4th ALS Drug Development Summit

The ALS research landscape is evolving rapidly, with groundbreaking advancements shaping the future of therapeutic development. From computational target discovery to novel molecular mechanisms, the industry is pushing boundaries to accelerate the delivery of effective treatments for ALS and FTD.
Recent milestone achievements underscore the potential of cutting-edge approaches in ALS research. Insitro’s collaboration with Bristol Myers Squibb has demonstrated the power of computational methods in target discovery, paving the way for more precise therapeutic strategies. Meanwhile, Dewpoint Therapeutics' partnership with Mitsubishi Tanabe Pharma America to advance TDP-43 condensate modulators highlights the growing interest in harnessing biomolecular condensates to combat ALS pathology.
At the same time, clinical advancements are making significant strides. QurAlis’ QRL-101 and QRL-201 trials offer new hope for ALS patients by targeting neurodegenerative mechanisms with innovative therapies. Alongside this, breakthroughs in biomarker platforms—such as TDP-43 PET tracers and neuro-metabolic markers—are refining patient stratification and enabling earlier disease detection, crucial steps in improving therapeutic outcomes.
As the ALS field progresses, 200+ scientific leaders will gather in Boston at the 4th ALS Drug Development Summit to address the most pressing challenges in therapy development. Discussions will center on target validation, biomarker utilization, and clinical trial optimization, all aimed at expediting the journey from discovery to patient-ready treatments.
Key Takeaways from ALS Industry Experts:
- Mechanistic Innovation: Gain novel insights into STMN2, UNC13A, mitochondrial dysfunction, and neuroinflammation as emerging therapeutic targets for ALS pathophysiology.
- Precision Biomarkers: Explore advancements in TDP-43 PET tracers, NFL, and neuro-metabolic biomarkers to enable earlier diagnosis and improve patient selection.
- Patient-Centric Clinical Design: Assess adaptive and decentralized clinical trial strategies that enhance inclusivity, reduce patient burden, and align with evolving regulatory expectations.
- Collaborative Impact: Discover how industry consortia such as Target ALS, Answer ALS, and AMP ALS are harmonizing data and fostering multi-stakeholder collaboration to accelerate therapy development.
Who Will You Meet?
The 4th ALS Drug Development Summit brings together 200+ key stakeholders from pharma, biotech, and academia, spanning translational medicine, clinical development, biomarker discovery, precision medicine, regulatory affairs, and medical affairs. Attendees will also include clinicians, patients, caregivers, and advocates—all dedicated to advancing meaningful research in ALS.
From C-Level executives to clinical research scientists, leading figures in the field will convene this May to explore the latest unpublished data, engage in dynamic panel discussions, and participate in deep-dive workshops not available anywhere else. The summit presents a unique opportunity to connect, collaborate, and contribute to the next wave of innovation in ALS drug development.