Complement Therapeutics GmbH, a clinical-stage biotechnology company developing next-generation therapeutics for complement-mediated diseases, announced that the US Food and Drug Administration (FDA) has granted Fast Track Designation for CTx001, the company's lead gene therapy candidate for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
The Investigational New Drug (IND) application for CTx001 was previously cleared to proceed by the US Food and Drug Administration, enabling advancement of the programme into clinical development.
Fast Track designation is intended to facilitate the development and expedite the review of drugs that treat serious conditions and have the potential to address unmet medical needs. It is designed to support more frequent interactions with the US Food and Drug Administration and bring important new therapies to patients sooner. The US Food and Drug Administration’s fast track designation has been granted to the lead gene therapy candidate CTx001 also for geographic atrophy.
This lead gene therapy candidate CTx001 is an investigational adeno-associated virus-based gene therapy designed to modulate multiple pathways of the complement system.
CTx001 has previously received US Food and Drug Administration IND clearance and will be evaluated in Opti-GAIN, a first-in-human phase I/II clinical trial.
CTx001 is a novel adeno-associated virus (AAV)-based gene therapy designed to deliver a truncated version of Complement Receptor 1 (mini-CR1), with the potential for long-term potent modulation of the classical and alternative pathways of the complement cascade. Geographic atrophy is an advanced form of dry age-related macular degeneration that leads to irreversible vision loss, with limited therapeutic options.
""The US Food and Drug Administration Fast Track Designation for CTx001 is an important regulatory milestone recognising the seriousness of geographic atrophy, the unmet medical need faced by patients as well as the innovative potential for CTx001 to meaningfully address this need"" said Dr. Rafiq Hasan, chief executive officer of Complement Therapeutics. ""Together with the recent Investigational New Drug clearance, this designation supports our efforts to efficiently advance CTx001 into clinical development as we work to address a leading cause of irreversible vision loss.""
CTx001 will be evaluated in the Opti-GAIN (Optimised Geographic Atrophy Interventional) first-in-human phase I/II clinical trial in patients with Geographic Atrophy secondary to Age-related Macular Degeneration, with first patient dosing expected in the United States.
The Opti-GAIN (Optimised Geographic Atrophy INterventional) clinical trial is an international first-in-human, open-label, Phase I/II study designed to evaluate the safety, tolerability, and preliminary efficacy of CTx001. The trial will enrol patients across leading retinal centres with first patient dosing expected in the United States in Q1 2026.
The clinical trial design is informed by data from i-GAIN, a natural history study involving over 230 participants, which has provided valuable insights into disease progression, imaging biomarkers, and patient stratification in geographic atrophy.
Geographic atrophy is an advanced form of dry age-related macular degeneration where patches of the retina, particularly in the macula (centre of vision), waste away, causing irreversible loss of central vision, often appearing as blurry or gray spots, affecting reading, driving, and face recognition. It's characterized by the death of light-sensing cells (photoreceptors) and supporting cells, creating distinct, map-like areas of atrophy, and typically affects both eyes.
It occurs in people with another eye condition called age-related macular degeneration. It is most common in people over age 60. Over time, geographic atrophy can lead to loss of central vision, which helps you see what is in front of you. This happens when the light-sensing tissue at the back of the eye, called the retina, is damaged. The damaged retina can get larger over time, causing increasing vision loss. Geographic atrophy can affect one or both eyes. There is no cure, but the FDA has approved some new therapies to slow down its progression. Other new treatments are being studied in clinical trials.
People with geographic atrophy may notice changes in vision that start slowly and get worse over time. Geographic atrophy often starts just outside the centre of the retina and slowly moves inward. As it grows, you can lose central vision or have blurred or blind spots.
Many people keep decent central vision early on, but as geographic atrophy spreads, this vision can be lost. Vision loss can become severe over time. It can make it hard to drive, read, watch TV and do household chores. People with geographic atrophy usually keep their side vision, known as peripheral vision.