Nanoscope Therapeutics Prepares BLA Submission for MCO-010 in Retinitis Pigmentosa Following FDA Meeting


 

Nanoscope Therapeutics Inc., a late-stage clinical biotechnology company dedicated to developing gene therapies for retinal degenerative diseases, has announced a successful FDA meeting for its clinical program evaluating MCO-010 for the treatment of retinitis pigmentosa (RP). Based on the positive regulatory feedback received during the meeting, Nanoscope will begin the submission of a Biologics License Application (BLA) in the first quarter of 2025. This marks a significant step forward in the company’s mission to bring effective treatments to patients suffering from RP.

Publish Date: 15-10-2024   Source: Nanoscope Therapeutics Inc.

Retinitis pigmentosa (RP) is a hereditary degenerative condition of the retina characterized by night blindness and a gradual loss of peripheral vision, eventually leading to complete blindness. The primary malfunction typically occurs in the rod photoreceptors, causing them to deteriorate progressively. Subsequently, cone cells also perish, likely due to oxidative damage and a lack of nourishment. The age at which symptoms first appear varies widely, not only between families but also among forms of RP. However, once symptoms manifest, the rate and manner of progression tend to follow a fairly predictable and stereotypical exponential pattern. Individuals who take vitamin A, E, or a combination of both have shown slower declines in ERG (electroretinogram) amplitudes compared to those who do not accept these supplements. The treatment of retinitis pigmentosa (RP), the FDA has approved Luxturna (voretigene neparvovec). Luxturna is a gene therapy designed to address a specific subgroup of RP patients with the RPE65 mutation, which accounts for approximately 0.3% -1 % of all RP cases.

  • RP is the most prevalent inherited retinal degeneration (IRD), impacting over 1.5 million individuals worldwide.

However, the current Retinitis Pigmentosa (RP) treatment market share, market uptake, and attribute analysis concerning the most potential emerging therapies (OCU400, Botaretigene sparoparvovec, EA-2353, BS01, etc..) has been provided under the market outlook section of the study covering 8 MM countries; The United States, EU5 (Germany, Spain, France, Italy, UK) Japan and China.

In terms of pharmacologic therapies, several pharmaceutical products are being approved and under different phases of development for the Retinitis Pigmentosa treatment. The key companies in the advanced development stage are Ocugen, Inc., MeiraGTx UK II Ltd, Endogena Therapeutics, Inc, Bionic Sight LLC, etc..

Based on solid domain and business knowledge, Thelansis Knowledge Partners has published the market outlook forecast report on Retinitis Pigmentosa (RP) to provide a clear understanding of disease area background, epidemiology, current and future competitions, the country-specific standard of care, and the complete market forecast for 2023 to 2033.

About Thelansis:

Thelansis specializes in pharmaceutical market outlook and market forecast reports. We published reports across the therapeutic area, including rare / ultra-rare and mainstream indications. Over the period, we have built a robust repository of 6,000+ Bio-pharma reports that cover Epidemiology studies and Market forecasting based on the KOL opinions.

Competitive intelligence and track of trial results throughout the phases of development executed by a team of a mix of Scientific and Business backgrounds. As an organization, the primary focus is to provide real-world data evidence and market insight to pharmaceutical companies for their decision-making.

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