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Neurogene Reports Interim P-I/II Trial Results on NGN-401 to Treat Pediatric Patients with Rett Syndrome

Shots: Neurogene has reported interim data from the pediatric cohort (4-10yrs.) of the ongoing P-I/II trial assessing NGN-401 for the treatment of females with Rett syndrome As of Oct 30, 2025, all pediatric pts (n=8) had functional gains across Rett syndrome domains, achieving 35 developmental milestones in fine motor, communication & ambulation. These gains were…

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Doc.com Launches AI & Blockchain Telemedicine Platform in the US

Shots: Doc.com launches its AI & blockchain-powered telemedicine platform in the US to improve patient access, data security, & care coordination Pts can securely connect with licensed in-state healthcare providers via mobile using the platform, with up to 15min. of complimentary teleconsultation offered during launch, available in eligible jurisdictions & subject to applicable regulations, followed…

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Lyell Immunopharma Acquires Global Rights to LYL273 from Innovative Cellular Therapeutics in ~$860M Deal

Shots: Lyell has acquired global rights to LYL273 (excl. mainland China, Hong Kong, Macau & Taiwan) for the treatment of metastatic colorectal cancer & other GCC-expressing cancers from ICT As per the deal, ICT will receive $40M upfront, 1.9M Lyell shares, ~$30M in clinical, ~$115M in late-stage regulatory, & ~$675M in sales milestones, plus ~1.85M additional…

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Neurona Therapeutics’ NRTX-1001 Secures the EMA’s Priority Medicines (PRIME) Designation for Drug-Resistant Focal Epilepsy

Shots: The EMA has granted PRIME designation to NRTX-1001 for adults with drug-resistant focal epilepsy, based on preclinical & emerging clinical data NRTX-1001 is also being evaluated in 2 ongoing P-I/II trials for drug-resistant unilateral & bilateral mesial temporal lobe epilepsy (MTLE), with P-III (EPIC) trial planned to begin in H2’25 for drug-resistant MTLE The…

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GSK Collaborates with Empirico to Advance EMP-012 Across Inflammatory Respiratory Diseases

Shots: GSK has entered into a worldwide exclusive license agreement with Empirico to develop & commercialize EMP-012 As per the deal,Empirico will lead P-I development of EMP-012, following which GSK will take over global development, regulatory filings, & commercialization, with Empirico receiving $85M upfront, ~$660M in development, regulatory & commercial milestones, & tiered royalties on…

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BeOne Medicines’ Sonrotoclax Receives FDA’s Breakthrough Therapy Designation for R/R Mantle Cell Lymphoma (MCL)

Shots: The US FDA has granted BTD to sonrotoclax for adults with r/r MCL & accepted BeOne’s request for participation in Project Orbis for concurrent global review of the therapy Decision was based on P-I/II (BGB-11417-201) assessing sonrotoclax in r/r MCL pts previously treated with BTKi & anti-CD20 therapy; Part 1 enrolled 22 pts on…

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