In this presentation, Timothy Miller, Co‑Founder and CEO of Forge Biologics, outlines the company’s unique hybrid CDMO (contract development and manufacturing organization) model that combines a commercial manufacturing platform with an internal clinical pipeline. Founded in 2020 and based in Columbus, Ohio, Forge has raised over $330 million across Series A‑C rounds and operates a 200,000 sq ft facility with 20 GMP suites, including the industry’s largest 5,000‑liter bioreactor and 1,000‑liter runs using a proprietary, clonally‑derived HEK293 “Ignition” cell line. Miller details Forge’s service portfolio—plasmid DNA (Phase 1/2 now, full GMP launching soon), AAV vector production, fill‑finish, process and analytical development, and cell banking—highlighting the end‑to‑end capability from research‑grade material to commercial fill. He emphasizes the company’s culture‑focused growth strategy, citing a 260‑plus employee workforce, a high‑volume internship program (Ignite), the She Forges women‑leadership initiative, and community engagement. The talk also showcases early clinical data from the RESCUE trial (bone‑marrow transplant followed by systemic AAV10‑GALC for Krabbe disease), reporting safety, increased GALC enzyme activity, and normalized motor development in treated children. Miller concludes with Forge’s vision to become one of the world’s largest dedicated gene‑therapy manufacturers, serving a diverse client base ranging from early‑stage startups to large public companies, and outlines upcoming expansions in capacity, plasmid GMP offerings, and novel technologies such as an ad‑helper plasmid.
Chapters
Good morning, everyone. Thanks for coming into the talk today. I'm going to tell you a little bit about Forge Biologics. It is really bright up here. Wow. So for those that are out there, I hope I can see you. But so I'll tell you a little bit about Forge today. So we are manufacturing gene therapies for life, and we're based out of Columbus, Ohio. We have a slightly, not quite what I think of as novel, but one of the more unique business models in this industry. And really, we're a CDMO of the soul. So we have actually a clinical pipeline. And, you know, tell you a little bit about that today, too. So we were founded in 2020. We have an amazing leadership team that I think a lot of you have actually met with. And but the combined total, we added all of our years up in the industry, and it's about 165 years worth of experience of hands-on gene therapy. So a lot of us have a strong therapeutic background. And have had to build out manufacturing as part of our careers. And so, you know, again, this had to live through a lot of pain problems with a lot of CDMOs. And so I'm able to help meet that need. And as we started to set this company up in 2020, we wanted to do something a little bit different. And so we set this up as the hybrid model, so the hub and spoke model, right? So we actually have a small clinical stage pipeline. 2 years has been a really crazy time for a lot of us, right? So when I left my last job, I didn't necessarily anticipate that I was going to have to raise a Series A fund in the middle of a pandemic. So it was very interesting coming out of JPMorgan 2020. But we've moved from an emerging to really an established CDMO in about 2 years, raised over $330 million in A, B, and C rounds with a lot of top-tier investors. Right now, the facility that we have is 200,000 square feet. And, you know, we have the capacity with 20 GMP suites. You'll see some more pictures of those, including the largest in the industry of 5,000-liter bioreactors. Bless you. We formed, you know, an independent scientific advisory body, really, to help us with some of our thought process in manufacturing. A lot of gene therapy leaders advanced our therapeutic pipeline into the clinic with the RESCUE trial, and we'll talk a little bit about data in that towards the end. Very typical things that you would expect out of a, I think, a good CDMO. We have a drug master file on file and available for clients to help accelerate. And more recently, we just announced our plasmid DNA offerings. So again, thinking about the three main quality attributes of research, the equivalent of Phase 1/2, Phase appropriate, and full GMP. We have Phase 1/2 compatible available now and launching full GMP next year. So we're trying to do this to help make sure that a lot of clients are able to meet that timeline that many of us try to accelerate towards. So being able to do plasmid on the front end and filling on the back end We can do it all. And the team is currently— this slide is just a little bit out of date— it's about 260+ employees at this point and looking to scale to almost 300 by the early parts of next year. So huge growth, and we need that to run a big CDMO. Trying to raise the bar on a lot of expectations. So for many of us that have lived through a lot of challenges, if this was 2015 and I was standing up here saying we have a 50-liter bioreactor scale at a suspension would have been leaders in the industry. But time's moved on and you got to think bigger, think bigger scale, think of better productivity. So, you know, we have our own HEK293 cell line. It's fully commercializable. Having lived through the kind of pain points of coming into a facility, using their cell line to understand that you actually couldn't take that all the way through, we went back, we rederived a cell line from the beginning, full traceability. So you can use the Ignition cell line for research grade very, very early all the way up through. So completed our master cell banking, just like any, again, a lot of check the boxes type of things. But we're also currently running at 1,000-liter scale. So we have run 1,000-liter GMPs using an HEK293 cell line. So for those of you that need a lot more AAV in particular, you can come and talk to us about that. Launched our plasmid offerings, have tons of analytical developments, helping some clients work on tech transferring in to do potency assays. And again, 20 GMP suites. You're going to hear me say it a couple more times, you know, but part of what makes Forge important is a lot of our culture. And we've had a lot of great interest into the company. We have had over 6,000 applications into the company just in 2022 alone, having a great acceptance rate. We've had almost 30% of our new hires relocating to Columbus. So Columbus and Ohio seems to be in the news a lot. With Intel, Amgen, lots of, I think, business pulling into Central Ohio and really trying to make this a hub of both manufacturing and industry overall. And we also, we love interns. So we've had a huge— our Ignite program is truly to try to build up that workforce development. Culture for us is really one of the biggest driving things that I think makes Forge special. We have our She Forges group, which is trying to help women really try to break through that 7% glass ceiling. Over 50% of the company is women. Our leadership team is half women. It's really things that we take a lot of pride in. We try to support a lot of the community. My favorite, one of my favorite pictures is the Forge glove box. Okay, some people really got creative on that one. And we are definitely Star Wars fans in the, in the team. So may the Forge be with you is one of our favorite things. And you might have seen some of the things around our mountain climbs that we try to do for foundation awareness. And an important part of, again, building team, building culture for the company. So what is next? What's coming up? Well, in 2023, we're going to be one of the largest producers, dedicated producers of manufacturing in the world. And these are all photos taken from our facility. Facility's up and running. There's actually bioreactors running cells. This is a piece of our what we call our Stage 2 build. And just looking down one part of the corridor, if you were on the other side of the building, you'd see the 5,000 liters already in suite. And getting ready to be scaled up. So what does that mean? So we've spoken with many of you in the audience, currently serving more than 25 clients, platforms applicable across multiple indications. We get asked a lot by investors, we get asked from potential clients that says, well, tell us about your client segmentation. Are you working with small groups, Series A, or you're working with big groups in the public domain? We're working with basically a lot of— all of them. It's interesting again, because coming out of a tough market, fundraising has been a challenge, I think, for some. But I can stand up here and say that AAV manufacturing is alive and well. And a lot of, I think, the boards that were going to do a lot of build-out have said, we're not going to build out, go find a qualified CDMO. And a lot of that has come to Forge. So, you know, and what are they working on? Well, we do both research-grade and we do GMP production. So you can come in and get very early started. We have a research production cell line that will help jump your process to get you into GMP a little bit faster. And we've made a lot of the different types of serotypes, and importantly, we're making some of the novel ones. So our team has experience on process development and analytical development on how do you think about, well, if we're going to scale up and purify some of these novel capsids, you know, how easy is that? What's some of the challenges? So again, we've made a lot of these capsids at many different scales. So we have a strong molecular development team, and we're not here to just make stuff. We're actually here to try to improve things. And so one of the products that we actually had was our ad helper. Dr. Dismuke, our chief technical officer, one of the things that he designed, but it's a way to make an ad helper plasmid that's a little smaller, it's a little safer, and it doesn't look like it's packaging at all any of the adenoviral helper strands, which is been some concern for some of the other plasmids that are out there. And again, mentioned our Ignition cells. So again, it's a 293 suspension cell line that is up and full running. Interesting, about 90% of our clients— we get asked this question a lot— about 90% of our clients come in and actually use this cell line. And it's important because the cell line was actually clonally derived and selected to be very— it produces about 40% fulls at very early stage. So things you put in, the better you put in, the better you get out. And as we think about that as a platform, more and more clients are using that to help drive this, this platform. And again, we have research, process development, and toxicology grade. You can do that up to 500 liters. And our research grade GMP pathway and full GMP plasmids coming in 2023. So that's, I think, a great way to segue into, you know, some of the other things that we're working on, which is Um, filling. So everybody needs to have their stuff filled. So you can come to Forge and you can do plasma production on one end, and you can do a full commercial fill on the other side. So aseptic filling with automation, lots of container closure studies. Come in and take a look. So talked for a moment about the fact that we're a hybrid company. So we do have a small therapeutic pipeline, and so we announced some of our exciting data, I think yesterday morning, by press release. Because it's being presented today at the European Society for Gene and Cell Therapy. So we all had to make a choice, come here and drink coffee, go to Edinburgh and drink scotch. Glad you all came here. But we announced yesterday or this morning that some really, really great data, which I'm going to show you. So to refresh what this is, why it's a first in human, it is a bone marrow transplant. And then after the bone marrow transplant, you're actually getting a systemic AAV10 delivered. And it's delivering the GALC gene for patients with Krabbe disease. So, so far we've treated a few patients, things look great, it's very, very safe. We're looking for enzyme activity. So we've seen a bunch of increased GALC enzyme activity in plasma and CSF. But more importantly, what we've actually seen is normal white matter myelination and a normalization of motor development in the two children treated. So having done gene therapy for almost 20 years now and been involved in a lot of clinical trials, I'm really happy to see this graph. So looking here, again, two subjects. You'll note the gray cone, that's an unaffected child's development. The yellow shaded cone is the one that's actually what an affected child with Krabbe disease looks like. The green arrows are where they get their bone marrow transplant. The blue arrows are where they got the AAV. And what you can see in particular is that, you know, I think we can say we've stabilized the motor development of subject 03, but for subject 01 that was treated a little bit earlier, we've essentially normalized their motor development. So don't see a lot of data like this in this space. Pretty cool. So stay tuned for more as we're hoping to close the cohort soon. And so with that, you know, FORGE is really trying to be a development engine and help all of you as well as try to serve our own patients as well. So we have lots of offerings. We can do cell processing, cell banking, process development. Everybody needs process development. It takes time. So come in and learn about process development. We can do plasmid manufacturing and of course your AAV production. So with that, I'll wrap and say thank you. I think I have 3 minutes in case there are any questions. Finished early. How about that? All right. Thank you.
