Year: 2024

Aspa Therapeutics: A Study of AAV9 Gene Therapy in Children With Canavan Disease

Clinical Trials / November 25, 2024

Aspa Therapeutics: A Study of AAV9 Gene Therapy in Children With Canavan Disease (CANaspire Clinical Trial) [https://treatcanavan.com/canaspire-trial/]   Aspa’s investigational gene therapy trial uses an AAV9 vector (adeno-associated virus serotype 9), which is designed to deliver functional copies of the ASPA gene throughout the body, including into the brain, to address the underlying cause of Canavan disease. The AAV9 …

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Aspa Therapeutics: A Natural History Study of Patients With Canavan Disease

News / September 24, 2024

Aspa Therapeutics: A Natural History Study of Patients With Canavan Disease (CANinform Study) [https://clinicaltrials.gov/study/NCT04126005] CANinform is a natural history study of patients with Canavan disease that will help researchers better understand the disease and provide a foundation for Aspa’s gene therapy program. The study is open to individuals with a confirmed diagnosis of Canavan disease.

BridgeBio press release about the RMAT designation for the Aspa clinical trial in Canavan Disease

News / September 16, 2024

09.10.2024 at 4:45 PM EDT – Receipt of RMAT Designation is based on preliminary clinical evidence from the CANaspire Phase 1/2 clinical trial, which showed functional improvements in all dosed patients indicating that BBP-812 has potential to address the unmet needs of individuals with Canavan disease – BridgeBio will leverage the benefits of RMAT designation, …

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In Memorial and Honor: May – July 2024

In Memory or In Honor Donation / August 12, 2024

ULF received donations in memorial or honorarium for the following during the months of May through July 2024. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at [email protected] or by calling 815-748-3211 In Honor of Dan Mulligan Troy Glendenning Tara Nunez Quantum Crossing, LLC Master Built …

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An Update from Passage Bio on Out-Licensing of Pediatric Programs to GEMMA Biotherapeutics

News / August 2, 2024

On August 1, 2024, Passage Bio shared they entered into a new agreement with GEMMA Biotherapeutics for the out-licensing of three pediatric gene therapy programs. Through this transaction, Passage Bio has granted GEMMA Biotherapeutics the rights for the development and commercialization of PBGM01 for GM1 gangliosidosis, PBKR03 for Krabbe disease, and PBML04 for metachromatic leukodystrophy.  …

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In Memorial and Honor – March 2024

In Memory or In Honor Donation / April 22, 2024

ULF received donations in memorial or honorarium for the following during the month of March 2024. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at [email protected] or by calling 815-748-3211 In Honor of BRAMBLEBOX TURTLE Melissa Kapoor Daxton Wallace Sandra Wallace Liz Thornton Kevin Dewhurst Mygelybelly …

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