Actio Biosciences, Inc.

We’re pleased to share our upcoming participation in two investor conferences, Stifel Financial Corp.’s 2024 Healthcare Conference on November 19th, and the Wells Fargo Virtual Private Biotech Symposium on December 12th. Read more: https://lnkd.in/eQpAHEuh

Coming up, our CEO, David B. Goldstein will join an insightful panel discussion at Personalized Medicine Coalition’s 18th Annual Personalized Medicine Conference on the future of personalized medicine for pediatric patients with rare genetic diseases. As advancements in genomic sequencing and #genetherapy transform healthcare, David and fellow industry leaders explore the possibilities and the challenges ahead. Discover more and join us for the conversation: https://lnkd.in/dzB6E7mM

We’re pleased to be a part of this year’s #ASHG24, closing the annual meeting on Saturday, November 9th with the Distinguished Speakers Symposium panel, “The Promise and Payoff of Human Genetics and Genomics: Paths from Bench to Bedside.” Actio CEO, David B. Goldstein Ph.D., will take the stage among other industry leaders to discuss translating basic science discoveries into new therapies, addressing both the scientific and ethical challenges of bringing these advancements to patients.      Learn more: https://lnkd.in/erqbVzgV 

For Actio, bringing meaningful therapies to patients is our top priority. To get a better, first-hand understanding of the patient journey, we worked with the Charcot-Marie-Tooth Association to host a virtual patient focus group for individuals diagnosed with Charcot-Marie-Tooth disease Type 2C, (CMT2C), also known as TRPV4 neuropathy. It's an honor to hear their stories and gain insights into the challenges they face every day. By listening to the voices of those directly impacted by #CMT2C, we can better tailor our research and development efforts to meet their needs. Learn and read more from our friends at CMTA below.

We’re pleased to share our participation in two upcoming investor conferences in October! → Our chief executive officer, David B. Goldstein, Ph.D., will take part in a fireside chat and 1x1 meetings at the #RBC2024 RBC Capital Markets Biotechnology Private Company Virtual Conference on Tuesday, October 1st. → Additionally, our team will participate in 1x1 meetings Leerink Partners Biopharma Private Company Connect on Wednesday, October 23rd. We look forward to these opportunities to connect with the industry and discuss our efforts to develop new therapeutics that target shared underlying biology in both rare and common diseases. Learn more: https://lnkd.in/eyM8X3jC

Actio's Co-Founder and CEO, David B. Goldstein, will take part in the "Data-for-purpose: Go big, go deep, or go long?" panel on BioCentury Inc.'s third day of Grand Rounds in Nashville next week, to discuss optimal ways to make data fit for purpose among other leading industry drug developers, academics and investors.    Sunil Sahdeo Ph.D., Actio Vice President, Head of Operations, will also be there to present a poster on practicing inclusive precision medicine at Actio.   Don't miss the insights. Learn more and join us: https://lnkd.in/gakuvuEn 

We’re overjoyed to have a few adorable new additions to our Actio family. There are some proud new parents in our mix and we couldn’t be happier for them - so we hosted celebrations to welcome their little ones. Wishing the proud, new parents endless moments of joy, cuddles, and sweet baby smiles!

It’s an honor to welcome Dimitrios Arkilo, M.D., as our chief medical officer. Dr. Arkilo brings a unique combination of experience in clinical practice and the biotech industry, with a focus on neurology, epilepsy and rare disease, including a number of childhood conditions.   We look forward to Dr. Arkilo’s expert guidance in driving clinical strategy across our portfolio, including two lead programs heading to the clinic in #geneticepilepsy and #CMT2C. Learn more: https://lnkd.in/epJj9Bw7

Today, we’re pleased to share that we've received both orphan drug designation and rare pediatric disease designation from the FDA for ABS-0871, a TRPV4 inhibitor, for the treatment of Charcot-Marie-Tooth Disease 2C, or #CMT2C.   Receiving these designations emphasizes the urgent need for treatments for CMT2C patients, who often live with debilitating symptoms like muscle weakness, vocal cord paresis and respiratory complications. We’re proud to be pursuing solutions for CMT2C with support from our STAR Alliance Partner, the Charcot-Marie-Tooth Association through their Patients as Partners platform. We will continue to advance this program as quickly as possible.    Read more: https://lnkd.in/eaEKKVbV

This week, we hosted our first fireside chat moderated by Actio co-founder John McHutchison, AO, MD, with Sheila Gujrathi, MD, biotech entrepreneur and executive, healthcare investor, and deeply experienced drug developer, to discuss novel clinical trial design and some of the most important lessons learned along the way. Her insights were highly valued among the Actio team and it was a pleasure to hear her perspective. Extending a huge thank you to Dr. Gujarati for joining us!