Every spring, the Target ALS Annual Meeting gathers talented scientists from our Innovation Ecosystem for three days of sharing groundbreaking research and forging new partnerships, all aimed at advancing ALS research. This year’s event marked a milestone with record-breaking attendance, showcasing the expanding influence and reach of our collective efforts. Attendees from academic institutions, biotech and pharmaceutical companies, venture capital firms, and nonprofit organizations joined us both in person and virtually, reflecting the robust growth and impact of our work. 

Following the conference, our CEO, Manish Raisinghani, reflects on the profound impact and inspiring insights shared during the meeting:

Letter from the CEO

Each spring, the Target ALS Annual Meeting brings members of our Innovation Ecosystem together over three days to share the latest breakthroughs and forge new collaborations, driving progress toward effective treatments for ALS. This year’s meeting had the largest attendance in history, with over 850 attendees present from academic institutions, pharmaceutical and biotech companies, venture capital firms and nonprofit organizations – both in-person and virtually – reflecting the growth of both our funded portfolio of scientists and the impact our work has had on expanding ALS research. 

Despite the record-breaking attendance, we have continued to foster close relationships and trust among the scientists present that has become a hallmark of our conference. This atmosphere is critical for researchers to feel comfortable sharing unpublished data from their ongoing work and engage in rigorous discussions that can lead to new ideas and new partners to accelerate research from the laboratory bench to the patient bedside. Underscoring this sentiment, Dr. Joseph Lewcock, Ph.D., Chief Scientific Officer at Denali Therapeutics said, “I’ve had the pleasure of being at every Target ALS meeting since the first one. I can see it’s been incredibly impactful to be involved, not just through the consortia that we’ve been a part of, but through the relationships we’ve built at this meeting.” 

This year, we heard presentations focused on our three investment areas: identifying novel drug targets by improving our understanding of disease biology, driving development of therapeutics to transform ALS from a terminal disease into a chronic disease, and discovering biomarkers to improve diagnosis, disease monitoring, and clinical trials. The presentations not only showed progress across each of these areas, but expanded our perspective of ALS – featuring new avenues of study that are not typically associated with the disease, like role of the immune system or the microbiome in the gut – and demonstrated how our model derisks and accelerates ideas towards the clinic – sharing clinical trial milestones for new therapeutics developed with our support. Some of the most exciting highlights include:

• Two biotech companies have advanced their active clinical trials, which are based on early-stage funding from Target ALS, to the next phase.
• Multiple research groups funded by Target ALS are applying cutting-edge gene editing technologies to develop new treatment strategies for ALS. 
• An emerging generation of researchers showcased their work exploring novel areas of ALS biology like the immune and digestive systems to find new drug targets.
• Exciting progress was presented on generation of large multi-omic data sets publicly shared through our new data portal. These two ingredients are key to accelerating application of Artificial Intelligence/Machine Learning.
• Promising progress was presented on new biomarkers which included blood-based and non-invasive approaches to diagnose and monitor ALS progression. 

Progress presented at the Annual Meeting has been made possible thanks to you and our Founder Dan Doctoroff’s pioneering $250 million Capital Campaign, which he’ll share more about in his concluding remarks. This bold endeavor has allowed us to triple our research investment in the past three years alone and with your support, we’ll continue to make significant strides towards finding effective treatments for ALS. 

Beyond science, perhaps the brightest highlight of the meeting for myself was meeting with people living with ALS and their loved ones who offered their voices to inform and motivate our efforts. In her remarks on the second day of the conference, Alisa Doctoroff gave the room a glimpse into hers and Dan’s daily experience of life with ALS, a perspective that scientists don’t often get to see firsthand. Her words underlined the urgency, but also the optimism, that herself and Dan lead us with every day. 

Because of you, Target ALS will continue to be audacious and urgent in our approach. We will continue to fund the best ideas from the brightest minds across the globe, furthering the drug discovery pipeline toward effective treatments for ALS. We believe that science driven by a meaningful mission can change the world. In our vision for a changed world, Everyone Lives. Thank you. 

Sincerely,

Manish Raisinghani

CEO, Target ALS