Andelyn partners with Celliaz to manufacture AAV gene therapy for retinitis pigmentosa

Andelyn Biosciences and Celliaz have entered a strategic partnership to support development and manufacturing of CLZ002, an investigational AAV-based gene therapy for retinitis pigmentosa.

Under the agreement, Andelyn will provide development and manufacturing services for the programme using its Curator Suspension Platform.

The work will include AAV2 vector and plasmid manufacturing to support IND-enabling studies and preparations for a planned Phase I/II clinical trial.

CLZ002 is being developed by South Korea-based Celliaz as a potential gene-agnostic treatment for retinitis pigmentosa.

Retinitis pigmentosa comprises a group of inherited retinal disorders that cause progressive vision loss and can result from mutations across more than 90 genes.

Rather than targeting an individual genetic mutation, Celliaz is developing CLZ002 to interfere with the transfer of PROX1 into Müller glia from neighbouring retinal cells.

The company said PROX1 suppresses the regenerative activity of Müller glia, which retain latent regenerative potential within the retina.

By preventing this transfer, CLZ002 is intended to restore the cells’ intrinsic regenerative capacity and potentially provide an approach applicable across multiple genetic forms of retinitis pigmentosa.

The programme uses an AAV2 capsid for delivery.

Matt Niloff, Chief Commercial Officer at Andelyn Biosciences, said the company will apply its experience in AAV manufacturing, ophthalmology and rare disease development to support progression of CLZ002 toward clinical testing.

The partnership also adds to Andelyn’s existing work supporting biotechnology programmes based in South Korea.

Kyung Hwa Kang, PhD, Chief Executive Officer of Celliaz, said the genetic diversity of retinitis pigmentosa creates challenges for approaches directed at individual disease-causing mutations.

She said CLZ002 is intended to take a different approach by targeting a mechanism involved in retinal regeneration rather than a specific underlying mutation.

Andelyn will support the programme through its integrated plasmid and viral vector manufacturing capabilities alongside the Curator Suspension Platform.

Celliaz plans to advance CLZ002 through IND-enabling development before initiating a Phase I/II clinical study.

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Related Topics and Keywords

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