Wave Life Sciences

Intellia Therapeutics is restructuring, discontinuing its NTLA-3001 program for alpha-1 antitrypsin deficiency, laying off over 25% of staff, and focusing on NTLA-2002 and nex-z for hereditary angioedema and transthyretin amyloidosis. Laura Sepp-Lorenzino retires as CSO, succeeded by Birgit Schultes.

75% of C-suite executives in life sciences are optimistic about 2025, citing growth and innovation. AI in biopharma is expanding, with increased investments and partnerships. Cancer vaccines and gene editing are advancing, with late-stage trials and new therapies. VC and IPO markets are rebounding, and M&A activity is expected to increase. Multiomics tools are expanding, and spatial biology is consolidating. Regulatory changes under Trump 2.0 may impact biopharma and health policy.

2024 marked significant progress in Huntington’s disease (HD) research, with breakthroughs in understanding somatic instability, advancements in drug development, and both challenges and triumphs in clinical trials. The HD community saw the power of collaboration and innovation, with new voices at HDBuzz and updates from global conferences. Despite setbacks, there's hope with ongoing trials and new drug approvals, moving closer to treatments that could slow or halt HD.

In 2024, Muscular Dystrophy News Today highlighted key developments in MD research, including gene therapy trials, new treatments like SAT-3247 and Elevidys, and clinical advancements for DMD and FSHD, emphasizing progress in muscle function and dystrophin production.

Joon Lee of Truist Financial maintains a Buy rating on Axsome Therapeutics with a $150 price target, citing positive results from AXS-05 studies for Alzheimer’s disease agitation. Axsome plans to submit a New Drug Application by 2025, with AXS-05 projected to generate $2.3 billion in sales by 2034. Needham also maintains a Buy rating with a $130 target.

Wave Life Sciences announced the submission of its first clinical trial application for WVE-007, an investigational siRNA designed to silence INHBE gene expression, inducing fat burning for sustainable weight loss without impacting muscle mass. Wave expects CTA approval and initiation of the first-in-human study in 1Q 2025.

Wave Life Sciences submitted its first clinical trial application for WVE-007, an investigational GalNAc-conjugated siRNA designed to silence INHBE gene expression, inducing fat burning for obesity treatment without impacting muscle mass. Wave expects CTA approval and initiation of the first-in-human study in 1Q 2025.