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AVROBIO (AVRO) Stock Up on Upbeat Data From Cystinosis Study

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AVROBIO (AVRO - Free Report) announced positive interim data from a phase I/II study, which is evaluating the safety and efficacy of AVR-RD-04, an investigational gene-therapyfor cystinosis, a rare but progressive genetic disease.

Initial results from the phase I/II study indicate that following administration of AVR-RD-04, participants produce and distribute functional cystinosin protein throughout the body, thereby preventing the accumulation of cystine crystals. A build-up of cystine crystal can cause progressive organ damage, including shortened lifespan. In fact, data from this study also showed key visual-motor integration, visual perception and motor coordination measures that were impacted by cystinosis to have stabilized and even improved in participants following administration of the gene therapy.

The phase I/II study, sponsored by the University of California San Diego (UCSD), is currently evaluating AVR-RD-04 in adult patients who were diagnosed with the infantile form of cystinosis, previously treated with cysteamine, the current standard of care (SOC) for cystinosis.

All patients administered a dose of AVR-RD-04, continue to remain off SOC. In fact, following discontinuation of the SOC, the average hand-grip strength of the participants was stable up to 27 months after being dosed with the gene-therapy candidate.

Shares of AVROBIO rose 4.7% on Tuesday (May 17) and again gained 5.4% in after-hours trading on that same day, following the above announcement. Yet, the stock has plunged 79.2% this year so far compared with the industry’s 22.9% decline.

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We note that AVR-RD-04 is designed to genetically modify a patient’s hematopoietic stem cells (HSC) to produce functional cystinosin protein as well as stabilize and even reduce the impact of cystinosis on different tissues.

Based on the above interim analysis of data, AVROBIO intends to self-sponsor a clinical study next year, evaluating the candidate. AVRO's current plan for AVR-RD-04 consists of a two-part strategy, which starts with a pre-renal transplant population followed by a post-renal transplant population. AVRO also plans to meet the FDA officials later this year to discuss its clinical strategy for AVR-RD-04.

Zacks Rank & Stocks to Consider

AVROBIO currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the overall healthcare sector are Abeona Therapeutics (ABEO - Free Report) , Alkermes (ALKS - Free Report) and Angion Biomedica (ANGN - Free Report) , each of which has a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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