Biologics

Complement Therapeutics Gene Therapy Cleared for Trial

Complement Therapeutics has received the green light from FDA to begin human testing of its lead gene therapy, CTx001, in patients with geographic atrophy secondary to age-related macular degeneration. The IND approval paves the way for the company to launch Opti-GAIN, a Phase 1-3 trial evaluating the safety, tolerability, and early efficacy of the one-time adeno-associated virus (AAV)-based therapy. The first patient dosing is expected in the U.S. in the first quarter of 2026, according to the company.

Complement says CTx001 is designed to deliver a truncated form of complement receptor 1 via an AAV vector, enabling long-term modulation of multiple complement pathways implicated in retinal degeneration. The company aims for the therapy to provide durable benefit with a single administration — a potential advance in a field where patients currently rely on frequent intravitreal injections to slow disease progression.

Geographic atrophy, an advanced form of dry AMD, affects around 1.5 million in the U.S. The disease leads to progressive and irreversible vision loss, and despite recent approvals of complement inhibitors, significant unmet need remains, the company says.

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome