Mary Parker:
I'm Mary Parker and welcome to this episode of Eureka's Sounds of Science. Today we are joined by NanoPalm CEO and founder Ali AlHasan. NanoPalm is dedicated to studying new in-vivo therapies for genetic diseases and is one of the first cohort of startup companies in Charles River's Incubator program. They are also the first company to organize a clinical trial for gene therapy in the history of Saudi Arabia. We are also joined by Charles River executive director, Ramin Baghirzade to tell us more about the Incubator program and his work with NanoPalm. Welcome, Ali and Ramin.
Ali Alhasan:
Thank you. I'm really glad to be here.
Mary Parker:
I'm super excited to hear more about the industry in Saudi Arabia and about your research in particular, so let's dive right in. Can we start with your background, Ali? What is your inspiration for starting NanoPalm?
Ali Alhasan:
Absolutely. Absolutely. So me and my co-founder, Samar Alsudir, we share the same pain. We both have relatives suffering from genetic diseases, ranging from sickle cell disease to muscular disabilities. We were colleagues, I was her mentor, she was my postdoc at the time, but then as we grow and as we develop more expertise, we sat down and said we think we can help those in need of curative treatment. And that's how we launched NanoPalm.
Mary Parker:
And I understand that the college university system in Saudi Arabia is truly excellent, so I assume that you had worked together at university before starting the company?
Ali Alhasan:
Yeah. We were working with a prominent R&D institute called King Abdulaziz City for Science and Technology, and that's where our breakthrough was developed originally. And then as part of the Saudi Vision 2030, that we startups in the biotech ecosystem, we were among the first spinoffs from this prominent R&D institute, KACST.
Mary Parker:
And Ramin, what is your career background? How did you get involved with Charles River's Incubator program?
Ramin Baghirzade:
I'm a global head for commercial gene therapy in Charles River, and as part of my role, we're always on the lookout for innovative companies, and how we can help and support them with their next major milestones. We saw a gap in the market and we saw a lot of great science being lost in translation, and in a time where investment in early development companies is limited, incubator programs like ourselves can play a crucial role in supporting this early stage ecosystem.
Mary Parker:
So Ramin, what drew Charles River to NanoPalm specifically?
Ramin Baghirzade:
So as part of the Incubator Program, we've been receiving applications from a lot of great, promising companies. I think what made NanoPalm stand out was its cutting edge science and was its commitment to support unmet medical needs, kind of a caliber of its scientific and management team, as well the vision and the tangible project plan to reach some of those upcoming milestones.
Mary Parker:
I mean, that makes sense considering, I understand that you have started this clinical trial, it seems like they were pretty well on their way before Charles River came along. So just out of curiosity, what kind of help can Charles River offer to companies that are in this Incubator Program?
Ramin Baghirzade:
So as part of our Concept to Cure Portfolio, so we work very closely with a cross functional team of scientific regulatory and manufacturing experts in drug discovery, safety assessments, research models, clinical and commercial manufacturing and microbial solutions. Essentially, with this array of experts, which are available, we are able, essentially, to tailor the support that each Incubator company needs to reach their next major milestone.
Mary Parker:
That's perfect. So Ali, can you tell us more about your company's current research? What are advanced lipid nano particles, I read that on your website, and why are they valuable for gene therapies?
Ali Alhasan:
Absolutely. Absolutely. Let's start with genetic diseases, the only way, it seems, to cure genetic diseases is to use medicines, really complex medicines like mRNA and CRISPR, and those medicines requires some help from nanotechnology in particular. Because it seems the only way to unleash the potential of those complex cures, mRNA and CRISPR, is the delivery. So our research, primarily in chemistry, we noticed that everyone doing the chemistry in what's called a mobile phase, that's how they make lipid nano particles. So we decided to go the opposite way, we make lipid nano particles as pattern on solid state or substrate. So basically printing them out using advanced nanotechnology called nanolithography. So most of our research was to harness the benefits of doing lipids on substrate, rather than a mobile phase, and then we collaborated with AI and deep machine learning to understand the properties, the biology kind of properties, physical properties and chemical properties of those pattern lipids and compare them to those mobile phase. And we discovered really interesting features and attributes that can change the way we treat patients with genetic diseases.
Mary Parker:
So from what I understand, the lipid nano particles are the delivery system for the gene therapy, and you used AI to figure out how to manufacture them more efficiently?
Ali Alhasan:
Exactly. Correct. And lipids are a great carrier, they just need to, so we solve certain issues like the current lipid carriers are often designed with unstable structures, composed of toxic components, and they are blind, they cannot target specific organ or specific cells, hence the AI can actually accelerate how we make them more stable, how we accelerate making them more safe and more targeted.
Mary Parker:
Would you say that the lipid nano particle is a better system of delivery than the typical viral vectors or is it just a different way of delivering it?
Ali Alhasan:
So the most common delivery is, as you said, the viral vector. That's the most common we use, the delivery method. And it's good to deliver plasma DNA for instance, but when it comes to more complex medicines like short RNA fragment or mRNA as is, viral will fail, not to mention their limited capacity and some safety concerns. So I think the lipids are more versatile, we can load them with really short RNA fragments, really long RNA fragments, and we can go back and forth between DNA or RNA, whatever we need, and also proteins. So viruses cannot deliver proteins. Viruses cannot deliver RNA at the moment.
Mary Parker:
That's really cool, I didn't know that. So Ramin, how do you see the role of companies like NanoPalm in the current gene therapy industry?
Ramin Baghirzade:
Well, it's important to highlight that the vast majority of salient gene therapy companies are small biotech, so we see companies like NanoPalm really as an innovation engine within the industry. Typically, it's kind of a high-risk, high-reward mode, so that also means that sends in why a lot of great science, as was discussed earlier, is unfortunately lost in translation. So if we don't have, let's say true innovators like NanoPalm, then they wouldn't be any kind of major breakthroughs within the industry, either.
Mary Parker:
Ali, can you tell me about NanoPalm's current clinical trial? What is a BioRobot? I also found that on your website, never heard that term before. And how could it potentially be used to treat sickle cell anemia?
Ali Alhasan:
Absolutely. Absolutely. So BioRobot, it's just our way of saying to everyone that now we have injectable gene therapies, as opposed to ex-vivo gene therapies. More in the current approved gene therapies in the market, I think there are nine or 10 of them, they have to take cells, collect cells from patient, ship them to a lab, where in Petri dish most of the therapy is happening, outside of the body. BioRobot was our way to explain now we can do things in-vivo, injectable gene therapies. Now we have three cures in our pipeline, the first one is, I'm glad to say it's in a clinical trial, an injectable gene therapy for sickle cell disease.
What's really unique here, what we observed so far is that patient experience is improved significant from couple months to one year therapy program when it comes to ex-vivo, to a half-day single visit to out patient clinic. There is no admission involved. Once these patient get our injections, the rest of gene therapy will happen inside the body, basically used by robot, will bind to specific cells in the bone marrow, and snip them mutation and correct the genetic defect. So I'm happy to say this is a still ongoing phase one clinical trial here in Saudi Arabia. It seems that the market of clinical trial is growing, we're happy to be among the first, we think we will expand and help other companies as well to perform clinical trials here.
The second one in the pipeline is actually still early, it's again, it's a disease called retinitis pigmentosa. Basically, patients are born with a genetic defect that causes blindness during the first 20 years of their lifetime. So those, again, are injectable and they're supposed to preserve the vision by correcting the mutation. Now, the mutation is actually because of genetic diversity we have, a mutation that's unique to Saudi population, another mutation unique to Arab patients, and so on, which is different from the Western world. So our medicine, this particular medicine is really tailored to a population that is prevalent in our region.
Mary Parker:
You know, I hadn't even considered that. That makes perfect sense, I know that different regions have different genetic anomalies and different patient needs.
Ali Alhasan:
Correct. And you remind me of the specific approach in therapy out there in the market, it's not working here, because really few patients are benefiting from a gene therapy in the market, but the majority of the patients which are 4,000, they are not benefiting from that because of different genetic background. I just want to, the third one we just discovered is for the cure to help those born with high cholesterol, inherited high cholesterol, called familial hypercholesterolemia. This is still early in the discovery, so Charles River can help us in so many ways.
Mary Parker:
How has Charles River helped you so far?
Ali Alhasan:
We joined Charles River 1st of May, and we've been doing some assessment of both levels, technical and business. And because with expertise from Charles River, they provided us with great mentors, one for the scientific area and one for the business aspect. And both mentors are really tremendous in figuring out where we need to grow. And so the past period was really back and forth assessment on how to put the most feasible milestones and approach and strategy as well, to advance in both aspects, technical and business.
Mary Parker:
Ramin, it must feel nice to hear that from one of the companies that we've been helping.
Ramin Baghirzade:
It sure does, it sure does. And NanoPalm has been a great partner as well. And we well know that it takes two to tango and it's quite critical as well to, there is obviously a lot of great science, but it also requires a certain type of mindset to kind of get you to the next level. And certain level of also there's a cultural feat in terms of being open to feedback and being open to coaching and mentorship, I think that makes a huge difference when it comes to success or a failure.
Mary Parker:
So I'd like to kind of put this in context, so Ali, can you describe the current state of the biotech industry in Saudi Arabia?
Ali Alhasan:
Absolutely. Absolutely. That's a great topic to cover because on one hand, we don't have a big pharma, a Saudi big pharma, right? On the other hand, we have a great health system. One of the best in the region, and globally as well. Our healthcare is of high standards. So it was really amazing that we don't have big pharma, but great healthcare. So great healthcare system is really feeding us with two important things for ecosystem, talent and infrastructure. When it comes to infrastructures, we have great clinical trials science, that's really instrumental for a biotech like us. Although they are only couple of them, but they will do the job, especially that they are operating at high standards.
In addition to clinical trial side, turns out we have great manufacturing facilities as well. Especially around the R&D and I&D stages, for example, King Abdulaziz City for Science and Technology. This is where we do most of our manufacturing practice. Speaking of that, we are actually a spinoff of that great institute, King Abdulaziz City for Science and Technology. And then once the manufacturing happens, we take then to the backyard, that's where the hospital is, that's where the clinical trial site in the National Guard Hospital. So infrastructure I would say, check. And maybe we could do more.
We have great clinicians. Great physicians. Great scientists, lab technicians. But we don't have the business mind of it yet. And probably this is the problem, is why we don't big pharma, but we have big healthcare system, business mind. So the last three years, the whole concept of startups, established. Now we need startups, now we need spinoffs. And it happened that we were one of the first to spinoff in Saudi Arabia. We think more startups will be generated and produced, and hopefully one of them will grow to be a big pharma, and then and only then we can say the biotech ecosystem is great. At the moment, it's still lacking.
Mary Parker:
It must be pretty exciting, though, to be kind of on the front lines of this growing industry in your country.
Ali Alhasan:
Yeah, you're right. Being a pilot company is like you're building the bridge while you are walking it as well. Really fun. And we enjoy it, it's very enjoyable experience.
Mary Parker:
So going forward, what are you hoping to accomplish at NanoPalm in the next five to 10 years?
Ali Alhasan:
We hope to, we don't want to put a value on curing a patient, use of breakthrough cures will helping huge number of patients. We have 300 million patients around the globe suffering from rare genetic diseases and we are turning a blind eye to, we cannot help them. So bringing even one cure to a market, yes, it's a multi-billion dollar business opportunity, but we think together we can make a multi-billion dollar market where we can do well while doing good at the same time. We hope we can cure the more of genetic diseases, of course one disease at a time, but we hope we become one of the next major global deep tech global biotech companies, not much tech bio companies, Lots of biotech, but not much tech volume, so we hope to be one of the next major ones.
Mary Parker:
Well I wish you all the luck in the world and I am really looking forward to seeing the results of all three of your clinical trials.
Ali Alhasan:
Thank you.
Mary Parker:
And thank you both for coming on the podcast and sharing this story with me, it's been fascinating.
Ali Alhasan:
Thank you, Ramin. Thank you, Mary.
Ramin Baghirzade:
Many thanks.