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

Retrieved on: 
Tuesday, October 10, 2023

In CD, normal brain development is impaired due to a mutation in the ASPA gene that encodes the enzyme aspartoacylase.

Key Points: 
  • In CD, normal brain development is impaired due to a mutation in the ASPA gene that encodes the enzyme aspartoacylase.
  • Following gene therapy, statistically significant gains in certain target white matter and myelin were observed.
  • In turn, improvements in multiple domains on the validated Gross Motor Function Measure and Mullen Scales of Early Learning were demonstrated.
  • Treated patients were routinely observed to outperform untreated age-matched natural history patients on the Mullen Scale of Early Learning.

BridgeBio and Resilience Announce Strategic Multi-Year Partnership to Advance BBP-631, BBP-812 and Future Gene Therapy Treatments

Retrieved on: 
Tuesday, October 3, 2023

Under the terms of the collaboration, BridgeBio will transfer its manufacturing process for its lead AAV-based gene therapy candidates to Resilience’s network of gene therapy sites.

Key Points: 
  • Under the terms of the collaboration, BridgeBio will transfer its manufacturing process for its lead AAV-based gene therapy candidates to Resilience’s network of gene therapy sites.
  • “Our partnership with BridgeBio seeks to accelerate development of innovative therapeutic options for patients in need,” said Rahul Singhvi, Sc.D., Chief Executive Officer of Resilience.
  • “Manufacturing is the most critical and costly aspect of developing gene therapy for patients with a serious unmet need.
  • “We appreciate the opportunity to collaborate with the experienced and knowledgeable team at Resilience on the manufacturing of our gene therapies.

BridgeBio and Resilience Announce Strategic Multi-Year Partnership to Advance BBP-631, BBP-812 and Future Gene Therapy Treatments

Retrieved on: 
Tuesday, October 3, 2023

Under the terms of the collaboration, BridgeBio will transfer its manufacturing process for its lead AAV-based gene therapy candidates to Resilience’s network of gene therapy sites.

Key Points: 
  • Under the terms of the collaboration, BridgeBio will transfer its manufacturing process for its lead AAV-based gene therapy candidates to Resilience’s network of gene therapy sites.
  • “Our partnership with BridgeBio seeks to accelerate development of innovative therapeutic options for patients in need,” said Rahul Singhvi, Sc.D., Chief Executive Officer of Resilience.
  • “Manufacturing is the most critical and costly aspect of developing gene therapy for patients with a serious unmet need.
  • “We appreciate the opportunity to collaborate with the experienced and knowledgeable team at Resilience on the manufacturing of our gene therapies.

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

Retrieved on: 
Thursday, September 28, 2023

These improvements in treated patients contrast the deterioration in untreated age-matched CD patients based on natural history.

Key Points: 
  • These improvements in treated patients contrast the deterioration in untreated age-matched CD patients based on natural history.
  • MRI-based improvements are correlated with improvements in motor and cognitive function in treated patients, which will be presented at upcoming conferences.
  • In CD, normal brain development is impaired due to a mutation in the ASPA gene that encodes the enzyme aspartoacylase.
  • The oligodendrocyte-targeting rAAV vector-based gene therapy is intended to restore ASPA function and brain development in patients with CD.

Burjeel Holdings to Launch Rare Disease Research & Development Project ‘NADER’ in Partnership with US-based BridgeBio Pharma

Retrieved on: 
Wednesday, July 12, 2023

ABU DHABI, United Arab Emirates and PALO ALTO, Calif., July 12, 2023 (GLOBE NEWSWIRE) -- Burjeel Holdings, one of the largest healthcare providers in the MENA region, and BridgeBio Pharma, Inc., a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, have announced a new project to revolutionize the field of early diagnosis and treatment of rare diseases or disorders in the UAE and the region. The two entities signed a preliminary, non-binding Collaboration Agreement establishing a mutual intention to work together on project ‘NADER’ (Needs Assessment and Therapeutics Development for Rare Diseases – ‘nader’ meaning ‘rare’ in Arabic). Genetic in origin, these often life-threatening or chronically debilitating diseases affect a small percentage of the population and are present throughout the person's entire life, even if symptoms do not immediately appear. The partnership will launch operations in Abu Dhabi to conduct clinical trials and research, leveraging the Emirate’s advanced infrastructure for innovation and life science.

Key Points: 
  • The partnership will launch operations in Abu Dhabi to conduct clinical trials and research, leveraging the Emirate’s advanced infrastructure for innovation and life science.
  • Project ‘NADER’ aims to revolutionize the field of early diagnosis and treatment in order to potentially improve patient outcomes.
  • Dr. Khaled Musallam, Group Chief Research Officer of Burjeel Holdings and project lead said, “Many rare diseases are highly clustered in our region due to our unique genetic pool.
  • As the collaboration unfolds, both Burjeel Holdings and BridgeBio anticipate significant progress in the field of rare diseases in the region.

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

Retrieved on: 
Wednesday, May 31, 2023

Olga Flamini, MD, PhD, Medical Director at Myrtelle, will deliver a presentation on Friday, June 2, 2023.

Key Points: 
  • Olga Flamini, MD, PhD, Medical Director at Myrtelle, will deliver a presentation on Friday, June 2, 2023.
  • These improvements in treated patients contrast the deterioration in untreated age-matched CD patients in Myrtelle’s natural history data set.
  • In CD, normal brain development is impaired due to a mutation in the ASPA gene that encodes the enzyme aspartoacylase.
  • “Connecting with patients and caregivers allows us to incorporate the patient voice into our drug development activities.”

BridgeBio Pharma Presents Updated Encouraging Clinical and Biomarker Data from its BBP-812 Canavan Disease Gene Therapy Program at the 2023 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting

Retrieved on: 
Monday, May 22, 2023

“Canavan disease is a rapidly progressive, rare neurological disease that affects children from birth and has no treatment options beyond supportive care.

Key Points: 
  • “Canavan disease is a rapidly progressive, rare neurological disease that affects children from birth and has no treatment options beyond supportive care.
  • The changes in key biomarkers, brain myelination and – critically – clinical function observed in children receiving BBP-812 in our study are compelling, and as we continue to advance our program we are hopeful that we will see outcomes that make a meaningful difference in the lives of children with Canavan disease and their families,” said Genevieve Laforet, M.D., Ph.D., vice president of clinical development at Aspa Therapeutics, the BridgeBio affiliate that is developing BBP-812 for Canavan disease.
  • “We are beyond grateful to the children and their families who are participating in CANaspire as well as the study investigators, who together are making it possible to explore the potential of BBP-812 as a therapy for Canavan disease.
  • Across individual participants, percent decreases in NAA from baseline ranged between 70% and 95% in cerebrospinal fluid (CSF), 29% and 88% in urine, and 8% and 75% in brain (by magnetic resonance spectroscopy)
    All participants in the CANaspire trial had urine NAA levels consistent with typical Canavan disease prior to receiving BBP-812.

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

Retrieved on: 
Tuesday, May 16, 2023

Clinical measurements of motor and cognitive function using validated assessment scales demonstrate mean absolute and percent improvements across multiple domains.

Key Points: 
  • Clinical measurements of motor and cognitive function using validated assessment scales demonstrate mean absolute and percent improvements across multiple domains.
  • Improvements in multiple anatomic and biomarker measurements by magnetic resonance imaging (MRI) and spectroscopy (MRS) have also been observed.
  • These improvements in treated patients contrast the deterioration in untreated age-matched CD patients in Myrtelle’s natural history data set.
  • In CD, normal brain development is impaired due to a mutation in the ASPA gene that encodes the enzyme aspartoacylase.

BridgeBio Pharma to Present Updated Data from its BBP-812 Canavan Disease Gene Therapy Program at the 2023 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting

Retrieved on: 
Monday, May 15, 2023

PALO ALTO, Calif., May 15, 2023 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, announced today that updated data from CANaspire, its Phase 1/2 clinical trial of BBP-812, an investigational intravenous (IV) adeno-associated virus serotype 9 (AAV9) gene therapy for the treatment of Canavan disease, will be presented at the 2023 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, taking place in Los Angeles, CA on May 16 – 20, 2023.

Key Points: 
  • PALO ALTO, Calif., May 15, 2023 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, announced today that updated data from CANaspire, its Phase 1/2 clinical trial of BBP-812, an investigational intravenous (IV) adeno-associated virus serotype 9 (AAV9) gene therapy for the treatment of Canavan disease, will be presented at the 2023 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, taking place in Los Angeles, CA on May 16 – 20, 2023.
  • Additionally, members of BridgeBio leadership, including Neil Kumar, CEO and founder, will be presenting on multiple panels discussing drug development and accelerated approval for gene therapy.
  • Initial biomarker and clinical findings from the CANaspire Canavan disease gene therapy trial: Exploration of connections between NAA and disease severity
    Presenter: Florian Eichler, M.D., director of the leukodystrophy service at Massachusetts General Hospital, Center for Rare Neurological Disease and lead CANaspire investigator
    Location: Concourse Hall 150 & 151, Los Angeles Convention Center
    Location: Concourse Hall 152 & 153, Los Angeles Convention Center
    Accelerate your drug development program in rare disease with robust natural history data: An evolving space

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

Retrieved on: 
Wednesday, May 10, 2023

The symposium will highlight historical and emerging views of oligodendrocytes in normal brain function and a growing array of disease states, including leukodystrophies and disorders of myelin production such as multiple sclerosis.

Key Points: 
  • The symposium will highlight historical and emerging views of oligodendrocytes in normal brain function and a growing array of disease states, including leukodystrophies and disorders of myelin production such as multiple sclerosis.
  • Oligodendrocytes perform numerous key functions in the brain - including the production of myelin, the insulating material that enables proper neuronal function – and are now appreciated as having roles in a range of diseases.
  • Myrtelle is developing a unique toolkit, including a proprietary class of recombinant adeno-associated virus (rAAV) vectors that directly target these cells.
  • “Given the intractable nature of many neurological diseases, these cells represent important brain components worthy of interrogation in the quest for novel interventions, including gene therapies.