Kyverna Therapeutics, Inc.

German physicians at Ruhr-University Bochum successfully treated two patients with chronic inflammatory demyelinating polyneuropathy (CIDP) using CAR-T cell therapy, achieving over 200% improvement in clinical scores and neurophysiological tests.

The CAR-T cell therapy industry has raised over $141.2 billion through various financing mechanisms, with estimates suggesting total industry funding could reach $281.7 billion when including undisclosed deals.

The global lupus nephritis market is projected to grow significantly by 2034, with the United States accounting for over 80% of the current $1.12 billion market share across major regions.

• The CAR-T cell therapy industry has raised over $141.2 billion through various financing mechanisms, with potential total value reaching $281.7 billion when including undisclosed deals. • Since 2014, 89 CAR-T companies have secured $7.7 billion in venture capital funding, while 42 companies have raised $6.4 billion through IPOs, demonstrating sustained investor confidence. • Despite a slowdown in M&A activity in 2024, the sector continues to expand with 170+ companies developing 1,944 therapies, including 244 trials targeting solid tumors beyond the current blood cancer focus.

• Donanemab's enhanced titration method significantly reduces ARIA-E frequency and severity while maintaining amyloid reduction in Alzheimer's patients. • Liraglutide demonstrates neuroprotective effects against Alzheimer's disease dementia, highlighting the potential of GLP-1 analogues in AD treatment. • Tolebrutinib shows promise in slowing disability progression in non-relapsing secondary progressive multiple sclerosis patients in the HERCULES trial.

UChicago Medicine launched a Phase 2 clinical trial to explore CAR T-cell therapy for systemic lupus erythematosus, inflammatory myositis, and systemic sclerosis.

• The lupus nephritis clinical trial pipeline is robust, featuring over 30 companies developing more than 35 therapies. • Key players like AstraZeneca, Roche, and Novartis are advancing drugs such as Anifrolumab and Ianalumab through clinical trials. • Recent trial activities include Adicet Bio dosing the first patient in a Phase I trial of ADI-001 and Kyverna presenting updated clinical data on KYV-101. • Various therapeutic modalities, including monoclonal antibodies and cell therapies, are being explored across different clinical stages.

KYV-101, an experimental cell therapy, has been granted orphan drug status by the European Medicines Agency for the treatment of myasthenia gravis (MG).

• The multiple sclerosis (MS) therapeutic landscape is expanding, with over 80 active pipeline therapies currently in development by more than 75 companies. • Recent clinical trials have yielded mixed results, with some therapies showing promise in specific MS subtypes, such as non-relapsing secondary progressive MS (nrSPMS). • Regulatory milestones have been achieved, including FDA approval for new formulations and fast-track designations for therapies targeting progressive MS. • Emerging therapies in the MS pipeline include monoclonal antibodies, oral treatments, and CAR-T cell therapies, offering diverse mechanisms of action and routes of administration.

• The gene therapy market is experiencing substantial growth, fueled by technological advancements, increased investments, and a deeper understanding of genetic diseases. • Key players are actively engaging in collaborations, mergers, and acquisitions to enhance their research and development capabilities and broaden their product portfolios. • Regulatory bodies like the FDA and EMA are expediting the approval process for gene therapies, acknowledging their potential in addressing unmet medical needs. • Ethical considerations and public perception surrounding gene editing, particularly germline modifications, remain critical factors influencing market dynamics and regulatory policies.