From Petri Dish to Product: How Maya Gosztyla is Rewiring CNS Drug Discovery
- Dominic Borkelmans
- Aug 11
- 8 min read
Updated: 5 days ago
Just under a year ago, Dr. Maya Gosztyla left the familiar surroundings of her university lab at UCSD to co-found a biotech start-up. She had just finished her PhD in biomedical sciences when a recruiter connected her with the team that would become her cofounders. Now, a year later, she has grown to thrive in the chaotic life of a biotech entrepreneur. She owns the COO role at the budding firm, where her passion for the underlying science fuels the startup’s bold ambitions.
BrainStorm Therapeutics is a small San Diego start-up on a mission to transform the notoriously slow and failure-prone process of drug discovery for central nervous system disorders. In an industry where 93% of attempts fail, any innovation that will reduce the failure rate is welcomed with open arms. BrainStorm is betting on a powerful combination: stem-cell-derived brain organoids and novel AI models. This approach aims to dramatically derisk the drug discovery process and provide pharmaceutical companies with viable treatment candidates for debilitating neurodegenerative diseases like Parkinson’s.
Why Maya swapped academics for entrepreneurship
Maya describes herself as slightly introverted, but when she talks about her science, there’s an energy that’s hard to miss. She hadn’t planned on joining a startup, until a recruiter from a previous job application introduced her to BrainStorm’s founding team. The match was clear: her PhD research on brain organoids aligned perfectly with the company’s goals, and her drive fit seamlessly with the team’s momentum.
Pursuing translational research had been Maya’s goal since before graduate school, an ambition sparked during her time at NCATS, the translational science center of the NIH. There, Maya was exposed to the drug discovery process and was, for the first time, able to see how, as a scientist, you could have a direct impact on people’s lives. Something many academics may never achieve in their lifetime.
She especially gravitated toward rare diseases, a focus driven by a pragmatic scientific mindset. With so many rare diseases untreated and so few researchers studying them, the chance of making a real impact in the field felt more tangible. And so, she bought into BrainStorm’s mission: to tackle the 90%-plus failure rate of clinical trials in neurological diseases using brain organoids as a new drug discovery platform.
BrainStorm’s sesame-seed organoids derisking drug development
BrainStorm positions itself as a feeder firm for larger CNS pharmaceutical players. They take human stem cells from patients and use them to create brain organoids. “They’re little 3D balls of brain cells, about the size of a sesame seed, that can act like your brain,” says Maya. “They show neural network activity the same way your brain does. They have the same kind of biochemical pathways. They really function like tiny, simple brains.”
BrainStorm grows the organoids from stem cells of patients with CNS disorders, from Parkinson’s to rare epilepsies. They use the custom ‘mini-brains’ to screen potentially valuable therapeutic molecules for the given disease. The goal is to find new molecules that reverse the disease phenotype and restore the organoid to a healthy state. For Parkinson’s, that means looking for molecules that will reverse the loss of dopamine neurons. For epilepsy, those fixing abnormal electrical activity.
Beyond the organoids, BrainStorm’s core technology stack includes a cutting-edge AI component. They have partnered with Nvidia to create what they describe as a biology-first approach to AI, where they apply an emerging class of AI models, trained on up to 100 million human cells. Rather than using these models to design molecules, a by now established approach, BrainStorm uses the AI to predict the most promising therapeutic targets for a given disease. After identifying the most promising targets, they design the appropriate molecules accordingly. The logic: “It doesn’t matter how good your molecule is if the target isn’t the correct one for your disease.”
Parkinson’s has emerged as an ideal candidate for the platform, given its well-understood etiology. “If you increase the number of dopamine neurons, you can cure the disease.” explains Maya. But while the logic is straightforward, only with the recent technological advances are researchers beginning to close the gap between concept and clinical reality. Beyond Parkinson’s, Maya and her team are looking for additional observable and reversible phenotypic disorders. This led them to explore rare epilepsies, where their organoids successfully replicate the same abnormal firing patterns seen in patients.
They’ve already made promising progress on Parkinson’s, using their AI model to identify a cluster of dysregulated genes present both in BrainStorm’s organoids and in the brains of people with Parkinson’s. These clusters have become top therapeutic target candidates. With fundraising underway, the team’s next priority is to experimentally validate the model’s predictions and determine whether targeting the identified gene clusters can actually yield effective drug candidates.
In the longer term, Maya and BrainStorm aim to tackle more complex diseases. Alzheimer’s being the white whale. But while those diseases come with even more financially appealing incentives, they are also much harder to decipher. There is a reason why, after decades of attempts, we have yet to discover any meaningful way to treat Alzheimer's. For now, BrainStorm is moving cautiously up the complexity scale of CNS disorders.
How Maya transitioned from the bench to budgeting
For Maya, leaving academia to start a biotech venture didn’t entirely feel like stepping into unknown territory. “At a startup, you still have some of that chaotic energy you sometimes find in academia,” she says. “Everyone is rushing, scrambling to get funding. I think that’s still a little bit there, which I actually kind of like. It makes the transition out of academia easier.”
Still, her lifestyle and day-to-day activities have changed significantly. She no longer spends entire days immersed in science, like she did in graduate school. Now, nearly half her time is spent on business-oriented tasks. She is pitching to investors, preparing presentations, and managing day-to-day budgeting. The learning curve can be steep, with entirely new material to master, and the stakes are high: the science stops the moment the funding does.
“At a startup, you still have some of that chaotic energy you sometimes find in academia”
One of the biggest surprises for Maya has been the sheer amount of networking involved. While most grad students do some networking, for a co-founder, it becomes part of the daily job. At nearly every event, Maya is there representing BrainStorm, always on the lookout for the next investor. According to Maya, networking isn’t something you’re born with. “I think it’s a muscle you have to build up. It’s something you get better at the more you do it.”
Some academic habits translate surprisingly well to her new role. Maya credits much of her daily efficiency to the time management skills honed during graduate school. “You can easily spend hours on work that doesn’t move the business forward,” she says. Learning how to prioritise, both short- and long-term, is something grad school teaches by necessity. For Maya, it’s become one of her most valuable skills.
The current state and the promise of organoids
Over the past few years, great progress has been made in the field of organoids. However, according to Maya, several core challenges remain for BrainStorm and the broader industry. One major limitation is that there’s still no reliable way to estimate drug dosage using organoids alone. As a result, BrainStorm relies on animal models to determine what dose of a new drug can safely be given to humans.
Furthermore, while working with real human cells is a major step forward, current organoids still fall quite some lengths short of capturing the full complexity of the brain. For example, BrainStorm’s organoids lack microglia, the brain’s resident immune cells, and don’t include a blood-brain barrier. These features could be added, but doing so would make the process significantly more expensive and inefficient, precisely the problems BrainStorm is trying to solve.
Even so, innovation in the field shows no signs of slowing down, says Maya. In the near future, BrainStorm may begin working with assembloids. An assembloid combines two or more different types of organoids into a single, integrated system. This approach makes it feasible to study complex, multi-system disorders of the brain. Take Alzheimer’s, for example, a disease that affects nearly every part of the brain. Targeting just the hippocampus, the brain’s memory center, is unlikely to be enough.
If these lingering challenges are overcome, and innovation continues in both AI and organoid complexity, Maya envisions a transformed drug discovery landscape. For one, drugs entering the clinic could have a significantly lower failure rate. No longer would 93% of efforts, and the funding behind them, be lost to failure. “If you’re starting from a human system, you're not going to see as much dropout after the initial screen. It’s de-risked from the beginning.”
More importantly, it means better drugs for brain diseases that currently have no effective treatments. And a reduction in the cost of those drugs. After all, most of what patients pay for isn’t the cost of one successful drug, it’s the cost of all the others that failed. Maya knows well that “if we reduce late-stage failures, we won’t have to pay as much for the ones that succeed.”
Maya’s advice for the next cohort of PhD neurofounders
For Maya, her first year as a biotech founder has been both exciting and deeply rewarding. Fortunately, it hasn’t just been fundraising and finances. In her day-to-day work, she still has the chance to engage directly with patients. Right now, BrainStorm is working with the CURE5 Foundation, founded by families of children living with CDKL5 deficiency disorder.
As part of this collaboration, they’re using the children’s stem cells to create personalized brain organoids for each patient. They then screen FDA-approved drugs to identify any that might be prescribed off-label to help reduce seizure activity. This isn’t something that will be directly profitable for BrainStorm; it’s simple drug repurposing. But it helps validate their discovery platform and gives Maya and her team the chance to make a tangible, immediate impact on these children's lives.
It’s no surprise, then, that many PhD students dream of making a similar leap into the startup world. To those with that ambition, Maya recommends getting exposed to the industry as early as possible. Internships, volunteering, or any hands-on experience in entrepreneurship can help build foundational skills, and clarify whether the lifestyle is the right fit.
Most importantly, she urges students to get out there and start conversations. In her experience, early-stage founders are eager to share their journeys, especially when there’s a shared background. And to the brilliant minds in labs who worry about making the leap to startup life, Maya offers a clear reminder: “You don’t need an MBA or anything like that.”
What you do need is curiosity, conviction, and a willingness to learn fast and ask often. Whether it’s pitching investors, growing organoids, or building therapeutic pipelines from scratch, Maya’s journey shows that the leap from bench to biotech isn’t just possible, it’s one of the most direct ways a scientist can impact lives through the power of their work.
About the Founder
Maya Gosztyla is the Co-Founder and COO of BrainStorm Therapeutics. She completed her PhD in Biomedical Sciences at UC San Diego and has been featured in Nature for her reflections on transitioning from academia to biotech, time management in research, and translational science.
Read more of her perspective in Nature Careers:
About the Firm
BrainStorm Therapeutics is an early-stage biotech company based in San Diego, California. The company focuses on accelerating CNS drug discovery using patient-derived brain organoids and biology-first AI models. By targeting diseases with clear, observable phenotypes, like Parkinson’s and rare epilepsies, BrainStorm aims to reduce the clinical trial failure rate and deliver more effective, affordable treatments.