completes $60.75M Series A Funding for Ultrasound-Guided, Nonviral Gene Therapy Platform Development


Nov 29, 2022

Eikonoklastes Therapeutics and Forge Biologics Announce Viral Vector Contract Development and AAV Manufacturing Partnership


Nov 16, 2022

Kriya Acquires Redpin Therapeutics, Adding Neurology Pipeline to Gene Therapy Portfolio



Nov 7, 2022

Cyagen and Neurophth Enter Global Strategic Collaboration to Develop AI-Designed AAV Gene Therapy Vectors for Ophthalmic Disorders


Nov 7, 2022

Nanoscope Therapeutics Expands Clinical and CMC Leadership Team with Gene Therapy Pharma Experience


Nov 2, 2022

LEXEO Therapeutics Bolsters Cardiac Gene Therapy Leadership with New Executive Appointment and Formation of Scientific Advisory Board


Oct 27, 2022

LEXEO Therapeutics to Present Clinical and Preclinical Data at Upcoming Scientific Conferences


Oct 18, 2022

Charles River and Nanoscope Therapeutics Announce Multifaceted Gene Therapy Manufacturing Partnership



Oct 18, 2022

LEXEO Therapeutics Receives Orphan Drug Designation for LX1004 from European Commission


Oct 11, 2022

Forge Biologics Announces Updated Positive Clinical Data in RESKUE, a Novel Phase 1/2 Gene Therapy Trial for Patients with Krabbe Disease


Oct 11, 2022

M6P Therapeutics Presents Novel AAV Gene Therapy Approach for the Treatment of Gaucher Disease at the ESGCT 29th Annual Congress


Oct 5, 2022

LEXEO Therapeutics to Present New Clinical Data from its Investigational Gene Therapy LX1001 for APOE4-Associated Alzheimer’s Disease at the 29th European Society of Gene and Cell Therapy Annual Meeting


Oct 3, 2022

AMPLIFYBIO ACQUIRES PACT PHARMA ASSETS TO ENHANCE CELL AND GENE THERAPY CHARACTERIZATION CAPABILITIES



Oct 3, 2022

Myrtelle and Forge Biologics Announce Viral Vector and Plasmid DNA cGMP Manufacturing Partnership


Sep 28, 2022

Code Biotherapeutics Names David Anderson, PhD, as Chief Scientific Officer


Sep 27, 2022

Forge Biologics Fuels Gene Therapy Manufacturing Engine with Launch of Plasmid DNA Manufacturing Services to Support AAV Clients


Sep 27, 2022

Neurophth announces first patient dosed in Phase III clinical trial for the gene therapy treatment of LHON


Sep 26, 2022

Atamyo Therapeutics Announces First Patient Dosed with ATA-100 Gene Therapy in LGMD-R9 Clinical Trial



Sep 20, 2022

Rocket Pharmaceuticals to Acquire Renovacor, Extending Leadership in AAV-based Cardiac Gene Therapy


Sep 20, 2022

Chroma Medicine Announces Formation of Scientific Advisory Board of Global Experts in Gene Editing and Cell and Gene Therapy


Sep 14, 2022

Tessa Therapeutics Recognized in “Most Promising Off-the-Shelf Therapies” Category at Asia-Pacific Cell and Gene Therapy Excellence Awards 2022


Sep 13, 2022

Nanoscope Therapeutics Announces Completion of Enrollment in STARLIGHT Phase 2 Clinical Trial of MCO-010 Optogenetic Gene Therapy for Stargardt Disease


Sep 12, 2022

Forge Biologics Announces $90 Million Series C Financing to Expand Client Offerings and Add Services to Enhance End-to-End Gene Therapy Manufacturing Platforms



Sep 8, 2022

Nanoscope Therapeutics to Present at the 3rd Annual Gene Therapy for Ophthalmic Disorders Meeting


Sep 7, 2022

GRO Biosciences Announces Addition of Nicole Paulk, PhD, to Scientific Advisory Board


Aug 30, 2022

Forge Biologics Reports Positive Clinical Data on Brain Development and Motor Function from the RESKUE Novel Phase 1/2 Gene Therapy Trial in Patients with Krabbe Disease at the SSIEM Annual Symposium


Aug 25, 2022

ElevateBio and the University of Pittsburgh Announce Creation of Pitt BioForge BioManufacturing Center at Hazelwood Green to Accelerate Cell and Gene Therapy Innovation


Aug 17, 2022

Philadelphia-based Biotech Company AUM LifeTech Awarded $400,000 NCI/NIH Grant to Advance Their Lung Cancer Immunotherapy Program Using RNA-Targeting Gene Therapy with Checkpoint Inhibitors



Aug 4, 2022

ElevateBio Announces the Formation of a New Company With George Daley, M.D., Ph.D., and Boston Children’s Hospital to Develop iPSC-Derived Allogeneic Immune Therapies


Jul 25, 2022

Nanoscope Therapeutics Announces First Patient Dosed in STARLIGHT Phase 2 Clinical Trial of MCO-010 Optogenetic Gene Therapy for Stargardt Disease


Jun 28, 2022

XyloCor Therapeutics Achieves Target Enrollment in Phase 2 EXACT Study of XC001 Novel Gene Therapy for Ischemic Heart Disease


Jun 27, 2022

Ambys Medicines Announces Formation of Clinical and Scientific Advisory Boards with Leading Liver Disease and Cell and Gene Therapy Experts


Jun 13, 2022

Charles River and ASC Therapeutics to Scale Manufacturing of Second-Generation Gene Therapy for Hemophilia A



Jun 13, 2022

Mnemo Therapeutics Launches RHU Project in Collaboration with Leading European Research Partners, Institut Curie and MEARY Cell and Gene Therapy Center, AP-HP USA - English France - Français


Jun 10, 2022

Aruvant Announces Data Presentation at European Hematology Association 2022 Hybrid Congress


May 24, 2022

ASC Therapeutics Joins U.S. Consortium of Gene Therapy Partners Including the NIH and FDA to Accelerate Treatments for Rare Diseases


May 20, 2022

CORRECTING and REPLACING ASC Therapeutics, UMass Chan Medical School, and the Clinic for Special Children Announce Podium Presentation of Safety and Efficacy in Murine and Bovine Models for Novel Gene Therapy in Maple Syrup Urine Disease


May 18, 2022

XyloCor Therapeutics Announces Presentation of Preliminary Clinical Data from Phase 1 Portion of the EXACT Phase 1/2 Study of XC001 Novel Gene Therapy for Refractory Angina at AATS and ASGCT



May 17, 2022

Forge Biologics Forms Independent Scientific and Manufacturing Advisory Board with Panel of Gene Therapy Leaders


May 17, 2022

Capsida Biotherapeutics Presents New Data Validating Potential of Its Targeted, Non-invasive Gene Therapy Platform at the Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT)


May 16, 2022

ASC Therapeutics Announces Podium and Poster Presentations of IND-Enabling Studies for ASC618, a Second-Generation Gene Therapy for Hemophilia A, at the 25th ASGCT Meeting, May 16-19, 2022 in Washington, D.C.


May 16, 2022

Atamyo Therapeutics Announces Significant Milestones for ATA-100 and ATA-200, its Gene Therapy Programs to Treat Limb-Girdle Muscular Dystrophy 2I/R9 and 2C/R5


May 16, 2022

Kriya Announces $270 Million Series C Financing to Advance Fully Integrated Gene Therapy Engine



May 9, 2022

International Society for Cell and Gene Therapy Presentations Illustrate Evox Therapeutics’ Proprietary Exosome Engineering Platform with New Data on Exosome Targeting and In Vivo Distribution


May 4, 2022

Umoja Biopharma Presents Data on its Synthetic Cytokine Receptor Platform at the International Society for Cell and Gene Therapy Annual Meeting 2022


Apr 26, 2022

ViGeneron announces follow-on collaboration with Daiichi Sankyo to develop a novel gene therapy for prevalent eye diseases


Apr 6, 2022

ViGeneron signs gene therapy strategic collaboration and option agreement with Regeneron for one inherited retinal disease target


Mar 29, 2022

ASC Therapeutics Receives Key Regulatory Designations in U.S. and Europe to Advance its Second-Generation Gene Therapy for Hemophilia A



Mar 22, 2022

QurAlis to Present at H.C. Wainwright Gene Therapy and Gene Editing Virtual Conference


Mar 15, 2022

FDA Grants Orphan Drug Designation to SBT101, the First Investigational AAV-Based Gene Therapy for Adrenomyeloneuropathy (AMN)


Mar 15, 2022

EXUMA Biotech to Present Preclinical Data from its Subcutaneous CAR-TaNK and In Vivo CAR Cell and Gene Therapy Programs at the American Association for Cancer Research Annual Meeting


Mar 9, 2022

Twist Bioscience and Kriya Sign Agreement to Discover Novel Antibodies for Applications in Oncology Using Gene Therapy


Mar 8, 2022

CODA Biotherapeutics Names Susan Catalano, Ph.D., as Chief Scientific Officer



Mar 4, 2022

SwanBio Therapeutics to Present New Preclinical Data Showcasing AAV-Based Gene Therapy Candidate for Adrenomyeloneuropathy at American Academy of Neurology Annual Meeting


Mar 2, 2022

LEXEO Therapeutics Announces Positive Initial Data from Ongoing Phase 1/2 Clinical Trial of AAV-based Gene Therapy Candidate LX1001 in Patients with Alzheimer’s Disease


Mar 1, 2022

Ray Therapeutics and Forge Biologics Announce Viral Vector Contract Development and cGMP Manufacturing Partnership


Feb 24, 2022

Atamyo Therapeutics Reaches Significant Regulatory and Financial Milestones for ATA-100, its Gene Therapy to Treat Limb-Girdle Muscular Dystrophy Type 2I/R9


Feb 16, 2022

FDA Grants Fast Track Designation to SBT101, the First Investigational AAV-Based Gene Therapy for Patients With Adrenomyeloneuropathy (AMN)



Feb 16, 2022

LEXEO Therapeutics Announces FDA Clearance of Investigational New Drug Application for LX2006, an AAV-based Gene Therapy Candidate for Friedreich’s Ataxia Cardiomyopathy


Feb 7, 2022

Nanoscope Therapeutics Announces Completed Enrollment of its Phase 2b Clinical Trial in Blind Patients for Optogenetic Gene Therapy to Restore Vision


Feb 7, 2022

Aruvant to Participate in the 2022 BIO CEO and Investor Gene Therapy Panel on February 16, 2022


Jan 25, 2022

Neurophth Therapeutics' Treatment of Leber's Hereditary Optic Neuropathy Gene Therapy NR082 was Granted Orphan Drug Designation by EMA


Jan 25, 2022

Nanoscope Therapeutics Announces FDA Clearance of IND for MCO-010 Gene Therapy in Stargardt Macular Degeneration Patients



Jan 25, 2022

SwanBio Therapeutics Announces FDA Investigational New Drug Clearance for First AAV-Based Gene Therapy for the Treatment of Adrenomyeloneuropathy


Jan 19, 2022

ElevateBio Announces Andrew Sandford Joins as President of ElevateBio BaseCamp


Jan 19, 2022

Neurophth Therapeutics Receives IND Clearance to Initiate Clinical Trial for China AAV-ND4 Gene Therapy NR082 in Leber Hereditary Optic Neuropathy


Jan 7, 2022

Kriya Expands Gene Therapy Pipeline and Establishes Its Rare Disease Therapeutic Area Division With the Acquisition of Warden Bio


Dec 22, 2021

Novartis to acquire Gyroscope Therapeutics, adding a one-time gene therapy that could transform care for geographic atrophy, a leading cause of blindness



Dec 8, 2021

Nanoscope Therapeutics' Optogenetic Gene Therapy to be Featured in Presentation at Stanford University's Innovate Retina Summit Dec. 11, 2021


Dec 7, 2021

CODA Biotherapeutics' Chemogenetic Gene Therapy Platform Can Control Focal Seizures in vivo


Dec 7, 2021

Capsida Biotherapeutics Poised to Capitalize on Industry-leading Gene Therapy Technology With New CEO, CSO, and CTO


Dec 6, 2021

Atamyo Therapeutics Obtains First Regulatory Authorization in Europe to Initiate a Clinical Trial for ATA-100, its Gene Therapy to Treat Limb-Girdle Muscular Dystrophy Type 2I/R9


Dec 3, 2021

Nanoscope Therapeutics Wins 'Top Retina Company' Award at 2021 Eyecelerator Conference for Its Optogenetics Gene Therapy to Restore Vision in People Blinded by Retinal Diseases



Dec 2, 2021

CODA Biotherapeutics Closes $28 Million Financing led by Pacira BioSciences


Dec 1, 2021

Astellas and Dyno Therapeutics Announce Research Collaboration to Develop Next-Generation AAV Gene Therapy Vectors for Skeletal and Cardiac Muscle


Nov 15, 2021

Nanoscope Therapeutics co-Founder and President to Present at World Orphan Drug Congress Europe 2021, Nov. 17, in Barcelona, Spain


Nov 15, 2021

Aruvant Appoints Moya Daniels as Senior Vice President of Regulatory Affairs and Quality Assurance


Nov 8, 2021

Gyroscope Therapeutics Announces Sanofi Investment of Up to $60 Million



Nov 4, 2021

EdiGene to Present Latest Research on A Novel Surface Marker and Migration of Hematopoietic Stem Cell (HSC) That Could Enhance HSC Gene Therapy and HSC Transplantation at the 63rd American Society of Hematology (ASH) Annual Meeting and Exposition


Oct 28, 2021

Ensoma Strengthens Scientific Advisory Board with Leaders in Oncology and Primary Immune Deficiencies


Oct 27, 2021

Forge Biologics to Present at Jefferies Gene Therapy / Editing Summit


Oct 26, 2021

Atamyo Therapeutics Submits CTA in Europe for ATA-100, Its Gene Therapy to Treat Limb-Girdle Muscular Dystrophy Type 2I/R9


Oct 19, 2021

SwanBio Therapeutics Announces New Preclinical Data Demonstrating Proof of Principle for SBT101 Gene therapy as a Potential Treatment of Adrenomyeloneuropathy



Oct 19, 2021

Boehringer Ingelheim and Partners to Accelerate Development of First-In-Class Gene Therapy for Patients with Cystic Fibrosis


Oct 12, 2021

AavantiBio Furthers Strategic Partnership with University of Florida to Research and Develop Next Generation Gene Therapy Capsids


Oct 11, 2021

Forge Biologics to Present at 2021 Cell and Gene Meeting on the Mesa


Oct 11, 2021

Ensoma Appoints Cell and Gene Therapy Pioneer Emile Nuwaysir, Ph.D., as Chief Executive Officer


Oct 7, 2021

ATP Unveils Non-Viral Gene Therapy Innovator Intergalactic Therapeutics with $75 Million in Series A Funding



Oct 4, 2021

Solid Biosciences and Forge Biologics Announce Viral Vector Contract Development and cGMP Manufacturing Partnership


Oct 4, 2021

StrideBio Announces Achievement of Significant Operational Milestones Supporting Transition of Novel Gene Therapy Pipeline to Clinical Stage


Sep 30, 2021

Gyroscope Therapeutics Announces Presentation of Positive Interim Phase I/II Data for Investigational Gene Therapy GT005 at Retina Society Annual Scientific Meeting


Sep 27, 2021

Neurophth Announces the Completion of GMP Manufacturing Facility for Gene Therapy Products According to International Standards


Sep 23, 2021

Quell Therapeutics Significantly Expands Clinical Manufacturing Capacity for its Engineered Treg Cell Therapies through a Collaboration with the Cell and Gene Therapy Catapult



Sep 20, 2021

Aruvant Announces ARU-2801 Data Presentations at the American Society for Bone and Mineral Research (ASBMR) 2021 Annual Meeting


Sep 9, 2021

LEXEO Therapeutics Closes $100 Million Series B Financing to Advance Clinical Stage Gene Therapy Pipeline and Enhance Preclinical Footprint


Sep 8, 2021

Tenaya Therapeutics Reports Second Quarter 2021 Financial Results and Provides Business Updates


Sep 8, 2021

Affinia Therapeutics Announces Indications for Lead Gene Therapy Programs to Advance the Treatment of Neurologic and Neuro-oncologic Diseases supported by Multi-Year Manufacturing Agreement with Lonza Group


Sep 8, 2021

Nanoscope Therapeutics Announces Presentation of Positive Results from Optogenetic Gene Therapy for Retinal Degenerative Diseases at the EURETINA 2021 Virtual Conference



Aug 24, 2021

Xalud Therapeutics Completes Oversubscribed $30 Million Series C Financing


Aug 19, 2021

Neurophth Expands Its Global Gene Therapy Leadership Team with the Appointment of Dr. Zhengbin Li as Head of Commercial


Aug 17, 2021

Zenith Epigenetics Announces a Publication in the Journal of Cancer Gene Therapy with Compelling Data for the Treatment of ER-Positive Breast Cancer Patients


Aug 11, 2021

Be Bio Appoints Leading Cell and Gene Therapy Executives, Krishnan Viswanadhan as President and Chief Operating Officer, and Brad Hartman as Chief People Officer


Jul 30, 2021

Integral Molecular Launches Transgene Protein Engineering Platform to Address Challenges in Gene Therapy



Jul 28, 2021

Kriya Therapeutics Completes Renovation of Its Facility for Scalable Gene Therapy Manufacturing


Jul 27, 2021

XyloCor Therapeutics Commences Phase 2 Component of Phase 1/2 EXACT Clinical Study of XC001 Gene Therapy for Refractory Angina


Jul 27, 2021

ASC Therapeutics Announces Partnership With University of Massachusetts Medical School to Co-Develop Novel Gene Therapy for Maple Syrup Urine Disease


Jul 22, 2021

Capsida Biotherapeutics Unveils Next-Generation Gene Therapy Manufacturing


Jul 21, 2021

LEXEO Therapeutics Expands Cardiac Gene Therapy Pipeline with Acquisition of Stelios Therapeutics and its Gene Therapy Programs for Rare Cardiovascular Diseases



Jul 20, 2021

Affinia Therapeutics Announces Collaboration with the Institute of Molecular and Clinical Ophthalmology Basel (IOB) To Rationally Design Novel CNS Promoters for Gene Therapy


Jul 15, 2021

Shape Therapeutics Secures $112 Million in Series B Financing to Advance Breakthrough RNA Technology Platforms to Democratize Gene Therapy


Jul 7, 2021

ASC Therapeutics Receives IND Clearance From the U.S. Food and Drug Administration for ASC618 Second-Generation Gene Therapy for Hemophilia A


Jul 6, 2021

Forge Biologics and Case Western Reserve University Announce Gene Therapy Workforce Development Partnership


Jun 29, 2021

Boston Children’s Hospital and ElevateBio Announce Five-Year Cell and Gene Therapy Collaboration



Jun 28, 2021

First in China! Neurophth announced the first patient has been dosed in G.O.L.D. clinical trial for the gene therapy treatment of LHON


Jun 22, 2021

Gyroscope Therapeutics Announces Research Collaboration Agreement with Children’s Medical Research Institute to Develop Novel Gene Therapy Capsids


Jun 21, 2021

Jasper Therapeutics and Aruvant Announce Research Collaboration to Study JSP191, an Antibody-Based Conditioning Agent, with ARU-1801, a Novel Gene Therapy for the Treatment of Sickle Cell Disease


Jun 14, 2021

Aruvant Announces ARU-2801 Data Publications and Advocacy Partnership


Jun 10, 2021

AUM LifeTech, Inc. to Present Preclinical Developments on Lung Cancer, Parkinson's Disease, and HIV at BIO Digital 2021



Jun 9, 2021

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


Jun 3, 2021

Forge Biologics Expands Gene Therapy Leadership Team with Appointment of John Maslowski as Chief Commercial Officer


Jun 3, 2021

Nanoscope's Optogenetic Gene Therapy Restores Clinically Meaningful Vision in 11 Patients Blinded by Retinitis Pigmentosa


Jun 3, 2021

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


May 20, 2021

Tenaya Therapeutics Receives Orphan Drug Designation and Presents Pre-Clinical Data for its Most Advanced Gene Therapy Product Candidate for Genetic Hypertrophic Cardiomyopathy



May 18, 2021

Interius BioTherapeutics Raises $76M Series A Financing to Advance Breakthrough Cell and Gene Therapy Platform


May 11, 2021

M6P Therapeutics Presents M002 Gene Therapy Preclinical Proof-of-Concept Data for the Treatment of Mucolipidosis Type II at the ASGCT 24th Annual Meeting


May 6, 2021

Dyno Therapeutics Closes $100 Million Series A Financing Led by Andreessen Horowitz to Accelerate AI‑powered Gene Therapy Platform


May 6, 2021

Jasper Therapeutics and Amplitude Healthcare Acquisition Corporation Announce Merger to Create a Publicly Listed Leading Biotechnology Company in Hematopoietic Stem Cell Transplantation


May 5, 2021

Excision BioTherapeutics Adds Gene Therapy Expert Nicole Paulk, Ph.D., to its Scientific Advisory Board



May 4, 2021

Myeloid Therapeutics Appoints Leading Immunologist, Cell and Gene Therapy Expert Bruce McCreedy, Ph.D., as Chief Scientific Officer


Apr 29, 2021

Chardan Acts as Exclusive Placement Agent in Forge Biologics' $120 Million Series B Financing


Apr 29, 2021

Capsida Biotherapeutics Debuts with $140 Million of Capital


Apr 27, 2021

ElevateBio’s BaseCamp Selected as the 2021 Facility of the Year Award Category Winner for Operational Excellence by the International Society for Pharmaceutical Engineering


Apr 21, 2021

Apic Bio Announces FDA Clearance of IND Application for Lead Gene Therapy Candidate APB-102 for the Treatment of SOD1 ALS



Apr 12, 2021

Mirum Pharmaceuticals and Vivet Therapeutics Enter into Exclusive Worldwide Option and License Agreement for Vivet’s Gene Therapy Programs Targeting Progressive Familial Intrahepatic Cholestasis


Apr 1, 2021

Neurophth Announces IND Approval by the NMPA for Leber Hereditary Optic Neuropathy Gene Therapy


Mar 22, 2021

XyloCor Therapeutics Completes Oversubscribed $41.9 Million Series A Financing To Advance Novel Gene Therapy Pipeline For Coronary Artery Disease


Mar 16, 2021

StrideBio Announces Closing of $81.5M Series B Financing


Mar 16, 2021

Be Bio Announces Appointment of Cell and Gene Therapy Veterans Joanne Smith-Farrell, Ph.D., as Chief Executive Officer and Rick Morgan, Ph.D., as Chief Scientific Officer



Mar 15, 2021

ElevateBio Scales Disruptive Cell and Gene Therapy Business Model with $525 Million Series C Financing


Mar 15, 2021

Tevogen Bio Announces Partnership With BioCentriq, New Jersey Innovation Institute’s Pioneering Cell and Gene Therapy Center, to Enhance Tevogen’s Development Capacity of Its Proprietary Targeted T Cell Therapy For COVID-19


Mar 1, 2021

LEXEO Therapeutics Announces License Agreement and Consolidation of Comprehensive Pre-clinical Data Package to Support Cardiac Friedreich's Ataxia Gene Therapy Program (LX2006)


Mar 1, 2021

SalioGen Therapeutics Announces Closing of $20 Million Series A Financing to Support Advancing a Novel Genome Engineering Platform for Non-Viral Gene Therapy


Feb 22, 2021

Anthony Nolan Cell and Gene Therapy Services and ONK Therapeutics Announce Collaboration



Feb 17, 2021

Affinia Therapeutics Announces Addition of Gene Therapy Scientific and Medical Experts to Leadership Team to Advance Novel Gene Therapy Platform and Programs to the Clinic


Feb 16, 2021

Forge Biologics Receives FDA Fast Track, Orphan Drug, and Rare Pediatric Disease Designations for FBX-101 Gene Therapy for Patients with Krabbe Disease


Feb 12, 2021

Gyroscope Therapeutics Announces Positive Interim Data from Phase I/II FOCUS Trial of Investigational Gene Therapy GT005


Jan 28, 2021

AskBio Adds CNS Gene Therapy Programs in Parkinson’s Disease and Multiple System Atrophy


Jan 28, 2021

M6P Therapeutics Receives Six Rare Pediatric Disease Designations from the U.S. FDA for Company’s Deep Pipeline of Programs for Lysosomal Storage Disorders



Jan 27, 2021

Neurophth Therapeutics Further Expands Ocular Gene Therapy Expertise with Appointment of Qiutang Li, Ph.D., as Chief Scientific Officer


Jan 25, 2021

Nanobiotix Subsidiary Curadigm Secures New Collaboration Agreement With Sanofi Focused on Gene Therapy Pipeline


Jan 25, 2021

Curadigm Announces Collaboration With Sanofi as Part of the Sanofi iTech Awards Program


Jan 25, 2021

Nanoscope Therapeutics Receives Orphan Drug Designation for gene therapy of blindness


Jan 22, 2021

Xalud Therapeutics Appoints Diem Nguyen, Ph.D., as Chief Executive Officer



Jan 19, 2021

Neurophth and AAVnerGene Enter Strategic AAV Capsids Partnership for Next-Generation Ophthalmic Gene Therapy


Jan 18, 2021

Ixaka (formerly Rexgenero) Launches as an Integrated Cell and Gene Therapy Company


Jan 18, 2021

ViGeneron announces research collaboration with Daiichi Sankyo to evaluate vgAAV for novel ophthalmic gene therapy


Jan 12, 2021

Gene Therapy Leader Katherine High Joins AskBio as President, Therapeutics


Jan 5, 2021

Genenta to Take Part in Gene Therapy Panel and Two Virtual Investor Events in January 2021



Jan 5, 2021

ViGeneron signs global collaboration agreement for ophthalmic gene therapy development


Jan 4, 2021

Forge Biologics Announces FDA Clearance of Investigational New Drug Application for Phase 1/2 Clinical Trial (RESKUE) of FBX-101 Gene Therapy for Patients with Krabbe Disease USA - English España - español France - Français Deutschland - Deutsch


Dec 14, 2020

Locanabio Announces $100 Million Series B Financing to Advance Portfolio of Novel RNA-Targeted Gene Therapies for Neurodegenerative, Neuromuscular and Retinal Diseases


Nov 19, 2020

AskBio and Selecta Biosciences Receive Orphan Drug Designation for MMA-101 to Treat Methylmalonic Acidemia


Nov 16, 2020

ElevateBio and AgBiome Announce LifeEDIT Will Join ElevateBio’s Portfolio of Innovative Cell and Gene Therapy Companies



Nov 16, 2020

Aruvant Receives Orphan Designation from European Medicines Agency for Gene Therapy ARU-1801 for Sickle Cell Disease


Nov 9, 2020

Decibel Therapeutics Announces $82M Series D Financing to Advance Gene Therapies to Restore Hearing and Balance


Nov 5, 2020

Taysha Gene Therapies Bolsters Manufacturing Capacity Through Partnership with Catalent


Nov 2, 2020

Decibel Therapeutics Announces Exclusive Licensing Agreements for Hearing Loss Gene Therapy Technology


Oct 29, 2020

Novartis Acquires Vedere Bio, a Novel Optogenetics AAV Gene Therapy Company



Oct 28, 2020

Gyroscope Therapeutics Announces First Patient Received Investigational Gene Therapy GT005 Via Orbit Subretinal Delivery System in Ongoing Phase I/II FOCUS Trial


Oct 22, 2020

Gene Therapy Company AavantiBio Launches with $107 Million Series A Financing from Perceptive Advisors, Bain Capital Life Sciences, RA Capital Management and Sarepta Therapeutics


Oct 20, 2020

Selecta Biosciences and AskBio Receive FDA Rare Pediatric Disease Designation for their Gene Therapy for Methylmalonic Acidemia


Oct 14, 2020

Dyno Therapeutics Enters Collaboration and License Agreement With Roche to Develop Next-Generation AAV Gene Therapy Vectors for CNS Diseases and Liver-Directed Therapies


Oct 12, 2020

Sickle Cell Disease Association of America and Aruvant Sciences Forge New Partnership to Educate Around Gene Therapy



Sep 24, 2020

Neurophth Therapeutics' Treatment of Leber's Hereditary Optic Neuropathy Gene Therapy NR082 was Granted Orphan Drug Designation by U.S. FDA


Sep 23, 2020

Vivet Therapeutics and Pfizer Inc. Enter into Manufacturing Agreement for Vivet’s Investigational Gene Therapy for Wilson Disease


Sep 22, 2020

Gyroscope Therapeutics Granted FDA Fast Track Designation for GT005, an Investigational Gene Therapy for Dry Age-Related Macular Degeneration


Sep 16, 2020

Forge Biologics Strengthens Gene Therapy Leadership with Key Hires in AAV Manufacturing, Regulatory Affairs and Finance to Support Strategic Growth


Aug 31, 2020

Taysha Gene Therapies Builds Experienced Executive Leadership Team to Advance Pipeline of Gene Therapies for Monogenic CNS Disease in Both Rare and Large Patient Populations



Aug 13, 2020

4D Molecular Therapeutics Receives FDA Fast Track Designation for 4D-310 Gene Therapy for Treatment of Fabry Disease


Jul 21, 2020

XyloCor Therapeutics Doses Patients in Phase 1/2 Trial Evaluating Novel Gene Therapy XC001 in Refractory Angina


Jun 16, 2020

4D Molecular Therapeutics Raises $75 Million in Series C Financing


Jun 1, 2020

Aruvant Appoints Dr. Palaniappan as Chief Technology Officer


May 29, 2020

Taysha Gene Therapies Launches with $30 Million Seed Financing and Unrivaled Strategic Partnership with the UT Southwestern Gene Therapy Program



May 22, 2020

AskBio Acquires BrainVectis to Expand its Clinical Pipeline for Neurodegenerative Diseases


May 19, 2020

AveXis receives EC approval and activates “Day One” access program for Zolgensma, the only gene therapy for spinal muscular atrophy (SMA)


May 11, 2020

Dyno Therapeutics Emerges from Stealth to Transform Gene Therapy Using Artificial Intelligence


May 11, 2020

Akouos to Present Data from Inner Ear Gene Therapy Platform at 23rd ASGCT Annual Meeting


May 11, 2020

Aruvant Appoints Joseph McIntosh, M.D. as Chief Medical Officer



Apr 28, 2020

Denovo Biopharma To Acquire Tocagen's Entire Replicating Gene Therapy Platform and Related Assets


Apr 23, 2020

SwanBio Therapeutics Announces Series A Expansion to Fund its Lead Program and Grow Pipeline in Gene Therapy for Neurological Diseases


Mar 30, 2020

Generation Bio and Vir Biotechnology to Collaborate on Research to Leverage Scalable Non-Viral Gene Therapy Platform for Durable Production of Monoclonal Antibodies Against Coronavirus That Causes COVID-19


Mar 18, 2020

HORAMA Signs Exclusive License Agreement with Leiden University Medical Center Targeting CRB1 Gene Mutations to Treat Inherited Retinal Dystrophies


Mar 18, 2020

AskBio Enters Research Collaboration and Licensing Agreement with University of North Carolina (UNC) for Angelman Syndrome



Mar 12, 2020

Tim Kelly Joins AskBio as President of Manufacturing to Lead AAV Vector Production for Gene Therapy


Mar 5, 2020

PsiOxus Therapeutics Announces Clinical Trial with Third Cancer Gene Therapy Treatment and Appointment of New Chief Medical Officer


Mar 3, 2020

Senti Biosciences Announces Dr. Jose Iglesias as Chief Medical Officer


Mar 2, 2020

CYTOO and AskBio Enter Research Agreement to Screen Gene Therapy Candidates for Rare Muscle Disorder


Feb 4, 2020

AskBio Announces First Patient Dosed in Phase 1 Trial Using AAV Gene Therapy for Congestive Heart Failure



Jan 29, 2020

AskBio Launches AskFirst™ Patient Advocacy Program to Educate and Support Participants in Gene Therapy Clinical Trials


Jan 14, 2020

ASC Therapeutics and Vigene Announce Long-Term Strategic Partnership for Gene Therapy Development and Manufacturing


Jan 7, 2020

Innovative Cellular Therapeutics Appoints Cell and Gene Therapy Manufacturing Expert Christopher Ballas, Ph.D., as SVP of Manufacturing


Dec 16, 2019

Castle Creek Pharmaceutical Holdings Completes Acquisition of Fibrocell Science


Dec 5, 2019

Limelight Bio Emerges with $75 Million to Develop Next Generation Gene Therapies to Overcome Limitations of Current Treatments



Nov 28, 2019

ViGeneron announces closing of series A financing to drive development of next generation gene therapy pipeline


Nov 21, 2019

CODA Biotherapeutics Deepens Gene Therapy Expertise with Industry Veteran, Annahita Keravala, Ph.D., and Key Promotions


Nov 19, 2019

AskBio Receives "Best Venture Funding" Award from Southeast BIO


Nov 19, 2019

Aruvant Appoints William Chou as Chief Executive Officer


Nov 7, 2019

SQZ Biotech and AskBio Announce Research Collaboration to Create Immune Tolerization Products for AAV Gene Therapies



Nov 7, 2019

SQZ Biotech and AskBio Announce Research Collaboration to Create Immune Tolerization Products for AAV Gene Therapies


Nov 6, 2019

Passage Bio to Present at Barclays Gene Editing & Gene Therapy Summit


Nov 6, 2019

Passage Bio to Present at Barclays Gene Editing and Gene Therapy Summit


Nov 5, 2019

CODA Biotherapeutics Announces Series A Extension, Bringing Total Round to $34 Million


Nov 5, 2019

Shape Therapeutics, Inc. Raises $35.5M Series A Financing, Led by NEA and Announces the Formation of a World-Class Scientific Advisory Board, to Advance a First-in-Class RNA Editing Gene Therapy Platform Focused on Curing Genetic Diseases



Oct 7, 2019

Decibel Therapeutics to Provide Corporate Update at Jefferies Gene Therapy Summit


Oct 1, 2019

ElevateBio Launches HighPassBio to Advance Novel Targeted T Cell Immunotherapies with Technology from Fred Hutchinson Cancer Research Center


Oct 1, 2019

Vor Biopharma Hires Senior Cell and Gene Therapy Leaders as Chief Technology Officer and Vice President of Research


Sep 17, 2019

AskBio Acquires Assets from RoverMed BioSciences, Adds Nanotechnology Precision to Delivery of AAV Gene Therapeutics


Sep 12, 2019

Akouos Discloses Lead Program, AK-OTOF, a Potential Gene Therapy for Sensorineural Hearing Loss



Sep 9, 2019

Passage Bio Announces New Gene Therapy Development Program for Patients with Charcot-Marie-Tooth Neuropathy Type 2A


Aug 22, 2019

AskBio Applauds Pfizer's Continuing Investments in Gene Therapy


Aug 13, 2019

AskBio Acquires Synpromics Ltd. and Expands its Gene Therapy Technology Portfolio


Aug 7, 2019

Selecta Biosciences Combines ImmTOR™ Platform with AskBio’s Industry-Leading Gene Therapy Platform in Strategic Partnership


Jul 26, 2019

Xynomic Pharma Will Present at 3rd World-China Immunotherapy & Gene Therapy Congress



Jul 26, 2019

Xynomic Pharma Will Present at 3rd World-China Immunotherapy and Gene Therapy Congress


Jul 10, 2019

Passage Bio Announces New Dedicated Gene Therapy Manufacturing Suite


Jul 2, 2019

HIV Cure Program of American Gene Technologies Adds University of Maryland School of Medicine’s Institute of Human Virology as New Study Site to Support the Development of a Cell and Gene Therapy for HIV


Jun 13, 2019

DPS Group and TRIA Designing ElevateBio's cGMP Manufacturing "Basecamp" Facility in Waltham


Jun 4, 2019

Prevail Therapeutics Announces IND Active for Phase 1/2 Trial of its Gene Therapy PR001 to Treat Parkinson’s Disease Patients with GBA1 Mutations



May 30, 2019

Passage Bio Announces Third Gene Therapy Development Program in Krabbe Disease and Supports Million Dreams Fundraising Gala


May 22, 2019

ElevateBio Announces that AlloVir Joins its Portfolio of Highly Innovative Cell and Gene Therapy Companies


May 16, 2019

ASC Therapeutics Licenses Critical Technologies for Hemophilia A Gene Therapy from Expression Therapeutics


May 15, 2019

Fibrocell Announces Collaboration with Castle Creek Pharmaceuticals to Develop and Commercialize FCX-007 Gene Therapy


May 13, 2019

ElevateBio Launches to Create and Operate a Portfolio of Highly Innovative Cell and Gene Therapy Companies



May 11, 2019

AskBio Partners with TPG and Vida Ventures


Apr 24, 2019

Rexgenero Presents Update on Phase III Clinical Trials with REX-001, a Cell-based Therapy for Critical Limb Ischaemia


Apr 23, 2019

Vivet Therapeutics Announces 2 Abstracts Accepted for Oral Presentation at 2019 American Society of Gene and Cell Therapy Annual Meeting


Apr 5, 2019

Dystrogen Therapeutics Announces That Gene Therapy Successfully Cuts Off Production of Neuron-Destroying Protein in Huntington's Disease


Mar 25, 2019

Alcyone Lifesciences To Present on Precision Therapy Delivery Technologies for Antisense Oligonucleotide and Gene Therapy at the 2019 Oligonucleotide Discovery and Delivery Conference.



Mar 20, 2019

Pfizer Secures Exclusive Option to Acquire Gene Therapy Company Vivet Therapeutics


Mar 13, 2019

Tenaya Therapeutics Strengthens Leadership Team with Appointment of Two Biopharmaceutical Industry Veterans


Feb 14, 2019

Passage Bio Launches with $115.5 Million Series A to Develop AAV-Delivered Therapeutics to Treat Rare Monogenic CNS Diseases


Jan 23, 2019

Talee Bio Announces $4.5 Million Award from the Cystic Fibrosis Foundation to Advance Development of Two Gene Therapy Product Candidates


Jan 7, 2019

Apic Bio Announces $40M Series A Financing to Advance Novel Gene Therapies for Rare Genetic Disorders



Dec 6, 2018

XyloCor Therapeutics Raises $17 Million in Series A Financing to Advance Gene Therapy Portfolio in Cardiovascular Disease


Dec 3, 2018

Aruvant Sciences Presents Data from Ongoing Clinical Study of Lentiviral Gene Therapy RVT-1801 for Sickle Cell Disease at 60th Annual Meeting of the American Society of Hematology


Nov 26, 2018

Roivant Launches Aruvant Sciences and Roivant Foundation in Partnership with Cincinnati Children's Hospital Medical Center


Nov 15, 2018

PsiOxus Therapeutics to Collaborate with the Parker Institute for Cancer Immunotherapy on Virus-Based Gene Therapy to Treat Solid Tumors


Oct 23, 2018

Senti Biosciences Announces Dr. Gary Lee as Chief Scientific Officer



Oct 23, 2018

PolyPid Announces Intention to Submit Confidential Draft Registration Statement for Proposed U.S. Initial Public Offering


Oct 23, 2018

Cybrexa Therapeutics to Present First Efficacy and Safety Data From Its Lead Program CBX-12 and Initial Data From CBX-13 at the American Association for Cancer Research (AACR) Virtual Annual Meeting II


Sep 12, 2018

MPM Capital and Versant Ventures Launch CODA Biotherapeutics to Accelerate Development of Chemogenetic Gene Therapy Platform


Sep 7, 2018

CORRECTING and REPLACING CureDuchenne Ventures Commits $500,000 to 4D Molecular Therapeutics to Advance Next-Generation AAV Delivery of Gene Therapy


Sep 5, 2018

4D Molecular Therapeutics Raises $90 Million Series B Financing



Aug 7, 2018

Akouos Raises $50 Million Series A Financing and Expands Team to Advance Precision Genetic Therapies That Restore and Preserve Hearing


Jul 10, 2018

4D Molecular Therapeutics and MedImmune Collaborate to Design, Develop and Commercialize AAV Gene Therapy for Chronic Lung Disease


Jun 20, 2018

Precision BioSciences CSO to Join Gene Therapy Session at DIA 2018


May 15, 2018

Alcyone Lifesciences Announces Presentation on Intrathecal Physical Methods of Delivery of Gene Therapeutics at 2018 American Society of Cell and Gene Therapy Conference


May 14, 2018

Precision BioSciences to Present In Vivo Gene Therapy and Ex Vivo Cancer Immunotherapy Data at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting



May 14, 2018

Precision BioSciences to Present In Vivo Gene Therapy and Ex Vivo Cancer Immunotherapy Data at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting


May 2, 2018

Agilis Biotherapeutics Presents on Gene Therapy Pricing Strategies at the 2nd Annual Gene Therapy for Rare Disorders Summit


Apr 24, 2018

Agilis Biotherapeutics Presents Platform Paper on AADC Deficiency Gene Therapy at American Academy of Neurology Annual Meeting


Apr 12, 2018

ViaCyte to Present at Alliance for Regenerative Medicine's 6th Annual Cell & Gene Therapy Investor Day in New York City


Feb 27, 2018

Generation Bio Announces $100 Million Series B Financing to Advance GeneWave™ Platform for Re-Dosable Gene Therapy



Dec 6, 2017

Obsidian Therapeutics Announces $49.5 Million Series A Financing to Develop Controllable Cell and Gene Therapy Products


Dec 5, 2017

Fortress Biotech Forms Subsidiary Tamid Bio to Develop Novel AAV Gene Therapies in Orphan Diseases With Unmet Medical Needs


Nov 30, 2017

Akouos to Build Premier Gene Therapy Company Focused on Hearing and Balance Disorders with Backing from Leading Life Science Investors and Strategic Licenses from Massachusetts Eye and Ear and Lonza


Oct 31, 2017

Agilis Biotherapeutics Announces Orphan Product Designation Approval in Europe for the Treatment of Friedreich Ataxia


Oct 17, 2017

AskBio and Columbus Venture Partners Launch Gene Therapy Company Viralgen



Oct 3, 2017

Agilis Biotherapeutics Updates on Progress in CNS Gene Therapy Programs


Sep 19, 2017

4D Molecular Therapeutics Receives $3 Million Follow-On Funding from Cystic Fibrosis Foundation Therapeutics for Gene Therapy IND Candidate for Cystic Fibrosis


Sep 6, 2017

Agilis Biotherapeutics Updates Progress of its CNS Gene Therapy for AADC Deficiency


Aug 22, 2017

Apic Bio Launches to Advance First-in-Class Gene Therapy for Treatment of Alpha-1 Antitrypsin Deficiency (Alpha 1)


Aug 2, 2017

Agilis Biotherapeutics and Gene Therapy Research Institution Enter into Strategic Partnership



Jun 29, 2017

Alcyone Lifesciences Enters into a Feasibility Study Agreement with Pfizer to Evaluate Alcyone's proprietary Pulsar™ Advanced Intrathecal Precision Delivery Platform Technology for use in Gene Therapy Targeting Central Nervous System Disorders


Jun 20, 2017

Cell Medica Acquires WT1 Cancer Immunotherapy from Cell and Gene Therapy Catapult


May 10, 2017

Agilis Biotherapeutics Announces Presentation of Data from Phase I/II Trial for Treatment of AADC Deficiency


Apr 24, 2017

ViaCyte to Present at ARM's 5th Annual Cell & Gene Therapy Investor Day in Boston


Jan 5, 2017

4D Molecular Therapeutics Enrolls First Patient in Natural History Study in Lead Clinical Program to Develop Gene Therapy Treatment for Choroideremia



Nov 17, 2016

Agilis Biotherapeutics Achieves FDA “Rare Pediatric Disease” Designation for AADC Gene Therapy Candidate


Oct 11, 2016

Agilis Biotherapeutics Announces Authorization of Phase IIb Clinical Study of Gene Therapy for AADC Deficiency


Aug 2, 2016

Agilis Biotherapeutics Announces FDA Orphan Drug Designation for the Treatment of Friedreich’s Ataxia (FA)


Jun 27, 2016

Agilis Biotherapeutics, First Gene Therapy Company Selected for NIH Therapeutics for Rare and Neglected Diseases Program


Mar 22, 2016

Agilis Biotherapeutics and T-TOP Clinical Research Enter into an Agreement for the Development of Gene Therapy Treatment of AADC Deficiency



Feb 18, 2016

Agilis Biotherapeutics and Waisman Biomanufacturing Enter Into Exclusive Manufacturing Agreement for Friedreich’s Ataxia Gene Therapy


Jan 28, 2016

Agilis Biotherapeutics and National Taiwan University Enter into Worldwide, Exclusive License Agreement for the Gene Therapy Treatment of AADC Deficiency


Jul 21, 2015

Lysogene and Alcyone Lifesciences Enter into a Collaboration for Mucopolysaccharidosis Type IIIA (MPS IIIA)


Jul 7, 2015

Dr. Olivier Danos, Cell and Gene Therapy Pioneer, Joins Alcyone Lifesciences Scientific Advisory Board


Jun 25, 2015

PBM Capital Group Invests in Xalud Therapeutics Gene Therapy Platform for Treatment of Neuro-Inflammatory Diseases



May 27, 2015

4D Molecular Therapeutics and Roche Sign Collaboration Agreement for Gene Therapy Vector Discovery and Product Development


May 20, 2015

AGTC and 4D Molecular Therapeutics Sign Collaboration Agreement for Gene Therapy Vector Discovery and Product Development


May 13, 2015

Agilis Biotherapeutics and the University of South Florida Enter Into Worldwide, Exclusive License Agreement for Angelman Syndrome Gene Therapy


Jun 3, 2014

Dr. John Monahan, a Veteran in Biotechnology and Gene Therapy Companies, Joins the Scientific Advisory Board of Agilis Biotherapeutics


Jan 7, 2010

Cell Medica Announces RandD Framework Agreement with theCenter for Cell and Gene Therapy at Baylor College of Medicine