LEO Pharma to Acquire Replay for Rare Skin Disease Gene Therapy Platform

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…

ByRidhi Rastogi2 days ago

NEWS

Novartis’ Itvisma Receives the CHMP Positive Opinion for Spinal Muscular Atrophy

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…

ByRidhi RastogiApril 27, 2026

NEWS

Regeneron Reports the US FDA Accelerated Approval of Otarmeni (lunsotogene parvec-cwha) in Genetic Hearing Loss

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…

ByRidhi RastogiApril 24, 2026

NEWS

Eli Lilly to Acquire Kelonia Therapeutics for ~$7B

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…

ByRidhi RastogiApril 21, 2026

NEWS

Ultragenyx Reports the P-III (Enh3ance) Trial for DTX301 AAV8 Gene Therapy in OTC Deficiency

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…

ByRidhi RastogiMarch 13, 2026

NEWS

Ocugen Reports Positive Preliminary P-II (ArMaDa) Data of OCU410 in Geographic Atrophy (GA) Secondary to Dry AMD

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…

ByRidhi RastogiJanuary 16, 2026

NEWS

Genethon Signs Worldwide Licensing Agreement with AskBio (Bayer AG) to Advance Gene Therapy for Pompe Disease

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…

ByPrince GiriJanuary 14, 2026

NEWS

Fondazione Telethon’s Waskyra Receives the EC Approval for Wiskott-Aldrich Syndrome

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…

ByDipanshu DixitJanuary 9, 2026

INSIGHTS+

J.P. Morgan Healthcare Conference Special: Dealmaker of the Year 2025 (Part 01)

Shots: Lilly leads with precision and purpose with 45 deals totaling $21.22B in 2025, focusing on platform-driven, science-led partnerships across oncology, gene therapy, RNA editing, and AI-enabled discovery while building durable innovation engines rather than chasing short-term wins Strategic dealmaking over sheer volume is evident in partnerships like precision oncology with Scorpion Therapeutics and CNS delivery with Sangamo, structured to…

ByPrince GiriJanuary 8, 2026

NEWS

Novartis’ Itvisma Receives the US FDA Approval for Spinal Muscular Atrophy

Shots: The US FDA has approved Itvisma (onasemnogene abeparvovec-brve) for the treatment of pts (≥2yrs.) living with spinal muscular atrophy (SMA) harboring a confirmed mutation in the SMN1 gene; US availability expected in Dec 2025 Approval was based on the P-III (STEER) trial & open-label P-IIIb (STRENGTH) study, showing Itvisma improved motor function & stabilized…

ByRidhi RastogiNovember 25, 2025

LEO Pharma to Acquire Replay for Rare Skin Disease Gene Therapy Platform

Novartis’ Itvisma Receives the CHMP Positive Opinion for Spinal Muscular Atrophy

Regeneron Reports the US FDA Accelerated Approval of Otarmeni (lunsotogene parvec-cwha) in Genetic Hearing Loss

Eli Lilly to Acquire Kelonia Therapeutics for ~$7B

Ultragenyx Reports the P-III (Enh3ance) Trial for DTX301 AAV8 Gene Therapy in OTC Deficiency

Ocugen Reports Positive Preliminary P-II (ArMaDa) Data of OCU410 in Geographic Atrophy (GA) Secondary to Dry AMD

Genethon Signs Worldwide Licensing Agreement with AskBio (Bayer AG) to Advance Gene Therapy for Pompe Disease

Fondazione Telethon’s Waskyra Receives the EC Approval for Wiskott-Aldrich Syndrome

J.P. Morgan Healthcare Conference Special: Dealmaker of the Year 2025 (Part 01)

Novartis’ Itvisma Receives the US FDA Approval for Spinal Muscular Atrophy

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