Exegenesis Bio has received FDA approval to advance its groundbreaking gene therapy, EXG102-031, aimed at treating neovascular age-related macular degeneration (nAMD). This represents a significant milestone in the company’s journey, marking its second investigational new drug (IND) approval and the first for a gene therapy targeting nAMD in North America.
Clinical Trial Launch
The inaugural phase 1 clinical trial for EXG102-031 is set to commence in the first quarter of 2023. The trial’s objectives include assessing safety, tolerability, and initial efficacy, with visual acuity and central retinal thickness serving as key metrics. This gene therapy is notable for its ability to target all known subtypes of vascular endothelial growth factor (VEGF) and angiopoietin-2 (ANG2), both critical factors in the progression of nAMD. Administration involves an intraocular injection of a recombinant adeno-associated virus vector that expresses a therapeutic fusion protein designed to neutralize these targets.
Acknowledging Milestones
Zhenhua Wu, CEO of Exegenesis Bio, expressed enthusiasm over the IND approval, emphasizing the company’s commitment to research and development. He highlighted the achievement as a validation of the robust capabilities in research, development, and regulatory compliance that Exegenesis has cultivated since its inception three years ago. Wu looks forward to accelerating the development of innovative therapies that address substantial unmet medical needs globally.
Previous Advances
Exegenesis Bio’s journey in gene therapy began with the IND approval for EXG001-307, a unique AAV gene therapy aimed at expressing the SMN gene. Preclinical studies showcased its efficacy in mouse models of spinal muscular atrophy, outperforming the currently approved gene therapy, onasemnogene abeparvovec (Zolgensma). The results indicated enhanced tissue-specific SMN expression along with reduced off-target toxicity.
Promising Results
In the preclinical data presented at the 2022 American Society of Gene & Cell Therapy (ASGCT) meeting, EXG001-307 demonstrated significant benefits, including extended survival and improved motor functions in animal models compared to existing therapies. Wu, who led the presentation, highlighted the therapy’s potential to revolutionize treatment paradigms for spinal muscular atrophy.
Expanding the Pipeline
EXG102-031 and EXG001-307 stand as the flagship candidates in Exegenesis’ pipeline, yet the company is actively pursuing additional programs. These include gene therapies for hemophilia A and phenylketonuria, alongside an undisclosed liver target currently in IND-enabling studies. Moreover, research is underway for conditions such as Parkinson’s disease, Bietti’s crystalline dystrophy, and other central nervous system and ocular diseases.
Future Outlook
The recent IND approval positions Exegenesis Bio at the forefront of gene therapy innovation, particularly in addressing retinal diseases. As the company embarks on clinical trials for EXG102-031, anticipation builds regarding the potential impact on patient care and outcomes in the realm of ophthalmology.
Key Takeaways
- Exegenesis Bio has received FDA clearance for EXG102-031, targeting nAMD.
- The phase 1 trial will evaluate safety and efficacy, launching in early 2023.
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EXG001-307 previously showcased promising results in spinal muscular atrophy models.
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The company’s pipeline includes therapies for hemophilia A, phenylketonuria, and neurological conditions.
In conclusion, Exegenesis Bio’s strategic advancements in gene therapy not only highlight its commitment to addressing significant health challenges but also signify a transformative potential for patients suffering from debilitating conditions. As clinical trials progress, the biotech community eagerly awaits the outcomes that could reshape treatment standards in ophthalmology and beyond.
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