Rafa's Moonshot

Neurodiversity is everywhere—even in the spotlight 🌍 These famous creatives have shown the world what’s possible with dyspraxia. #DyspraxiaAwareness #Neurodiversity #RafasMoonshot #DifferentNotLess #FamousAndNeurodivergent

Imagine if tying your shoes felt like solving a Rubik’s Cube every morning 🧩 That’s life with dyspraxia—a neurodevelopmental condition where the brain struggles to plan and coordinate movement. Dyspraxia, also known as Developmental Coordination Disorder (DCD), makes that everyday activities like writing, tying shoelaces, or even riding a bike require extra focus, innovation, and persistence. For many, dyspraxia isn’t just about a few clumsy moments—it’s a constant challenge that shapes how they interact with the world. Yet, despite these obstacles, people with dyspraxia often develop unique ways to adapt and excel. 💪 Here’s a closer look at the key features of dyspraxia: 🤸 Movement & Coordination: Even basic actions can feel like a puzzle, affecting both fine motor (like handwriting) and gross motor (like running) skills. 🚧 Everyday Hurdles: Simple routines—getting dressed, preparing a meal—can demand extra effort and time. 💡No Impact on IQ: Despite trouble with planning and sequencing, intelligence isn’t affected. 🔄 Common Overlaps: Often coexists with ADHD, dyslexia, or autism, reflecting the complexity of neurodevelopmental diversity. 🩺 Therapy & Support: There’s no cure, but occupational therapy, physical therapy, and classroom adjustments make a huge difference. At Rafa’s Moonshot, we believe in spotlighting conditions like dyspraxia to foster understanding, empathy, and solutions. Just as we’re on a mission to transform STXBP1 research, we’re also inspired to push for broader awareness around all forms of neurodiversity. The right support system—through tailored therapies, practical tools, and community backing—can help individuals with dyspraxia navigate life with greater confidence and ease. ✨ #Dyspraxia #Neurodiversity #RareDiseaseAwareness #RafasMoonshot #Inclusion #Empowerment #DCD #OccupationalTherapy

What if we could discover life-saving treatments—without testing on a single animal? 🧠🐭 The FDA is now paving a path toward a more ethical and efficient approach to drug development by embracing brain organoids, AI-driven models, and organ-on-a-chip technology. Science is driving this evolution with human-based systems that better reflect our own biology. Lab-grown tissues that behave like real human organs, coupled with AI algorithms to predict drug behavior, can fast-track discovery, lower development costs, and reduce ethical concerns—all while delivering more accurate insights from the start. Here’s how it’s unfolding: 🧠 Brain Organoids: Lab-grown clusters of human brain cells that mimic real neural structures, offering deeper insights into neurological diseases. 🤖 AI-Driven Models: Advanced simulations that predict a drug’s toxicity and effectiveness—often more quickly than animal tests. ⚙️ Organ-on-a-Chip: Miniaturized systems that replicate human organ functions, helping researchers see how treatments interact with the body in real time. By combining these cutting-edge methods, scientists can test drug interactions, predict side effects, and refine treatments without putting animals at risk. Beyond the ethical benefits, this means speeding up clinical breakthroughs, reducing costs, and better capturing the complexity of human biology. It’s a testament to what’s possible when innovation, urgency, and a shared purpose come together. Here’s hoping more organizations follow suit—together, we can reshape the future of medicine. #FDA #DrugDiscovery #Innovation #RareDiseaseResearch #RafasMoonshot #AI #Organoids #ScienceForGood

Bringing back an extinct predator with CRISPR isn’t science fiction anymore—it’s this week’s headline. 🐾 Scientists just announced the birth of three genetically engineered wolf pups—Romulus, Remus, and Khaleesi—designed to resemble the long-extinct dire wolf. CRISPR turned ancient DNA into living biology—and the implications go far beyond wolves. Their method? 📜 Ancient DNA 🧬 CRISPR gene editing 🐺 And a modern gray wolf genome By altering 20 specific genes and implanting the embryos into dog surrogates, they didn’t just make headlines—they raised a question scientists have been circling for years: Can we really bring something back from extinction? That debate aside, the real breakthrough is in the science. 🔬 The tools used to build these pups—whole genome sequencing, precision editing, CRISPR-Cas9, synthetic biology—are the same tools we’re using to tackle single-gene neurological disorders like STXBP1. And this is where the science gets even more powerful. In STXBP1 disorders, one tiny mutation can break a child’s ability to walk, talk, or move. The neurons are there, the brain is there, but the communication is broken. CRISPR offers a way to fix the message at the source. Not treat symptoms - correct the mutation. 🧠 At Rafa’s Moonshot, we’re leveraging the same frontier technologies—AI, CRISPR, stem cell modeling—not to bring back the past… …but to rewrite the future for children living with devastating conditions today. De-extinction may be headline-worthy. But gene repair is life-changing. And we’re just getting started. To read more about this breakthrough, see this Time article: https://lnkd.in/ehbK2wEU #CRISPR #STXBP1 #GeneticMedicine #DeExtinction #Biotech #GeneEditing #RafasMoonshot #FutureOfMedicine

You don’t notice movement—until it stops working. Walking, writing, smiling… it’s all second nature. Until one day, it’s not. That’s the hidden weight of motor disorders—and why they matter more than we think. 🧠 The Body’s Command Center, Interrupted Motor disorders are a group of conditions that mess with the brain’s ability to coordinate movement. Some are hyperkinetic—with too much movement, like tics, tremors, or the jerky rhythm of chorea. Others are hypokinetic—like Parkinson’s disease, where motion slows down or stiffens. Either way, they affect what we often take for granted: walking, talking, balance, even writing your name. 📍Symptoms Can Sneak Up - Involuntary jerks or shakes - Muscle stiffness or cramps - Unsteady walking or posture changes - Trouble with coordination These signs might appear suddenly or progress slowly. They might be rooted in genetics, injuries, infections—or arrive without clear warning. 🩺 Diagnosis and Treatment Are a Journey Understanding the "why" behind the movement takes time. Neurological exams, imaging, sometimes genetic testing… they’re all pieces of the puzzle. Treatment could mean meds, therapy, or even surgery—but early action is everything. 🚀 At Rafa’s Moonshot, we believe that awareness leads to answers. When families know what to look for, they can take action sooner. And when experts collaborate across science, medicine, and tech—we push the boundaries of what’s possible. Because movement matters. And for the families dealing with motor disorders, every step forward counts. ✨ #MotorDisorders #MovementMatters #NeuroResearch #STXBP1Awareness #RafasMoonshot

🚀 Yesterday at the PharmaTech Founders Forum – Investor Edition, Sagi Gidali took the stage to share the story behind Rafa’s Moonshot: a mission born from urgency, built on science, and powered by execution. In a room full of investors, founders, and scientists, one message came through loud and clear: In TechBio, breakthrough science is just the beginning. 💫 To create real impact, you need vision, team, and the relentless drive to turn research into results. Sagi spotlighted how Rafa’s Moonshot went from diagnosis to drug discovery in under 6 months—applying startup thinking to rare disease research. 🧠 From AI-powered simulations to preclinical testing 💊 From academic labs to investor pitches 📈 From one child’s diagnosis to a platform for all of STXBP1 We’re not just building a foundation. We’re building momentum. Thanks to Sam Cronin and Google for Startups Israel for hosting such an inspiring event—and to the VCs, founders, and changemakers who continue pushing the PharmaTech space forward. #STXBP1 #TechBio #RareDiseaseInnovation #RafasMoonshot #PharmaTech #DrugDiscovery #StartupThinking #NeuroScience

🧠 The brain has 86 billion neurons. 🔗 Trillions of connections. 🧬 One tiny protein—STXBP1—can throw it all off. That’s the reality of STXBP1 disorders—where a single gene impacts how neurons communicate, disrupting everything from movement to cognition. Understanding how the brain works at the molecular level is key to finding a treatment. Here’s what the science tells us: ⚡ STXBP1 is a critical player in brain communication. Neurons send signals using chemical messengers called neurotransmitters. STXBP1 helps release these messengers at just the right time. When the gene is mutated, signals don’t flow properly—and brain function is compromised. 🔬 One mutation, widespread effects. STXBP1 disorders can cause epilepsy, developmental delays, and movement disorders—not because the brain lacks neurons, but because those neurons can’t “talk” to each other as they should. 🔄 The brain can rewire itself. Through a process called neuroplasticity, the brain can form new pathways—especially in early childhood. That’s why early diagnosis and intervention are so important. The sooner we understand what's happening, the sooner we can support the brain’s ability to adapt. 🤖 AI is revolutionizing brain research. At Rafa’s Moonshot, we’re using tools like BioNeMo, AlphaFold, and molecular simulations to study how STXBP1 mutations affect neuron function—and to screen for drugs that could restore it. It’s science at the speed of urgency. Every discovery about the brain brings us closer to understanding how STXBP1 works—and how to fix it. Because when we understand the science, we can build the solutions. ✨ #STXBP1 #Neuroplasticity #BrainScience #AIinMedicine #RareDiseaseResearch #RafasMoonshot

AI is collapsing drug timelines—from 10+ years to just a few. At Rafa’s Moonshot, we’re not just watching it happen. We’re living it. 🚀 When baby Raphael was diagnosed with STXBP1, his parents—Sagi and Ella—were told the truth: ❌ No treatment. ❌ No roadmap. ⏳ No time to wait. So they searched for the shortest path to a solution. And they turned to AI. Everything changed. Today, AI can: 🧬 Simulate mutated proteins 💊 Screen millions of drug candidates in days 🔮 Predict efficacy before a single lab test What once took a decade now takes a fraction of the time. For rare diseases like STXBP1, that’s not just innovation. That’s survival. 💔 We’re using tools like AlphaFold, BioNeMo, cheminformatics, and patient-derived models to push the limits of what’s possible. And it’s working. 🌍 The world is changing—fast. #AIinMedicine #RareDiseaseResearch #STXBP1 #DrugDiscovery

🎤 Telling the story of two years of science, heartbreak, and breakthroughs—in just five minutes—is no small task. But yesterday, Sagi Gidali did exactly that on stage at Tel Aviv Sparks Innovation. In his first-ever Ignite talk, he told Rafa’s story—from diagnosis to drug development—sharing how a rare genetic diagnosis led two parents to launch a research initiative that’s accelerating the path to treatment for STXBP1 disorders. Ignite is a unique storytelling format: ⏱️ 5 minutes 📽️ 10 slides ⏳ 30 seconds per slide No stopping. No going back. Huge thanks to Or Daniel and the Ignite Learning community for making space for stories like ours—and to everyone who showed up, asked questions, and reminded us why this work matters. Thank you to everyone who attended - Sagi left the stage inspired, hopeful, and more motivated than ever. 💫 #STXBP1 #RareDiseaseResearch #IgniteTalk #Innovation #RafasMoonshot #ParentingAndScience #StartupMindset #HopeForFamilies

Did you know that every thought, movement, and memory begins with a neuron? 🎇 These small but powerful brain cells act as messengers—passing electrical and chemical signals that allow us to breathe, speak, learn, and connect. In STXBP1 disorders, this communication system breaks down. Neurons rely on the STXBP1 protein to release neurotransmitters—the chemical messengers that make signaling possible. When STXBP1 doesn’t function properly, those signals misfire or stop altogether, leading to serious challenges in cognition, movement, and development. At Rafa’s Moonshot, our research is focused on restoring proper neuronal function. That means improving neurotransmitter release, stabilizing the STXBP1 protein, and enhancing how neurons behave—at the molecular level. Here’s how we’re approaching it: 🔬 AI-driven drug discovery – Using platforms like BioNeMo and cheminformatics tools, we screen thousands of compounds to find those that may help stabilize STXBP1 function. 🧪 Molecular modeling and simulations – We study how specific STXBP1 mutations affect neuron behavior and use those insights to predict which treatments could restore normal activity. 🐛 Preclinical testing in model organisms – In C. elegans models with STXBP1 mutations, we test candidate drugs to observe their effects on movement and neural function. Neurons are at the center of what’s disrupted in STXBP1—and they’re also the key to unlocking treatments. By combining AI, lab science, and living models, we’re building the clearest picture yet of what’s going wrong—and what might make it right. ✨ #STXBP1 #Neurons #AIinDrugDiscovery #Neuroscience #RareDiseaseResearch #RafasMoonshot