Catalent and Nanoscope Therapeutics Expand Partnership to Advance Commercial Readiness of Vision-Restoring Gene Therapy MCO-010
Catalent, Inc., a global leader in development, manufacturing, packaging, and supply solutions for the pharmaceutical and biotechnology industries, has expanded its strategic partnership with Nanoscope Therapeutics to support the late-stage clinical development and future commercial launch of MCO-010, the biotechnology company’s lead optogenetic gene therapy designed to restore vision in patients with advanced retinal degenerative diseases.
The expanded collaboration represents an important milestone as Nanoscope advances MCO-010 toward potential regulatory approval in the United States. Under the new agreement, Catalent will provide commercial-compliant packaging, labeling, and distribution services while also managing the commercial packaging validation program required to support Nanoscope’s Biologics License Application (BLA).
The companies announced that a rolling submission of the BLA to the U.S. Food and Drug Administration (FDA) is already underway, marking another significant step toward making the investigational therapy available to patients affected by irreversible vision loss.
The strengthened partnership reflects both companies’ commitment to ensuring that manufacturing and commercial supply capabilities are fully prepared as MCO-010 progresses through the final stages of regulatory review.
Strengthening a Long-Term Collaboration
Catalent and Nanoscope Therapeutics have worked together throughout the development of MCO-010, supporting various manufacturing and clinical supply activities.
The newly expanded agreement builds upon that relationship by extending Catalent’s responsibilities beyond clinical development into commercial preparation.
In addition to providing packaging and distribution services, Catalent will oversee the validation of commercial packaging processes, an important regulatory requirement for biologic therapies seeking marketing approval.
Commercial packaging validation helps demonstrate that products can be consistently packaged, labeled, and distributed according to regulatory quality standards while maintaining product integrity throughout the supply chain.
These activities are particularly important for advanced biologic medicines such as gene therapies, where maintaining strict quality controls is essential to ensuring patient safety and therapeutic effectiveness.
Preparing for Potential Commercial Launch
As Nanoscope Therapeutics approaches potential commercialization, establishing a reliable commercial supply infrastructure has become an increasingly important priority.
Transitioning from clinical development to commercial manufacturing involves significantly larger production volumes, expanded quality systems, regulatory inspections, global logistics planning, and coordinated distribution capabilities.
Catalent’s extensive experience supporting commercial biologics launches positions the company to help Nanoscope successfully navigate this transition.
Through the expanded partnership, Catalent will provide commercial-ready packaging operations capable of supporting future product distribution while ensuring compliance with applicable regulatory standards.
This infrastructure is intended to support product availability immediately following potential regulatory approval.
Supporting the Biologics License Application
A key component of the expanded collaboration involves Catalent’s support for Nanoscope’s ongoing Biologics License Application.
Unlike traditional drug approval submissions, biologic products often require extensive documentation covering manufacturing processes, packaging systems, quality controls, product stability, and distribution procedures.
Commercial packaging validation serves as an essential element within this regulatory framework.
By completing these validation activities before approval, Nanoscope aims to minimize potential delays between regulatory authorization and commercial product launch.
The companies confirmed that the FDA rolling submission process is currently in progress.
Rolling submissions allow sponsors of eligible therapies to submit completed portions of their regulatory application as they become available rather than waiting until every section is finalized.
This process can facilitate more efficient regulatory review while enabling earlier interaction with regulatory authorities.
Advancing an Innovative Vision Restoration Therapy
MCO-010 represents one of the most advanced investigational optogenetic gene therapies currently under development for inherited retinal diseases.
Unlike many gene therapies that target individual genetic mutations, MCO-010 has been designed as a disease-agnostic treatment capable of restoring vision regardless of the specific genetic cause of retinal degeneration.
This broader applicability could allow the therapy to benefit a wider population of patients affected by advanced retinal diseases.
The investigational treatment focuses on restoring visual function rather than simply slowing disease progression.
Researchers believe this approach has the potential to address one of the greatest unmet needs in ophthalmology—helping patients who have already experienced significant photoreceptor loss and currently have few therapeutic options.
Addressing Retinal Degenerative Diseases
Retinal degenerative diseases encompass a group of inherited conditions characterized by the gradual deterioration of photoreceptor cells responsible for detecting light.
As these specialized cells progressively die, patients experience worsening vision that may eventually lead to severe visual impairment or complete blindness.
Among the conditions targeted by MCO-010 are retinitis pigmentosa and Stargardt disease, both of which remain challenging to treat, particularly during advanced stages.
Retinitis pigmentosa comprises a group of inherited retinal disorders that progressively reduce peripheral vision, night vision, and eventually central vision as photoreceptor cells deteriorate.
Stargardt disease primarily affects central vision and often develops during childhood or early adulthood, leading to substantial visual disability over time.
While advances in gene therapy have produced treatments for certain rare inherited retinal disorders, many patients remain ineligible because available therapies target only specific genetic mutations or require surviving photoreceptor cells.
Optogenetics Offers a Different Therapeutic Strategy
MCO-010 utilizes an innovative optogenetic approach that differs fundamentally from conventional retinal gene therapies.
Rather than replacing defective genes within dying photoreceptor cells, the therapy introduces genetic material that enables other surviving retinal cells to become responsive to light.
Specifically, the treatment reprograms retinal neurons that remain intact after photoreceptor degeneration, allowing them to detect light signals and transmit visual information to the brain.
This strategy seeks to bypass damaged photoreceptors while restoring meaningful visual function.
Because many retinal cells survive even after extensive photoreceptor loss, researchers believe this approach may benefit patients with advanced disease who would otherwise have limited treatment options.
Potential Benefits for Late-Stage Disease
One of the distinguishing features of MCO-010 is its focus on patients with late-stage retinal degeneration.
Many currently available investigational treatments require patients to retain a significant number of functioning photoreceptors.
Unfortunately, individuals with advanced disease often no longer meet these criteria.
MCO-010 was specifically developed to address this unmet need by targeting remaining retinal cells rather than relying upon surviving photoreceptors.
If successful, the therapy could potentially restore useful vision even after substantial retinal degeneration has occurred.
This patient population has historically faced limited therapeutic opportunities, making the development of new treatment approaches particularly significant.
Catalent Highlights Commitment to Innovation
Ricky Hopson, Group President of Clinical & Specialty Services and Chief of Staff at Catalent, emphasized the company’s commitment to supporting innovative therapies throughout every stage of development.
According to Hopson, expanding the collaboration with Nanoscope reflects Catalent’s broader mission of helping biotechnology companies successfully translate scientific innovation into approved therapies available for patients worldwide.
He noted that Catalent intends to continue supporting Nanoscope as MCO-010 advances toward commercialization and expressed confidence in the therapy’s potential to make a meaningful difference for individuals affected by retinal degenerative diseases.
Nanoscope Emphasizes Commercial Readiness
Nanoscope Therapeutics also highlighted the importance of securing experienced commercial manufacturing and packaging partners during this critical stage of development.
Co-Founder and Chief Executive Officer Sulagna Bhattacharya explained that as commercial production of MCO-010 continues to expand, reliable packaging and distribution capabilities become increasingly essential.
According to Bhattacharya, Catalent’s manufacturing expertise, operational reliability, and commercial experience provide confidence as the company prepares for potential global commercialization.
She emphasized that the partnership will help ensure readiness to meet future patient demand while maintaining the highest quality standards throughout product manufacturing and distribution.
Building for Global Commercialization
Although regulatory activities are currently focused on the United States, the expanded partnership also reflects long-term planning for broader international commercialization.
Commercial packaging systems capable of supporting multiple regulatory jurisdictions can simplify future market expansion while improving supply chain efficiency.
Catalent’s global manufacturing and distribution network may provide important infrastructure as Nanoscope pursues additional regulatory approvals beyond the United States.
Establishing scalable commercial capabilities early allows biotechnology companies to better respond to increasing demand following successful product launches.
Meeting the Needs of Patients with Limited Options
Patients living with inherited retinal degeneration frequently experience progressive vision loss despite ongoing medical care.
As diseases advance, available treatment options become increasingly limited, particularly for individuals who have already lost substantial numbers of photoreceptor cells.
By focusing specifically on restoring visual function rather than slowing degeneration alone, MCO-010 seeks to address an important unmet need within ophthalmology.
If approved, the therapy could provide new hope for patients who currently have few alternatives capable of improving meaningful vision.
The expanded partnership between Catalent and Nanoscope Therapeutics marks another important milestone in the development of MCO-010 as the investigational optogenetic gene therapy advances toward potential regulatory approval. By adding commercial packaging validation, labeling, and distribution services to their existing collaboration, the companies are strengthening the manufacturing and supply infrastructure needed to support a successful product launch.
With the FDA rolling Biologics License Application already underway, preparations are shifting from clinical development toward commercial readiness. At the same time, MCO-010 continues to represent a promising therapeutic approach for patients with advanced retinal degenerative diseases, including retinitis pigmentosa and Stargardt disease. If approved, the therapy could become an important new treatment option by restoring light sensitivity in surviving retinal cells and offering hope to individuals with severe vision loss who currently have limited therapeutic alternatives.
About Catalent
Catalent, Inc. is a leading global contract development and manufacturing organization (CDMO) championing the missions that help people live better and healthier lives. Every product that Catalent helps develop, manufacture and launch reflects its commitment to improve health outcomes around the world through its Patient First approach.
Catalent provides unparalleled service to pharma, biotech and consumer health customers, delivering on their missions to transform lives. Catalent tailors end-to-end solutions to meet customers’ needs in all phases of development and manufacturing. With thousands of scientists and technicians and the latest technology platforms at nearly 40 global sites, Catalent supplies billions of doses of life-enhancing and life-saving treatments for patients annually. For more information, visit www.catalent.com.
About Nanoscope Therapeutics
Nanoscope Therapeutics is developing disease-agnostic, vision-restoring optogenetic therapy for millions of patients blinded by retinal degenerative diseases. Following positive results from the RESTORE Phase 2b/3 multicenter, randomized, double-masked, sham-controlled clinical trial for retinitis pigmentosa (RP) (NCT04945772), a rolling BLA submission to the FDA has been initiated.
If approved, MCO-010 has the potential to be the standard of care for RP patients, administered as a one-time, in-office injection without the need for genetic testing. The company has also shown promising results in the STARLIGHT Phase 2 clinical trial of MCO-010 in Stargardt disease (SD) (NCT05417126) and plans to initiate a Phase 3 registrational trial in 2026.
MCO-010 has received FDA Fast Track and Orphan Drug designations for both RP and SD, along with RMAT designation for SD, as well as PMDA Sakigake and Orphan designations for inherited retinal dystrophies (IRDs) and EMA, SFDA Orphan designations for IRDs. A Phase 2 program for MCO in geographic atrophy (GA) is expected to start in 2026. Other IND-ready programs include Leber congenital amaurosis (LCA).