Shots:
Minaris is advancing its dual-business strategy through specialized Advanced Therapies and Advanced Testing units, enabling sponsors to streamline development, manufacturing, biosafety testing, and commercialization within one integrated global organization.
Minaris is focused on solving key cell & gene therapy manufacturing challenges by reducing variability, accelerating tech transfer, improving “first time right” execution, and lowering…
Shots:
PharmaShots’ May Edition explores how AI is transforming commercial strategies across the healthcare ecosystem by enabling predictive analytics, personalized engagement, dynamic launch planning, and real-time omnichannel optimization while balancing compliance and measurable ROI.
The magazine highlights key industry movements shaping global biopharma, including AI-driven drug discovery collaborations, strategic M&A activity, advancements in cell and…
Shots:
LEO Pharma has entered into a definitive agreement to acquire Replay, expanding its dermatology pipeline with Replay’s herpes simplex virus-based gene therapy platform for rare genetic skin disorders
As per the deal, LEO will acquire Replay for $50M upfront, plus milestone payments & tiered single-digit royalties, while adding Replay’s HSV design and manufacturing expertise…
Shots:
The CHMP has recommended Novartis' Itvisma (onasemnogene abeparvovec) for the treatment of pts (≥2yrs.) living with 5q spinal muscular atrophy (SMA) harboring a confirmed bi-allelic mutation in the SMN1 gene
Opinion was based on the P-III (STEER) trial & P-IIIb (STRENGTH) study, where STEER showed a 2.39-point improvement in the HFMSE with effects sustained…
Shots:
Otarmeni was approved under CNPV for pediatric & adult pts with sev. to profound and profound sensorineural hearing loss due to biallelic OTOF gene variants, with preserved outer hair cell function & no prior cochlear implant. It will be provided free of charge in the US by Regeneron
Approval was based on the P-I/II…
Shots:
Eli Lilly has entered into a definitive agreement to acquire Kelonia Therapeutics, expanding Lilly’s genetic medicine capabilities
As per the deal, Lilly will acquire Kelonia for ~$7B in cash, incl. $3.25B upfront, & additional clinical, regulatory & commercial milestone payments; closing expected in H2’26
Acquisition will add Kelonia’s in vivo gene placement system (iGPS0) & its lead asset, KLN-1010, a one-time IV…
Shots:
Ultragenyx has reported the P-III (Enh3ance) study data assessing DTX301 AAV8 gene therapy vs PBO for the treatment of ornithine transcarbamylase (OTC) deficiency
DTX301 reduced 24hr. plasma ammonia by 18% vs PBO at Wk. 36, with ammonia levels maintained in the normal range, despite a 27% mean decrease in scavenger drugs & ~13% increase…
Shots:
Ocugen has reported preliminary 12mos. data from its P-II (ArMaDa) trial (n=51) of OCU410 (AAV5-RORA) in GA secondary to dry AMD, with ~50% of pts evaluated to date
Trial showed a 46% reduction in GA lesion growth vs control (medium + high dose), with the medium dose achieving a 54% reduction; 50% pts achieved…
Shots:
Genethon has entered into an exclusive, worldwide licensing agreement with AskBio for the use of a patented component of AskBio’s investigational gene therapy AB-1009 for the treatment of Pompe disease
The licensed component is based on a transgene encoding a truncated form of GAA, developed by Genethon, which demonstrated preclinical efficacy in correcting glycogen…
Shots:
The EC has approved Waskyra (etuvetidigene autotemcel), an ex vivo gene therapy for Wiskott-Aldrich Syndrome (WAS)
Waskyra is a single-administration therapy that uses a patient’s CD34+ hematopoietic stem and progenitor cells, modified with a lentiviral vector carrying the WAS gene
The EC’s decision follows a CHMP opinion issued in November 2025 recommending marketing authorization, with the same therapy also approved by…

