隶属于 BAYER AG
相关临床试验
6
2 进行中
药物批准
0
批准总数
监管机构
0
监管机构数
成立时间
2001
进行中（未招募）
2
33.3%
已完成
3
50.0%
招募中
1
16.7%
暂无批准数据
- EpilepsyGTx and CDMO Viralgen have partnered to manufacture EPY201, an investigational AAV gene therapy for focal refractory epilepsy, ahead of first-in-human clinical trials. - Viralgen will deploy its proprietary Aava platform, which enables high-yield, scalable AAV manufacturing across serotypes and has supported production of 1,500 AAV batches. - EPY201 (AAV9-CAMK2A-EKC) is delivered directly into the seizure focus via intraparenchymal administration to reduce local neuronal hyperexcitability while limiting exposure beyond the target region. - Focal refractory epilepsy affects roughly 10 million patients worldwide, including two million across the US, UK and EU, and the first-in-human trial is expected to begin in the first half of 2027.
- AskBio presented baseline characteristics from the Phase 2 GenePHIT trial of the investigational gene therapy umiposgene parvec (AB-1002) at the European Society of Cardiology Congress in Munich, Germany. - GenePHIT randomized more than 170 participants across 64 sites in 12 countries, making it one of the largest gene therapy trials conducted in heart failure to date. - The enrolled population reflects a representative non-ischemic heart failure with reduced ejection fraction (HFrEF) cohort receiving guideline-directed medical therapy. - Initial efficacy and safety outcomes from GenePHIT are expected in the first half of 2027.
- Advocates living with limb-girdle muscular dystrophy from more than 20 states held over 60 congressional meetings during The Speak Foundation's LGMD Day on the Hill. - The community is pressing for federal research investment, expanded Department of Defense funding access, and clearer regulatory pathways for rare-disease treatments. - Momentum is building around what could be the first FDA-approved LGMD therapy, a treatment for the LGMD 2I/R9 subtype developed by BridgeBio. - The Speak Foundation honored Reps. John Joyce and Jake Auchincloss with its 2026 Congressional Champion for Limb-Girdle Muscular Dystrophy Awards.
- More than 300 million people worldwide live with one of over 7,000 identified rare diseases, yet diagnostic delays average four to five years and can stretch to nine years in the US. - Biotech leaders argue that meaningful innovation requires embedding patient voices into trial design, endpoints, and real-world evidence rather than relying on clinical data alone. - The FALCON study in primary mitochondrial disease co-created endpoints with patients, prioritizing fatigue and functional strength as the outcomes that matter most. - Rare diseases impose a heavy burden, with an estimated annual cost of nearly $1 trillion in the US and elevated rates of depression (39%) and anxiety (44%) among patients.
- Viralgen, a leading AAV gene therapy CDMO, has partnered with Elaaj Bio to advance ELJ-101, an investigational gene therapy targeting CDKL5 Deficiency Disorder, a rare pediatric neurological condition. - The collaboration leverages Viralgen's AAV manufacturing expertise to support process development, scale-up manufacturing, and analytical characterization for the preclinical-stage program. - CDKL5 Deficiency Disorder is a severe developmental and epileptic encephalopathy characterized by early-onset seizures and significant neurodevelopmental impairment caused by CDKL5 gene alterations. - The teams plan to initiate the first clinical trial for ELJ-101 in early 2027, with current efforts focused on scaling manufacturing processes to support clinical development.
- AskBio has introduced its commercial manufacturing process to supply ametefgene parvec (AB-1005) for the REGENERATE-PD Phase II trial following FDA IND amendment approval. - The investigational gene therapy delivers GDNF protein to the brain via adeno-associated viral vector to potentially slow Parkinson's disease progression and improve motor outcomes. - REGENERATE-PD is enrolling 127 participants aged 45-75 with moderate-stage Parkinson's disease across sites in Germany, Poland, the United Kingdom, and the United States. - The therapy has received multiple regulatory designations including FDA Fast Track, RMAT designation, and Japan's SAKIGAKE status for Parkinson's disease treatment.
- AskBio has completed enrollment of 173 participants in its GenePHIT Phase 2 clinical trial evaluating AB-1002 gene therapy for heart failure with reduced ejection fraction. - The investigational therapy targets protein phosphatase 1 through a single intracoronary infusion and aims to improve symptoms and survival in patients with non-ischemic cardiomyopathy. - Initial results from the randomized, placebo-controlled trial are expected in the first half of 2027, representing a significant milestone for addressing the 64 million people worldwide living with heart failure. - Previous Phase 1 data published in Nature Medicine demonstrated preliminary safety and efficacy, supporting advancement to this larger Phase 2 study.
- Bayer achieved five pivotal worldwide drug approvals in 2025, marking a landmark year of strategic execution for its pharmaceutical division. - The company's cardiovascular portfolio expanded with asundexian meeting primary endpoints in the Phase III OCEANIC-STROKE study for secondary stroke prevention. - Oncology growth accelerated with FDA approval of Hyrnuo (sevabertinib) for HER2-mutant non-small cell lung cancer and continued success of Nubeqa (darolutamide). - Women's health advanced with regulatory approvals of Lynkuet (elinzanetant) as the only hormone-free therapy approved in the EU for moderate to severe vasomotor symptoms.
- Bayer has entered a global licensing agreement with Soufflé Therapeutics to develop heart-targeted siRNA therapy for rare dilated cardiomyopathy, leveraging proprietary cell-selective delivery technology. - The company formed a three-year collaboration with Cradle to integrate generative AI technology into its R&D workflows for accelerating therapeutic antibody development and optimization. - Bayer's AskBio subsidiary received FDA IND clearance for AB-1009, an AAV gene therapy for late-onset Pompe disease, with phase I/II trials expected to begin patient recruitment in early 2026. - Recent regulatory approvals include elinzanetant (Lynkuet) for menopause symptoms and accelerated approval for sevabertinib (Hyrnuo) in HER2-mutated non-small cell lung cancer.
- AskBio announced FDA acceptance of its IND application for AB-1009, an AAV gene therapy for late-onset Pompe disease, advancing the program to Phase 1/2 clinical testing. - The therapy has received FDA Fast Track and Orphan Drug designations, highlighting the recognized unmet medical need for improved treatments in this rare genetic disorder. - AB-1009 aims to address the underlying genetic defect by increasing production of the deficient enzyme, potentially reducing patients' reliance on chronic enzyme replacement therapies. - The PROGRESS-GT LOPD trial will evaluate safety and tolerability of a single intravenous infusion in adults with late-onset Pompe disease, with first patient recruitment anticipated in early 2026.
