Latest / The BioHub - by Avetix / Episode 209 - Pam Stetkiewicz Lyora Therapeutics

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Episode 209 - Pam Stetkiewicz Lyora Therapeutics · The BioHub - by Avetix

• 0:06
  Welcome back to another episode of the Biohub podcast.
• 0:09
  Today, I'm thrilled to be joined by Pam Stekovich, the CEO of
• 0:14
  Laura Therapeutics. In today's episode, we're going
• 0:17
  to talk a little bit more about Pam's career and the recent
• 0:20
  announcement of the formation of Laura Therapeutics out of
• 0:24
  stealth mode. So, Pam, thank you so much for
• 0:26
  coming on. Thank you for having me, George,
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  I'm delighted to be here. Yeah, looking forward to it.
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  I mean, firstly, congratulations on the recent announcements on
• 0:35
  the formation of LIOR and your appointment as CEO.
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  To start with, can you walk us through your career journey,
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  Pam? And then ultimately, what led
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  you to joining LIOR? Yeah, I'd be happy to do that,
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  George. Yeah, we're thrilled to publicly
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  announce the launch of Lior Therapeutics and I'm glad to
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  walk you through a bit more about my career journey and how
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  I got here. I'm a scientist by training.
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  I, I got my PhD from Johns Hopkins and you know, I started
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  out my career as a scientist. I was a post doc at Genetics
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  Institute, which was then bought by Wyeth and Pfizer.
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  But I think it was this, this was really where my career
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  started in industry. And it was probably the first
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  time actually that I and maybe the only time that I had an
• 1:32
  epiphany in my career. I was a scientist and I got
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  invited to join a cross functional development team.
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  And you know, a scientist perspective is obviously a
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  critical element of moving any drug forward.
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  But sometimes being in the lab can be a bit isolating where you
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  don't see the whole picture of drug development.
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  So I got to join this cross functional team and I was like,
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  wow, there is so much to getting a great science experiment in
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  the lab to a drug in a person. And so it was at this point that
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  I really wanted to get a more holistic view of drug
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  development. And I made a a decision after
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  being through six years of a PhD program, three years of a post
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  doc, to jump into project management and because I wanted
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  to understand all the elements of drug development and then
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  decide what I really wanted to do.
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  So this was like a big change in in my career trajectory.
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  And I just want to note that I probably could have never
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  succeeded if I didn't have mentors.
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  I had a great mentor, Paul Schendel, who really taught me
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  everything about project management.
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  Then after that, working there for a couple years, I got an
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  opportunity to come over to Novartis.
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  Novartis was just starting up in Cambridge.
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  I was actually the 15th employee to join Novartis Cambridge Group
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  and I was at Novartis for 13 years.
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  I'm so grateful for all the opportunities I got at Novartis.
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  I had many different roles spanning from science, project
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  management, portfolio management.
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  I had LED innovation group for four years in which we looked at
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  external opportunities that were outside Novartis's 10
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  therapeutic areas. And there I actually LED a gene
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  therapy program for hearing loss, which really kind of
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  changed my view around what can be possible for treatments for
• 3:45
  patients. And at the end of the my career
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  in Novartis, I ended up doing a role in BD and alliances.
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  I think Novartis gave me a tremendous amount of
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  opportunities. I work with tons of smart
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  people. I had great mentors there and
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  learned so much. So I'm thankful that for that
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  experience. But after Novartis, you know, 13
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  years in pharma kind of makes you think about like how long
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  should I be in pharma before I get labeled as kind of only able
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  to perform in pharma? So I took the leap and moved to
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  biotech. I got an opportunity to work in
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  editas medicine right when it was being formed in 2016.
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  It's a gene editing company and was one of the kind of premier
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  gene editing companies that started around that time.
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  And this was a tremendous opportunity for me to grow,
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  enroll and to take everything I learned from Farmer and apply
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  those learnings, not all of them, but some of them to a
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  biotech type of company. And I'm really proud that I LED
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  the team around edit 101, which was the first in vivo CRISPR
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  editing reagent to go into or therapy to go into patients
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  ever. And this was for a rare form of
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  inherited retinal disease called LCA 10.
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  So this was really drove my passion around IR D's and really
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  helping patients with this one and done therapy.
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  After that, I did a short stint at Flagship and had lots of
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  learnings there and moved to Arbor where I really expanded my
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  role. I became Chief Operating Officer
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  at Arbor and spent 4 1/2 years in that role.
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  I had expanded my remit to project management, oversight,
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  operations, CMC and quality, regulatory and toxicology and
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  corporate strategy. So this was a great expansion of
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  role and I hired some really great people into those roles
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  that executed Florida State. But it also helped me understand
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  interactions with the board and I got to participate in Series B
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  fundraise and was more active in their Series C fundraising
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  activities. So that was working closely with
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  Devin Smith and was a really great learning experience.
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  But Luke Vandenberg reached out to me while I was at Arbor and
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  enticed me with this CE role for Leora Therapeutics.
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  Luke is known in the field for all his work on Aavs which are
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  viruses that package genetic medicines and also as an
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  entrepreneur. He started seven companies to
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  date and has been very successful in his career.
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  Also the other Co founder is Eric Pierce who's a clinician
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  scientist and actually he collaborated early with Editas
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  and Ed worked briefly with him around the Edit 101 program.
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  So the combination of Luke's experience in company building,
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  Eric's experience in being a clinician scientists and my
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  experience in operational and executional roles seem like a
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  great complementary skills that can really Dr. Leora forward.
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  So that's how we landed here today.
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  Absolutely no. Thank you for that.
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  And obviously since the recent announcement, sounds like Glora
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  has a, you know, robust pipeline.
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  Talk us through a bit more detail around Leora and the
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  products that you're developing. Yeah, Yeah.
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  So, Leora, we've set up the company and which we're really
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  focused and asset driven. We have three programs in our
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  portfolio. The three programs are all
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  focused around inherited retinal degenerative, degenerative
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  diseases. These programs have all been
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  preclinically validated and they intervene at the root cause of
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  disease. The three programs we have been
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  working on all emanated from the science of Eric Pierce's lab and
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  they have moved forward. Eric have has collaborated with
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  different pharmaceutical and biotech companies around these
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  programs and unfortunately some of both the companies have had
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  strategic shifts and are no longer working in this
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  particular area. So we, we acquired the assets
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  back through Mass Ioneer and Mass General and which we have
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  the rights to the programs. We're going to take these
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  programs and tweak and optimize them a little bit and then drive
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  them quickly into the clinic. So right now we're funding 2 of
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  the three programs, LYA one O 1 and LYA one O 2.
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  LYA One O 1 is a gene augmentation approach in which
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  we're looking to help people with retinitis pigmentosa
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  associated with mutations in the PRPF 31 gene.
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  We're really excited about this program as it has a straight
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  shot to the clinic. We've we have development plans
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  laid out in which we believe we can move this program quickly
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  towards clinic. The second program is LYA One O
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  2 and it's to address patients with Usher syndrome type 2 and
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  and mutations in Exxon 13 here. This is a bigger patient
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  population and and a very meaningful diseases.
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  Both these diseases patients have night vision and peripheral
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  vision loss as early teens or 20s and then have a slowly loose
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  vision over time until the 40s or 50s where they have
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  blindness. So we hope to intervene and save
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  those patients from progressing to blindness.
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  How do these two core programs differ from the current
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  approaches in those indications as well?
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  Yeah. Thanks, George.
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  This is a really important point.
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  We're going after these programs, one because we have
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  validated science too, because there's unmet medical need out
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  there. There is no approved therapies
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  for either of these programs and there is clinical testing right
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  now going on for these two indications.
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  However, we really differentiate from those and that we are gonna
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  deliver our therapeutics by ABS. We'll deliver them subretinally
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  so we can target the cells in the back of the eye, the cells
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  that need to have the gene incorporated in there and it's
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  going to be 1 and done treatment.
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  What we mean is we'll deliver it once in the life of the patient
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  and it should be durable therapy for the rest of the life time up
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  for the patient. This is really important to
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  patients. We've taught throughout my
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  career being in the gene editing space over the last 10 or 11
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  years. Patients always comment around
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  therapeutics that have you have to undergo treatment on a
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  regular basis such as ASOS or oligonucleotide treatment really
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  are not ideal because A they have access to doctors, B they
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  have to get intravitreal injections throughout the life
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  and C they have to have access to insurance in order to build
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  for these therapies. So it really impacts the stress
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  levels and the and for patients and families.
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  Yeah, the you noticed that obviously the AAV as the
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  delivery mess method, it is that the sole reason why these are
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  superior for IRDS? I think for two reasons.
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  One, because they're Aavs and they're going to be 1 and done.
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  2 because the Aavs allow you to target the access access to
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  cells better. So Asos have to travel through
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  the intravitreal space, get through barrier membrane to get
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  to these cells. So we have a much higher chance
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  of getting to our target cells. And three, we're going after the
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  root cause of the disease. Some of the Asos go after
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  modifier genes and not the root cause of the gene disease.
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  Yeah, fantastic. I guess talking sort of more
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  broadly, you spend a huge amount of your career within gene
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  therapy. Obviously it's had a rough last
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  few years in terms of the environment.
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  Do you do you feel there's change in the air?
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  You sound really optimistic. Yeah, I.
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  Guess you have to be optimistic in this field, George, but in
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  order to keep your sanity. But I really am optimistic.
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  And I think if you look at the data, actually the ophthalmology
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  gene therapy programs have outperformed other segments.
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  And So what makes me believe this one, if you look, Lily did
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  two big deals at the end of 2025, one with Mira and one with
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  that Bureau. So those were very appealing
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  deals. But then if you look at 25 to
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  26, a lot of the series fundraise and series AB and C,
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  we've seen companies like Rygays and avant-garde and Splice Bio
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  and Etsena and Mira all get serious fundraising.
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  And you probably saw it just recently, Mira received 400
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  million from Oberland Capital for their X linked retinitis
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  pigmentosa program. So I think we're seeing that
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  there's a lot of excitement. And then maybe just one other
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  point too is that there are several BLA filings.
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  Like to date, we've only really had Lux Turner approved for
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  RP65, but we're on the cusp of several companies coming in with
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  BLA filings, like four or five filings over the next year or
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  so. I think actually getting
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  approval for AV delivered ophthalmology programs is really
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  gonna incentivize the market to invest here.
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  And maybe lastly, our business model I think resonates with
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  this kind of asset driven fast the clinic approach and and so
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  that's why I feel optimistic we're not building up a huge
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  platform that then we're pulling out programs, we're really
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  driving specific assets towards the clinic.
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  Yeah. And, and touching on that point,
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  because I know you mentioned a few times, obviously you know,
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  you're going into the clinic rapidly, you know, you're
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  developing programs that were, you know, perhaps shelved by
• 14:55
  Biogen and and Editas. What's your anticipated timeline
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  on this? We are actively seeking
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  investors and and that I'm going to make the positive assumption
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  that we get the investors to not slow down our timelines and Dr.
• 15:10
  quickly. I think the lead program we
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  believe can be an Ind enabling studies in early 2027 and then
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  followed by clinical about a year and a couple months after
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  that. So we're we're very short term
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  to the clinical initiation. The Ashtuay LYA 1O2 programme is
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  a little bit behind that one, but also going to be very
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  focused and driving towards the clinic.
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  Yeah. What are the, I know you touched
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  on obviously fundraising and pushing into the clinic.
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  What are the biggest goals and milestones for LIOR and now over
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  sort of 6121824 months ahead? Yeah, great question.
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  I think for us, in the next 6 months, we hope to get some
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  substantial fundraising. We're looking for $24 million in
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  order to get our LYA One O 1 program to proof of concept read
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  out and the second program, I and D submission ready.
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  So that's our goal right now. I think we're very focused on
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  fundraising and hope to accomplish that in the next 6
• 16:22
  months. And then it's really about
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  moving the programs forward and executing around the lead
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  program. In the next year, we'll have
• 16:32
  completed manufacturing and GLP talks and that will be critical
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  to Derisa program and the RS2A program.
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  We're optimizing it now. So we hope in the next 6 to 12
• 16:46
  months to have a a development candidate out of that program.
• 16:50
  Fantastic. Talking about the sort of
• 16:52
  financing environments, I know obviously you touched on it at
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  Arbor as well. What, what are you seeing right
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  now? What?
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  What's the environment like? Yeah, I think it's, I think it's
• 17:02
  actually getting, it's heating up a bit.
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  Yeah, we come out of these big meetings, JPM and Bio and I
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  think there's a lot of excitement around the great
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  science that's out there and the potential to help patients with
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  unmet medical need. So I think the environment is
• 17:21
  actually changing right now. And you know we've we've kind of
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  put together our strategy, our portfolio, our development
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  plans, our intellectual property so that we're sewing up all the
• 17:35
  aspects of our program to be to enable quick movement.
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  We've to reached out and talked to investors and made some
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  pitches. I really feel very optimistic on
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  the feedback we've received. There are people that understand
• 17:49
  the type of AAB gene therapy we're doing.
• 17:52
  It's very, very selective. We're taking a very small amount
• 17:58
  of AAB and, and, and our genetic medicines are packaged in that
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  and then we're putting those AA BS in a very small quantity in
• 18:07
  the back of the eye. So there's no PIO distribution
• 18:11
  and and you know kind of systemic impact that are
• 18:16
  associated with other therapies. So I think a lot of investors
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  that understand our our modality of delivering these drugs are
• 18:25
  very keen on the Aavs and that the advantages they bring to our
• 18:30
  programs. So I think that's been very
• 18:32
  focused. I think the investors like our
• 18:36
  our capital efficient model that we are driving very quickly to
• 18:40
  major value inflection points and we're not you know kind of
• 18:45
  spending a lot of money on peripheral things that we're
• 18:48
  going to keep our team very focused and lean.
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  We have a very experienced team in place.
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  I just recently also hired a colleague that I worked with,
• 18:59
  Chris Wilson for a long time at Editas Medicine and he's going
• 19:03
  to help drive things forward from a science perspective.
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  So we have a lean model that's really pushing things forward
• 19:10
  that seems to resonate with investors at this particular
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  time. And I do feel optimistic that
• 19:16
  we're going to find the right investors that have a common
• 19:19
  mission to our mission, that we can actually change patients
• 19:23
  lives with these therapies and that it will be a smart
• 19:27
  investment for them. Yeah, absolutely.
• 19:30
  Well, we hope everything goes well in the short term with the
• 19:33
  financing. It's obviously a really exciting
• 19:36
  time for you with coming out of stealth mode recently.
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  So I mean, Pamela, thank you for coming on.
• 19:43
  Thank you for talking through Laura's story.
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  As a bit of a tradition on the podcast, we do always ask final
• 19:49
  question around, you know, if you could sit down and have
• 19:52
  dinner with any scientists, innovative past or present, you
• 19:55
  know, who would that be and why? That's a great question, George,
• 20:02
  and it really makes you think, you know, whether it be somebody
• 20:05
  from the past or or somebody today, you know, I've had the
• 20:09
  great pleasure of I I really believe that crisper gene
• 20:14
  editing is a scientific breakthrough that's been
• 20:18
  momentous and has huge potential.
• 20:20
  We have to figure out how to execute on that potential.
• 20:25
  And you know, I've interacted a lot with Feng Zhang.
• 20:28
  He started two of the companies Co founded two of the companies
• 20:31
  that I worked at at a Tawson Arbor.
• 20:34
  So I would like to meet Jennifer Doudna to see here her
• 20:37
  perspective. But if I had to sit down and
• 20:39
  have dinner with somebody, I'd really like that to be Kathy.
• 20:44
  Hi, she probably doesn't need a a huge introduction, but she is
• 20:50
  a clinician scientist and she was really instrumental in the
• 20:54
  early days of gene therapy and she continues to be a force out
• 20:59
  to today. You know, throughout my career,
• 21:03
  I've always been a champion of women in science.
• 21:05
  And, you know, sometimes I think women of my generation kind of
• 21:10
  had a struggle to figure out how to get ahead in science.
• 21:15
  But, you know, Kathy's a little bit older than I am, and I would
• 21:18
  love to hear her journey and how she overcame obstacles.
• 21:22
  You know, she started an early hemophilia gene therapy and then
• 21:27
  she broadened out and worked with Jean Bennett, who's also a
• 21:30
  pioneer in the field of of ophthalmology.
• 21:34
  And you know, she not only went from being a, an academic and
• 21:41
  working in active academic labs and teaching, but then she
• 21:45
  transitioned to industry and she was a Co founder of Spark
• 21:48
  therapeutic, CEO of VASPIO, and now she's ACEO of another
• 21:53
  ophthalmology company. And so I would really love to
• 21:57
  understand like she's a massive problem solver.
• 22:00
  She showed resilience and and true leadership in every
• 22:07
  discipline she's been in. So I'd really love to hear her
• 22:11
  journey and what she has to say to younger women's scientists
• 22:14
  today. Yeah, fantastic answer.
• 22:18
  Brilliant. No, look, we really appreciate
• 22:20
  you coming on, Pam. Like personally like really
• 22:22
  looking forward to, to seeing the successes that Laura has
• 22:25
  over the next few years as well. So yeah, thanks so much for
• 22:28
  coming on and and telling the story.
• 22:30
  Yeah, George, thank you so much for taking time to talk and hear
• 22:34
  about Leora. Yeah, we really hope we can
• 22:37
  bring these therapies to patients.
• 22:39
  Thank you. Fantastic.