AstraZeneca to Acquire EsoBiotec for Cell Therapy Advancement
AstraZeneca (LSE/STO/Nasdaq: AZN) has announced the acquisition of EsoBiotec SA, a Belgian biotechnology company pioneering in vivo cell therapies. EsoBiotec's Engineered NanoBody Lentiviral (ENaBL) platform, which has demonstrated promising early clinical activity,...
Dyne’s DYNE-251: 18-Month Data Shows Unprecedented DMD Improvement
WALTHAM, Mass., March 16, 2025 -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company developing life-transforming therapies for genetically driven neuromuscular diseases, today announced positive long-term clinical data from its ongoing Phase 1/2 DELIVER...
Gene Therapy Investment Declines Amidst Successes and Economic Headwinds
Gene therapy, despite its potential to cure rare diseases like sickle cell disease, is experiencing a decline in early investor interest, as higher-reward sectors such as obesity and cancer attract more capital. This shift comes as sales for some of the novel gene...
Sofinnova’s Biovelocita II Fund Closes at $181M, Backing Three New Biotech Companies
Sofinnova Partners has announced the final close of its Biovelocita II accelerator fund, raising €165 million ($181 million), exceeding its target. The fund, backed by pharmaceutical giants including Amgen, Bristol Myers Squibb, and Pfizer Ventures, will support the...
RegCell Raises $45.8M for US Expansion of Treg Therapies
RegCell, a biotechnology company developing epigenetic reprogramming-based regulatory T-cell (Treg) therapies for autoimmune diseases and transplantation, has closed an oversubscribed $8.5 million seed round and is transitioning to a U.S.-based corporation. The seed...
Prime Medicine Announces AATD Gene Editing Program
Prime Medicine, Inc. (Nasdaq: PRME) has announced a preclinical program for alpha-1 antitrypsin deficiency (AATD), expanding its liver-focused gene editing pipeline. The company anticipates filing an investigational new drug (IND) or clinical trial application (CTA)...
Novartis’ Intrathecal Zolgensma Shows Efficacy in Older SMA Patients
Spinal muscular atrophy (SMA), a devastating neuromuscular disease characterized by progressive muscle weakness and atrophy, has seen remarkable advancements in treatment over the past decade. Among these breakthroughs, Zolgensma (onasemnogene abeparvovec) stands out...
Wugen Starts Pivotal Trial of Off-the-Shelf Allogeneic CD7 CAR-T (WU-CART-007)
ST. LOUIS, March 20, 2025 – Wugen, Inc., a clinical-stage biotechnology company focused on allogeneic, off-the-shelf cell therapies, has announced the initiation of its pivotal Phase 2 study evaluating WU-CART-007, a potential first-in-class, investigational CAR-T...
Beacon Therapeutics published 24-month safety and efficacy data from the AGTC-501 HORIZON Trial in the American Journal of Ophthalmology
Beacon Therapeutics announces the publication of 24-month data from the Phase 1/2 HORIZON trial of laru-zova (AGTC-501), a treatment for X-linked retinitis pigmentosa (XLRP) caused by RPGR gene mutations, in the peer-reviewed American Journal of Ophthalmology. The...
Arbor Raises $73.9M Series C to Support Clinical Trials of ABO-101, a Novel Treatment for Primary Hyperoxaluria Type 1
Arbor Biotechnologies, a pioneering gene editing company established nine years ago by CRISPR luminary Feng Zhang, has successfully closed a $73.9 million Series C financing round, spearheaded by ARCH Venture Partners and TCGX. This infusion of capital represents a...
New mRNA Vaccines Target a Host of Viral and Bacterial Pathogens
By Caroline Seydel, GEN News, March 3, 2025 The extraordinary success of mRNA vaccines in the rapid response to the COVID-19 pandemic has precipitated a monumental shift in the landscape of vaccine development. Companies worldwide are now strategically leveraging the...
Beam Therapeutics Announces Groundbreaking Positive Initial Data from BEAM-302 Phase 1/2 Trial for Alpha-1 Antitrypsin Deficiency
CAMBRIDGE, Mass., March 10, 2025 (GLOBE NEWSWIRE) – Beam Therapeutics Inc. (Nasdaq: BEAM), a pioneering biotechnology company at the forefront of precision genetic medicines through base editing, today announced transformative positive initial safety and efficacy data...
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