Ultragenyx Receives FDA Accelerated Approval for GENGLYCOS Gene Therapy to Treat the Underlying Cause of GSDIa

Shots: Ultragenyx reported the US FDA accelerated approval to GENGLYCOS (pariglasgene brecaparvovec-opnr; DTX401) for adults and pediatric patients (≥8yrs., n=46) with glycogen storage disease type Ia (GSDIa) The approval was supported by the P-III (GlucoGene) study, in which GENGLYCOS (n=20) demonstrated a statistically significant reduction in cornstarch requirements at 48wks. vs PBO (n=24) (p<0.001). The…

ByPrince Giri1 day ago

INSIGHTS+

EMA Marketing Authorization of New Drugs in July 2026

Shots: The European Commission (EC) approved multiple new medicines in July 2026 across rare diseases, respiratory disorders, aesthetics, and oncology, involving companies including Novartis, Boehringer Ingelheim, AbbVie’s Allergan Aesthetics, and AstraZeneca. EC approvals included Itvisma (onasemnogene abeparvovec) for patients aged ≥2 years with 5q spinal muscular atrophy (SMA) and confirmed bi-allelic SMN1 mutations; Jascayd (nerandomilast) for adults with idiopathic pulmonary…

ByDipanshu Dixit3 days ago

NEWS

PTC Therapeutics to Acquire Sangamo Therapeutics’ ST-920 Gene Therapy Program

Shots: PTC Therapeutics has been selected as the winning bidder to acquire ST-920, a BLA-stage, one-time AAV gene therapy for Fabry disease, from Sangamo Therapeutics through a competitive bankruptcy auction As per the terms, PTC Therapeutics will pay $111M upfront, with up to $100M in contingent milestone payments tied to certain regulatory approvals; expected to…

ByRidhi RastogiAugust 13, 2026

EVENTS

BIOPLUS-INTERPHEX KOREA 2026 | October 28-30 | COEX, Seoul

BIOPLUS-INTERPHEX KOREA (BIX) 2026 is Korea's premier biopharmaceutical business event, bringing together global leaders across the entire biopharmaceutical value chain. Scheduled to take place from October 28-30, 2026, at COEX, Seoul, South Korea, the event serves as a platform for innovation, knowledge exchange, technology showcase, and strategic business partnerships. From drug discovery and clinical development…

BySaurabh ChaubeyAugust 5, 2026

NEWS

4DMT Reports P-IIb (PRISM) Trial Data on 4D-150 in a Broad Wet AMD Population

Shots: 4DMT reported 2yr. data from the P-IIb (PRISM) trial evaluating intravitreal 4D-150 at 3E10 vg/eye (n=30) & 1E10 vg/eye (n=15) in 45 pts with wet age-related macular degeneration P-IIb subgroup incl. 15 recently diagnosed pts treated with 3E10 vg/eye, which was also selected for the P-III (4FRONT) trials, with the 2-year Phase 2b data…

ByRidhi RastogiJuly 20, 2026

NEWS

Novartis’ Itvisma Receives the EC Approval for Spinal Muscular Atrophy

Shots: The EC has approved 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 Approval was based on the P-III (STEER) trial & supportive P-IIIb (STRENGTH) & P-I/II (STRONG) studies, where STEER showed a 2.39-point improvement in the…

ByRidhi RastogiJuly 2, 2026

NEWS

Merck KGaA to Acquire Bio-Techne for ~$11.3B

Shots: Merck KGaA has entered into a definitive agreement to acquire Bio-Techne, bringing together two companies with complementary portfolios spanning research tools, diagnostics, bioprocessing, and advanced therapeutics As per the deal, Merck KGaA will acquire Bio-Techne for $73/share in cash, representing a total enterprise value of ~$11.3B; closing is expected by late 2026 or early…

ByRidhi RastogiJune 25, 2026

VIEWPOINTS

Judith Greciet on Targeting Iron Dysregulation: PulseSight’s Next-Generation Approach to Geographic Atrophy

Shots: PulseSight Therapeutics is advancing a novel approach to Geographic Atrophy (GA) with PST-611, a first-in-class non-viral gene therapy designed to restore iron homeostasis by targeting transferrin biology—an upstream driver of retinal degeneration. At ARVO 2026, PST-611 delivered encouraging Phase I results, demonstrating an excellent safety and tolerability profile, stable visual acuity, and early signals of potential functional…

BySaurabh ChaubeyJune 22, 2026

EVENTS

London Biotechnology Show 2026 | 18-19 November | Excel London

Get ready to connect with the innovators, investors, and industry leaders shaping the future of biotechnology at the London Biotechnology Show 2026. Taking place alongside London Life Sciences Week, the event is set to become one of the UK’s most influential gatherings for the global biotech and life sciences ecosystem. Bringing together 4,000+ attendees and…

BySaurabh ChaubeyJune 12, 2026

VIEWPOINTS

Dr. Orla Cloak on Manufacturing Excellence: Minaris Vision for Advanced Therapies

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…

BySaurabh ChaubeyMay 18, 2026

Ultragenyx Receives FDA Accelerated Approval for GENGLYCOS Gene Therapy to Treat the Underlying Cause of GSDIa

EMA Marketing Authorization of New Drugs in July 2026

PTC Therapeutics to Acquire Sangamo Therapeutics’ ST-920 Gene Therapy Program

BIOPLUS-INTERPHEX KOREA 2026 | October 28-30 | COEX, Seoul

4DMT Reports P-IIb (PRISM) Trial Data on 4D-150 in a Broad Wet AMD Population

Novartis’ Itvisma Receives the EC Approval for Spinal Muscular Atrophy

Merck KGaA to Acquire Bio-Techne for ~$11.3B

London Biotechnology Show 2026 | 18-19 November | Excel London

Dr. Orla Cloak on Manufacturing Excellence: Minaris Vision for Advanced Therapies

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