News

• (07.09.2026)
• (Canavan Disease)

Beyond the Biomarkers

As we work to advance gene therapy for rare, fatal genetic conditions such as Canavan disease, it is easy to become focused on the data points and statistics that are essential for regulatory progress. Yet those numbers tell only part of the story.

• (06.09.2026)
• (Canavan Disease)

Proven Durability: Groundbreaking Gene Therapy Persistence Data

Groundbreaking data has offered new insights into Canavan disease an ultra rare and fatal genetic brain disorder.

• (05.19.2026)
• (Canavan Disease)

Real-World Signals from Treated Children

Clinical trials are signaling fascinating insights into Canavan diease, an ultra rare and fatal genetic brain disorder.

• (05.11.2026)
• (Press Release)

Myrtelle to Present Clinical Data and Regulatory Progress for MYR-101 Gene Therapy Program at ASGCT 2026

Myrtelle has been conducting clinical trials and changing the landscape of Canavan disease an ultra rare and fatal genetic brain disorder.

• (04.30.2026)
• (Uncategorized)

Irish Baby Among World’s First to Receive Experimental Gene Therapy

An Irish baby received experimental gene therapy. It targets Canavan disease, an ultra-rare, fatal genetic brain disorder with no known cure yet.

• (04.29.2026)
• (Uncategorized)

The Next Frontier in Newborn Screening and Why It Matters for Canavan Families

• (03.25.2026)
• (Uncategorized)

The Rare Disease Conversation Is Getting Louder. Here’s Where Myrtelle Stands.

• Jordana Holovach (03.05.2026) 3
• (Press Release)

Rare Disease Day: Lighting the Path for the Canavan Community

• Jordana Holovach (10.06.2025) 3
• (Press Release)

Myrtelle Announces Presentation of Encouraging Interim Results from its Phase 1/2 Gene Therapy Trial for Canavan Disease at the 2025 Cell & Gene Meeting on the Mesa

• Jordana Holovach (09.16.2025) 3
• (Uncategorized)

Myrtelle Announces Nature Medicine Publication of Interim Results from Its Phase 1/2 Clinical Trial of Investigational Gene Therapy rAAV-Olig001-ASPA for Canavan Disease

• Jordana Holovach (07.08.2025) 3
• (Canavan Disease)

Myrtelle Launches Commercial-Stage Manufacturing of First-in-Class Gene Therapy for Canavan Disease

• Jordana Holovach (05.13.2025) 3
• (Canavan Disease)

Myrtelle to Present Breakthrough Insights on FDA’s Groundbreaking START Pilot Program at the American Society of Gene and Cell Therapy (ASGCT) 2025 Annual Meeting in New Orleans on Thursday, May 15th

• Jordana Holovach (10.01.2024) 3
• (Canavan Disease)

Myrtelle Announces Significant Reduction in N-Acetylaspartate (NAA), a Key Biomarker, in Patients Treated in Its Phase 1/2 Clinical Trial of the Investigational Gene Therapy rAAV-Olig001-ASPA for Canavan Disease 

• Jordana Holovach (06.05.2024)
• (Press Release)

Myrtelle Announces that the FDA has Selected rAAV-Olig001-ASPA Gene Therapy Candidate for the Treatment of Canavan Disease for the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program

• Jordana Holovach (06.04.2024) 57 mins
• (Events)

Now Available: Myrtelle ASGCT Symposium Presentation

Myrtelle shared clinical trial updates and future directions for Oligodendrocyte targeting AAVs at the 27th Annual Meeting of the ASGCT.

• Jordana Holovach (05.07.2024) 3 mins
• (Events)

Myrtelle to Host a Symposium on Canavan Disease Clinical Trial Updates and Future Directions for Oligodendrocyte Targeting AAVs at the American Society of Gene and Cell Therapy 27th Annual Meeting in Baltimore on Wednesday, May 8, 2024

Join Myrtelle Inc. at the ASGCT 27th Annual Meeting on May 8, 2024, in Baltimore, for a pivotal symposium on Canavan disease. Explore the latest clinical trial updates and groundbreaking research on oligodendrocyte targeting AAVs in gene therapy.

• Jordana Holovach (04.02.2024) 3 mins
• (Q & A)

Myrtelle’s rAAV-Olig001-ASPA Gene Therapy Candidate for Canavan Disease Receives Regenerative Medicine Advanced Therapy (RMAT) Designation from the U.S. Food and Drug Administration

Myrtelle’s rAAV-Olig001-ASPA Gene Therapy Candidate for Canavan Disease Receives Regenerative Medicine Advanced Therapy (RMAT) Designation from the U.S. Food and Drug Administration

• Jordana Holovach (03.05.2024)
• (Canavan Disease)

Inheritance Patterns of Canavan Disease: Navigating Options When Only One Mutation is Identified

• Jordana Holovach (12.07.2023) 15 minutes
• (Events)

Exciting Update! Explore Myrtelle’s Insightful Slide Presentation on Gene Therapy for Canavan Disease Preliminary One-Year Results from Meeting on the Mesa

• Jordana Holovach (11.06.2023)
• (Events)

Myrtelle Announces Presentation at the 5th Annual Gene Therapy for Neurological Disorders Summit: Optimizing Gene Therapies Targeting the CNS to Deliver Safe & Efficacious Treatment Options For Neurological Disorder Patients

• Jordana Holovach (10.09.2023)
• (Canavan Disease)

Myrtelle Announces Presentation of Positive 12-month Post Treatment Data in its First-in-Human Clinical Study of rAAV-Olig001-ASPA Gene Therapy at the 2023 Cell & Gene Meeting on the Mesa held by the Alliance for Regenerative Medicine

• Jordana Holovach (10.06.2023)
• (Press Release)

Myrtelle Announces Presentation at Hydrocephalus Association & Rudi Schulte Research Workshop on Developing Non-Invasive Hydrocephalus Therapies: Molecular and Cellular Targets

• Jordana Holovach (06.06.2023) 1 hour 17 minutes
• (Events)

Watch Now: Myrtelle ASGCT Symposium Presentations

• (06.06.2023) 14 mins
• (Events)

Watch Now: Dr. Christopher Janson: ASGCT Presentation on Myrtelle’s Phase 1/2 Gene Therapy Clinical Trial in Patients with Canavan Disease

• Jordana Holovach (05.31.2023) 5 mins
• (Press Release)

Myrtelle to Present Positive 6-month Post-Treatment Data in Its First- in-Human Clinical Study of rAAV-Olig001-ASPA Gene Therapy in Canavan Disease at the National Tay-Sachs and Allied Diseases Annual Family Conference

• Jordana Holovach (05.16.2023) 5 mins
• (Press Release)

Myrtelle Announces Presentation of Positive 6-month Post-Treatment Data in Its First-in-Human Clinical Study of rAAV-Olig001-ASPA Gene Therapy in Canavan Disease at the American Society of Gene and Cell Therapy 26th Annual Meeting

• Jordana Holovach (05.10.2023) 3 mins
• (Events)

Myrtelle to Host Symposium at ASGCT

Myrtelle to Host Symposium on Gene Therapies Targeting Oligodendrocytes and Implications in Brain Function and Disease States at the American Society of Gene and Cell Therapy 26th Annual Meeting in Los Angeles on Friday, May 19, 2023

• Jordana Holovach (04.25.2023) 3 minutes
• (Press Release)

Myrtelle’s rAAV-Olig001-ASPA Gene Therapy Candidate for Canavan Disease Receives Innovative Licensing and Access Pathway Designation from the UK Medicines and Healthcare Products Regulatory Agency

Myrtelle Inc., (“Myrtelle” or the “Company”), a clinical stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, today announced that the Medicines and Healthcare Products Regulatory Agency (MHRA), the healthcare regulatory body of the United Kingdom (UK), granted Innovative Licensing and Access Pathway (ILAP) designation to the…

• Jordana Holovach (04.20.2023) 4 minutes
• (Canavan Disease)

Myrtelle Announces Positive 6-month Post-Treatment Data in Patients in Its First-in-Human Clinical Study of rAAV-Olig001-ASPA Gene Therapy in Canavan Disease

Analyses at 6 months showed increases in myelin, white matter and other MRI and MRS measurements as well as functional improvements on validated assessment scales Encouraging initial efficacy data support further development of rAAV-Olig001-ASPA as a potential therapeutic approach for children with Canavan disease

• Jordana Holovach (01.11.2023) 3 minutes
• (Press Release)

Myrtelle and rAAVen Therapeutics to Develop Novel Gene Therapy Vectors

Wakefield, Mass., January 11th, 2023 – Myrtelle Inc.,(“Myrtelle”) a clinical stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, and rAAVen Therapeutics, (“rAAVen”) an AAV engineering company focused on generating innovative vectors for the next generation of gene therapies, today announced a partnership to develop novel recombinant adeno-associated virus (“rAAV”) vectors to…

• Jordana Holovach (01.10.2023) 4 minutes
• (Press Release)

Myrtelle Announces Positive Interim Data in Phase 1/2 Clinical Trial of Its Proprietary Investigational Gene Therapy rAAV-Olig001-ASPA in Canavan Disease

Phase 1/2 clinical study is evaluating Myrtelle’s first-of-its-kind proprietary adeno-associated virus (rAAV) vector designed to enable targeting of oligodendrocytes Eight patients have been treated in the ongoing Phase 1/2 First-in-Human clinical trial with favorable findings observed to date Assessments of all treated patients through three-months of follow-up show improvements in neuroimaging and functional scales Wakefield,…

• Jordana Holovach (12.14.2022) 3 mins
• (Press Release)

Myrtelle’s rAAV-Olig001-ASPA Gene Therapy Candidate for Canavan Disease Receives Orphan Drug Designation from the European Medicines Agency

WAKEFIELD, Mass.–(BUSINESS WIRE)–Myrtelle Inc., (“Myrtelle” or the “Company”), a clinical stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, today announced that the European Medicines Agency (EMA) has granted orphan drug (OD) designation for rAAV-Olig001-ASPA, the company’s lead gene therapy product candidate for the treatment of Canavan disease. The OD application is…

• Jordana Holovach (12.14.2022) 3 mins
• (Canavan Disease)

Gene Therapy – Measuring its Impact with Dr. Christopher Janson

Dr. Christopher Janson, the Principal Investigator on Myrtelle’s Canavan Disease gene therapy trial, addresses questions frequently asked by the families in relation to the disease and gene therapy

• Jordana Holovach (10.04.2022) 4 mins
• (Canavan Disease)

Myrtelle Completes Dosing of 8 Patients with Canavan Disease in Its Phase 1/2 Clinical Trial of the Investigational Gene Therapy rAAV-Olig001-ASPA

Myrtelle Inc. has successfully completed dosing 8 patients with Canavan Disease in its Phase 1/2 clinical trial for the investigational gene therapy rAAV-Olig001-ASPA, demonstrating favorable safety and tolerability, along with significant improvements in functional scales and brain white matter content in early assessments.

• Jordana Holovach (10.03.2022) 2 mins
• (Press Release)

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

Myrtelle Inc. and Forge Biologics have announced a manufacturing partnership to advance Myrtelle’s novel gene therapy for monogenic hearing loss, Myr-201, into clinical trials for patients with autosomal recessive deafness 8 (DFNB8).

• Jordana Holovach (09.09.2022) 4 mins
• (Canavan Disease)

Myrtelle’s rAAV-Olig001-ASPA Gene Therapy Candidate for Canavan Disease Receives Advanced Therapy Medicinal Product Classification from the European Medicines Agency

Myrtelle Inc. has received Advanced Therapy Medicinal Product (ATMP) classification from the European Medicines Agency (EMA) for its lead gene therapy product candidate, rAAV-Olig001-ASPA, aimed at treating Canavan disease, facilitating its regulatory pathway and development in the EU.

• Jordana Holovach (08.15.2022) 1 mins
• (Gene Therapy)

Our Approach Has The Potential To Correct Myelin-Based Deficits In A Range Of Disorders

• Jordana Holovach (07.08.2022) 4 mins
• (Canavan Disease)

Myrtelle Announces Positive Data for Its investigational Proprietary rAAV-Olig001-ASPA Gene Therapy in Canavan Disease at the National Tay Sachs & Allied Diseases Association Conference

Analyses of the first three patients at 6 months post-treatment showed increases in white matter and myelin and improvements on validated functional scales To date, 5 patients have been treated in the ongoing Phase 1/2 First-in-Human clinical trial with favorable safety and tolerability

• Jordana Holovach (05.19.2022) 3 mins
• (Gene Therapy)

Myrtelle Enters into a Worldwide Exclusive License Agreement with Rescue Hearing to Develop and Commercialize Gene Therapy for the Treatment of Hearing Loss

Myrtelle Inc. has entered into a worldwide exclusive licensing agreement with Rescue Hearing Inc. to develop a novel gene therapy for DFNB8 genetic hearing loss, aiming to repair and restore damaged cells and promote neuron survival, offering potential hope for patients with this condition.

• Pelizaeus-Merzbacher Disease (05.12.2022) 5 mins
• (Gene Therapy)

Myrtelle Inc. Announces Expansion of Its Pipeline with a Novel Gene Therapy Program for Pelizaeus-Merzbacher Disease (PMD)

Myrtelle Inc. announced the launch of a new research program for Pelizaeus-Merzbacher Disease (PMD), a rare brain disorder, using gene therapy to target oligodendrocytes and address the underlying cause of myelin production disruption.

• Jordana Holovach (03.15.2022) 4 mins
• (Canavan Disease)

Myrtelle Receives FDA Fast Track, Rare Pediatric Disease, and Orphan Drug Designations for its Proprietary Gene Therapy for the Treatment of Canavan Disease

Myrtelle Inc., a gene therapy company, has received FDA Fast Track, Rare Pediatric Disease, and Orphan Drug designations for its gene therapy aimed at treating Canavan Disease, highlighting the urgency of developing a treatment for this devastating childhood disease.

• Jordana Holovach (02.15.2022) 3 mins
• (Canavan Disease)

Myrtelle Announces Positive Preliminary Clinical Data for Its Proprietary Gene Therapy in Canavan Disease

Myrtelle’s Phase 1/2 clinical trial introduces first-of-its-kind proprietary recombinant adeno-associated virus (rAAV) vector designed to enable targeting of oligodendrocytes.

• Jordana Holovach (01.11.2022) 3 mins
• (Canavan Disease)

Myrtelle Announces Successful Completion of Initial Stage of Phase 1/2 Clinical Trial of Proprietary Gene Therapy for Canavan Disease and Expands Treatment to Younger Patients

Myrtelle Inc. has announced the successful administration of gene therapy in its Phase 1/2 clinical trial for Canavan disease, treating three children aged 3 to 5 years, with plans to expand the study to younger age groups based on recommendations from the Data Monitoring Committee.

• Jordana Holovach (12.14.2021) 2 mins
• (Canavan Disease)

Myrtelle Announces Exclusive Worldwide Gene Therapy Licensing Agreement for Canavan Disease

Myrtelle Inc. has entered into an exclusive worldwide licensing agreement with Pfizer Inc. for an investigational recombinant adenovirus (rAAV) gene therapy aimed at addressing Canavan disease (CD), a devastating childhood genetic neurological disorder.

• Jordana Holovach (12.07.2021) 1 mins
• (Gene Therapy)

Health Care Hero: Dayton Children’s doctor offers hope, groundbreaking clinical trials for patients

Dr. Robert Lober is a leading pediatric neurosurgeon and brain cancer researcher at Dayton Children’s Hospital.