In Duchenne muscular dystrophy (DMD), deficiency of dystrophin leads to cardiomyocyte membrane instability, abnormal calcium influx, and progressive fibrotic remodeling of cardiac tissue. Histone deacetylase 6 (HDAC6) contributes to disease progression by regulating cytoskeletal dynamics and proteostasis in dystrophic muscle cells. Consequently, inhibition of HDAC6 represents a potential therapeutic strategy for addressing both the skeletal and cardiac manifestations of DMD.
Entering its first major cardiovascular disease collaboration with a biopharma company, while it advances two internal gene therapies, Tenaya Therapeutics Inc. signed on with Alnylam Pharmaceuticals Inc. to deliver up to 15 novel genetic targets that could lead to new heart disease medicines. The deal comes with $10 million up front, and up to $1.13 billion is available to South San Francisco-based Tenaya if all targets meet certain milestones, leading to approved therapeutics that Alnylam develops and commercializes.
Entering its first major cardiovascular disease collaboration with a biopharma company, while it advances two internal gene therapies, Tenaya Therapeutics Inc. signed on with Alnylam Pharmaceuticals Inc. to deliver up to 15 novel genetic targets that could lead to new heart disease medicines. The deal comes with $10 million up front, and up to $1.13 billion is available to South San Francisco-based Tenaya if all targets meet certain milestones, leading to approved therapeutics that Alnylam develops and commercializes.
Gene therapy has had its commercial struggles in the past year. The cost to patients is in the millions and fewer are stepping forward for treatment than companies would like. While development continues in this game-changing field, some have struggled with regulatory authorities during development while others have just stepped away altogether.
Wall Street must wait until 2025 for data from the higher-dose cohort in Tenaya Therapeutics Inc.’s phase Ib/II study with gene therapy TN-201 for MYBPC3-linked hypertrophic cardiomyopathy (HCM), but shares (NASDAQ:TNYA) closed Dec. 17 at $1.41, down $1.47, or 51%, after results from the first cohort rolled out.
Biopharma companies have announced additional layoffs and restructurings in the past few weeks, after more than 7,800 job cuts were reported by BioWorld this year through late April. Through mid-May, total layoffs in the biopharma sector have increased to 9,848.
Heart failure with preserved ejection fraction (HFpEF) has limited treatment options and remains a medical challenge. HFpEF is characterized by diastolic dysfunction with a normal ejection fraction. Previous findings have shown that inhibiting histone deacetylase 6 (HDAC6) alters the mechanisms contributing to dilated cardiomyopathy.
With one approved myosin inhibitor on the market and another coming up fast, researchers such as those at Tenaya Therapeutics Inc. are casting for new strategies to treat hypertrophic cardiomyopathy (HCM).
The FDA has awarded orphan drug designation to Tenaya Therapeutics Inc.'s gene therapy product candidate, TN-401, for the treatment of arrhythmogenic right ventricular cardiomyopathy (ARVC). TN-401 is an adeno-associated virus (AAV)-based gene therapy being developed for the treatment of genetic ARVC caused by plakophilin-2 (PKP2) gene mutations.
