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

NEWS

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…

ByRidhi RastogiNovember 13, 2025

NEWS

Eli Lilly Strengthens its Gene Therapy Capabilities with Adverum Acquisition

Shots: Eli Lilly has entered into a definitive agreement to acquire Adverum Biotechnologies, incl. its asset Ixo-vec As per the deal, Lilly will acquire Adverum for $3.56/share plus a CVR of ~$8.91 per share, tied to milestones: for ~$1.78 per CVR on Ixo-vec’s FDA approval within 7yrs. of closing & ~$7.13 per CVR upon achieving $1B…

ByRidhi RastogiOctober 27, 2025

NEWS

AviadoBio Enters Exclusive Option & License Agreement with UgeneX Therapeutics to Advance UGX-202

Shots: AviadoBio has entered into an exclusive option & license agreement with UgeneX Therapeutics to develop & commercialize UGX-202 As per the deal, AviadoBio will receive an option to develop & commercialize UGX-202 for retinitis pigmentosa & other indications globally (excl. Greater China), while UgeneX will receive upfront payments, r&d milestones, & sales milestone payments…

ByRidhi RastogiOctober 9, 2025

NEWS

Remedium Bio Joins Forces with Eli Lilly to Advance Gene Therapies for Obesity and Type 2 Diabetes

Shots: Remedium Bio has entered into a multi-target research & development collaboration with Eli Lilly to develop gene therapies targeting multiple targets for the treatment of Obesity & Type 2 Diabetes, leveraging Remedium’s Prometheus platform As per the deal, Remedium will receive an upfront, equity investment as well as development & commercial milestone payments, with tiered…

ByRidhi RastogiSeptember 9, 2025

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

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

AviadoBio Enters Exclusive Option & License Agreement with UgeneX Therapeutics to Advance UGX-202

Remedium Bio Joins Forces with Eli Lilly to Advance Gene Therapies for Obesity and Type 2 Diabetes

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