{"id":93195,"date":"2025-08-19T01:29:11","date_gmt":"2025-08-19T01:29:11","guid":{"rendered":"https:\/\/www.newsbeep.com\/us\/93195\/"},"modified":"2025-08-19T01:29:11","modified_gmt":"2025-08-19T01:29:11","slug":"fda-clears-opus-genetics-ind-for-best1-ird-gene-therapy","status":"publish","type":"post","link":"https:\/\/www.newsbeep.com\/us\/93195\/","title":{"rendered":"FDA clears Opus Genetics’ IND for BEST1 IRD gene therapy"},"content":{"rendered":"

Opus Genetics<\/a> has received FDA clearance for its investigational new drug<\/a> (IND) application of the gene therapy OPGx-BEST1, indicated to treat bestrophine-1 (BEST1)-related inherited retinal disease (IRD).<\/p>\n

Let\u2019s start with a look at BEST1.<\/p>\n

The BEST1 gene is responsible for providing instruction<\/a> on making the bestrophin protein, which acts as a channel to manage charged (chlorine ion) atoms\u2019 movement in and out of cells within the retina.<\/p>\n

Variants (mutations) in the BEST1 gene (as well as the PRHP2 gene) can result in the development of abnormally-shaped channels unable to control chloride flow.Also to keep in mind: BEST1 plays a key role in the retinal pigment epithelium (RPE), which is critical for vision.<\/p>\n

As a result of these BEST1 mutations: BEST1-related IRDs (bestrophinopathies) can develop, impacting an estimated 9,000 patients<\/a> across the United States.<\/p>\n

A notable IRD included in this: Vitelliform macular dystrophy (VMD), a rare genetic eye disorder<\/a> affecting the macula that can lead to worsening vision loss.<\/p>\n

Breaking this disease down, VMD can manifest into one of two forms<\/a>: early-onset and adult-onset.The early-onset form is known as Best disease, which typically develops in childhood (though it can also appear later in life).And how does OPGx-BEST1 target this macular degeneration?<\/p>\n

The BEST1 gene therapy delivers a functional copy of the BEST1 gene to RPE cells to create a BEST1 protein and stabilize homeostasis between RPE cells and photoreceptors.<\/p>\n

How it accomplishes this: By leveraging an adeno-associated virus (AAV) vector via Opus Genetics\u2019 proprietary AAV-based gene therapy platform.<\/p>\n

The intent: To return RPE cells to their normal function and enable them to properly support photoreceptors\u2014restoring vision.<\/p>\n

Duly noted. Now, what does this IND mean?<\/p>\n

A step forward in OPGx-BEST1\u2019s clinical evaluation, to say the least.<\/p>\n

In general: An IND is submitted to the FDA<\/a> by a company or investigator in order to move from the preclinical stage of a proposed investigational drug on to human-based clinical trials.<\/p>\n

Included in this submission:Pharmacology and toxicology data from preclinical researchProposed clinical trial protocolsManufacturing and quality control info<\/p>\n

Notably: The FDA will generally review a submission to ensure the safety and rights of research subjects as well as the quality of the scientific evaluation pertaining to the proposed drug.<\/p>\n

And for OPGx-BEST1\u2019s case?<\/p>\n

With its IND clearance in place, Opus Genetics intends to initiate<\/a> a phase 1\/2 clinical trial.<\/p>\n

Nice! Before we talk more about that, what did its preclinical performance look like?<\/p>\n

Promising \u2026 and this is based on 2024 research<\/a> presented at the Association for Research and Vision in Ophthalmology (ARVO) annual meeting.<\/p>\n

The IND-enabling study: Evaluated a unilateral subretinal injection of OPGx-BEST1 (in three varying doses) administered (versus vehicle) in a group of canines diagnosed with bestrophinopathy (canine multifocal retinopathy).<\/p>\n

The results: OPGx-BEST1 was well tolerated and resulted in no significant ophthalmic and systemic toxicity at two of its doses (low- and mid-doses)\u2014with subtle clinical (funduscopic) signs of potential retinal toxicity noted among patients treated with a high-dose version.<\/p>\n

Interesting \u2026 so what do we know about this upcoming study?The design: A multicenter, open-label trialThe purpose: To evaluate the safety, tolerability, and preliminary efficacy<\/a> of a single subretinal injection of OPGx-BEST1 in a to-be-determined number of patientsThe participants: Patients diagnosed with genetically-confirmed BEST1-related IRDThe outcome measures: Exploring the biological activity via functional and anatomical endpoints\u2014including changes in visual function and retinal structureAnd the timeframe for its initiation?<\/p>\n

Per<\/a> the company: Before the end of 2025 (second half [H2]).<\/p>\n

As for the big-picture significance: With no treatment currently approved for BEST1-related IRDs, OPG-BEST1 represents a potential groundbreaking opportunity for this patient base.<\/p>\n","protected":false},"excerpt":{"rendered":"Opus Genetics has received FDA clearance for its investigational new drug (IND) application of the gene therapy OPGx-BEST1,…\n","protected":false},"author":2,"featured_media":93196,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[50],"tags":[200,79],"class_list":["post-93195","post","type-post","status-publish","format-standard","has-post-thumbnail","category-genetics","tag-genetics","tag-science"],"_links":{"self":[{"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/posts\/93195","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/comments?post=93195"}],"version-history":[{"count":0,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/posts\/93195\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/media\/93196"}],"wp:attachment":[{"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/media?parent=93195"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/categories?post=93195"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/tags?post=93195"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}