GEMMA Biotherapeutics

GEMMABio Founder, President, and CEO and Franklin Biolabs Founder and Executive Chair James M. Wilson, MD, PhD will discuss the future of gene therapy at #STATBreakthrough East on Thursday morning, March 20! https://lnkd.in/g9xXZP7K #GeneTherapy #RareDisease #biotech

Thank you Rojan Vakili for sharing your family's story with our staff last week on Rare Disease Day. It was so inspiring, and our employees truly appreciated learning about your family's experience.

GEMMABio is ranked the #1 startup on the 2025 RealLIST for Greater Philadelphia by Technical.ly! The RealLIST Startups are young companies ranked by major events like big fundraises, participation in accelerators, notable investors, and noble missions.? Check out the feature here: https://lnkd.in/eBKxz29q

On Rare Disease Day 2025, check out this Q&A by Drug Discovery World for insights from a range of industry experts, including James M. Wilson, MD, PhD from GEMMA Biotherapeutics. https://lnkd.in/eRAxQ8eP

Today is Rare Disease Day! Join GEMMA Biotherapeutics in recognizing the 300 million people worldwide living with a rare disease. Today, we wear stripes as the zebra is a powerful symbol for rare diseases. Historically, medical professionals were taught, “When you hear hoofbeats, think horses, not zebras”—a reminder to consider common diagnoses before rare ones. However, we now know that rare diseases are more prevalent than previously understood, affecting 1 in 10 Americans. By wearing stripes, you can show your solidarity and help raise awareness for the hundreds of millions of individuals who deserve our recognition and support. Be sure to visit www.rarediseaseday.org to learn how you can participate in Rare Disease Day activities, find helpful resources for educating others and spreading awareness, and ramp up your social media in honor of the rare community! #rarediseaseday #raredisease #rarediseaseadvocacy?

It’s Rare Disease Week! This week culminates with Rare Disease Day, on Friday 28th February, a global movement that aims to recognize the 300 million people worldwide who are living with a rare disease. We observe Rare Disease Day to champion equity in social opportunity, healthcare, and access to diagnosis and treatments for individuals living with a rare disease. This week, 600+ events will be held around the world to acknowledge, celebrate, and support those who are impacted, enabling progress and amplifying advocacy. This year’s Rare Disease Day theme is “More than You Can Imagine”, which shines a spotlight on coming together as a community in honor of rare diseases. Because together, we can do more than you can imagine. Watch the official RDD 2025 Video here - https://lnkd.in/dE56sfWf (1 min) More than 10,000 rare diseases affect over 300 million people worldwide, and only 5% have an FDA-approved treatment. Not one rare disease has an available “cure”. By recognizing Rare Disease Day on February 28th, we can help raise awareness and drive support for critical research and patient advocacy. Check out www.rarediseaseday.org for tons of resources to help you advocate for and support the rare disease community. #rarediseaseday #raredisease #rarediseaseadvocacy?

Check out this new feature on the Bioculture Group! It is entitled 'Caring for Life: Bioculture’s Journey from Biodiversity to Global Health Innovation.' https://lnkd.in/eDXeskYN

Congratulations to GEMMABio Founder, President, and CEO, James M. Wilson, MD, PhD, who will be honored today with the PMWC - Precision Medicine World Conference 2025 Pioneer Award for Gene and Cell Therapies.?The award recognizes his groundbreaking work in viral vector-based gene therapies, significantly impacting the treatment of rare genetic diseases.?His keynote presentation is entitled 'The Promise of Genetic Medicines Unless You're Too Poor or Too Rare.' #PMWC25 #RareDiseases #GeneTherapy

Congratulations to GEMMABio Founder, President, and CEO, James M. Wilson, MD, PhD, who will be honored today with the WORLDSymposium 2025 Roscoe O. Brady Award for outstanding innovation and accomplishment in the field of lysosomal disease research and therapy.?His award presentation is entitled Lysosomal Diseases: A paradigm for personalized medicine. #LysosomalDisease #RareDiseases #GeneTherapy

Read the Endpoints News article by Ryan Cross about the first-ever case of in vivo gene editing in an infant here: https://lnkd.in/e2-iF_j2 Following treatment with iECURE, inc.’s new gene therapy, a newborn with Ornithine Transcarbamylase Deficiency (OTCD) appears to have had a complete clinical response.