BridgeBio posts early data for gene therapy targeting Canavan disease [Seeking Alpha]
BridgeBio Pharma, Inc. (BBIO)
Last bridgebio pharma, inc. earnings: 3/2 08:29 am
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Source: Seeking Alpha
BridgeBio posts early data for gene therapy targeting Canavan diseaseJun. 22, 2022 11:12 AM ETBridgeBio Pharma, Inc. (BBIO)By:Dulan LokuwithanaSA News Editordesigner491/iStock via Getty ImagesThe shares of BridgeBio Pharma (NASDAQ:a Phase 1/2 clinical trial.An ultra-rare and fatal disorder, Canavan disease is caused by a mutation of the ASPA gene that codes for aspartoacylase (ASPA), a protein that breaks down a compound called N-acetylaspartate (NAA). Genetically inherited Canavan disease has no approved therapies.Highlights of the results include a 77% decline of NAA in the cerebrospinal fluid of the first patient six months after the treatment.Meanwhile, the second patient was found to have an 89% reduction of NAA in CSF at month three post-treatment.A decline in brain NAA signals that the AAV9-based gene therapy candidate BP-812 reached its target behind the blood-brain-barrier expressing the functional ASPA enzyme, BridgeBio (So far, none of the patients have experienced a drug-r
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News
- Sentynl Therapeutics Receives MHRA Authorization of NULIBRY® (fosdenopterin) for Treatment of MoCD Type A in Great Britain [Yahoo! Finance]Yahoo! Finance
- BridgeBio Pharma Reports Inducement Grants under Nasdaq Listing Rule 5635(c)(4)GlobeNewswire
- BridgeBio Pharma Presents Cardiac Magnetic Resonance (CMR) Imaging Evidence Consistent with Clinical Improvement Observed in the ATTRibute-CM Phase 3 Study in Patients with Transthyretin Amyloid Cardiomyopathy (ATTR-CM) [Yahoo! Finance]Yahoo! Finance
- BridgeBio Pharma Presents Cardiac Magnetic Resonance (CMR) Imaging Evidence Consistent with Clinical Improvement Observed in the ATTRibute-CM Phase 3 Study in Patients with Transthyretin Amyloid Cardiomyopathy (ATTR-CM)GlobeNewswire
- Noetik Appoints Dr. Shafique Virani as Chief Business Officer [Yahoo! Finance]Yahoo! Finance
BBIO
Earnings
- 2/22/24 - Miss
BBIO
Sec Filings
- 3/14/24 - Form 4
- 3/14/24 - Form 4
- 3/6/24 - Form 424B5
- BBIO's page on the SEC website