Summary
Regeneron Pharmaceuticals has announced a partnership with Tessera Therapeutics to develop a gene editing treatment for alpha-1 antitrypsin deficiency (AATD), a rare liver and lung disease. The deal involves a $150 million upfront payment from Regeneron to collaborate on the program and split development costs and profits, with potential milestone payments of $125 million. Tessera will lead the first-in-human trial, with Regeneron taking over for future development and commercialization.
Access impact
The partnership between Regeneron and Tessera has significant implications for pricing and reimbursement of the potential gene editing treatment for AATD. If successful, this treatment could become a top target among drugmakers for the rare disease, potentially leading to competition and potentially lower prices. Additionally, the one-time treatment approach may also impact reimbursement decisions, as it may be seen as a more cost-effective option compared to ongoing treatment options for AATD.
Top-3 domain lens
Clinical Effectiveness: The article mentions that the gene editing treatment being developed by Tessera, TSRA-196, is designed to precisely correct the underlying genetic mutation in AATD and durably restore production of the AAT protein through a one-time treatment. This could have a significant impact on the clinical effectiveness of the treatment, potentially providing a more durable and effective solution for AATD patients.
Budget Impact & Resources: If approved, the one-time gene editing treatment for AATD could potentially have a significant impact on healthcare budgets. While the upfront cost may be higher, the long-term cost of ongoing treatment for AATD could be reduced, potentially making it a more cost-effective option for payers.
Evidence Quality & Robustness: The article mentions that Tessera has presented preclinical data for their gene editing treatment and is expecting to begin human testing by the end of 2025. This suggests that there is still a level of uncertainty and lack of robust evidence for the treatment, which could impact its evaluation by payers and HTA agencies.
Hashtags
#MarketAccessRiskAssessment #Pricing #Reimbursement #MarketAccess #MARArating #Regeneron #Tessera #AATD
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Source
source: https://www.biopharmadive.com/news/regeneron-gene-editing-deal-tessera-AATD/806650/