Raising Biotech

In this episode of Raising Biotech, Surani explores the untapped world of tRNA therapeutics with Flagship Pioneering-founded company Alltrna. The company raised $109 million in a series B in August 2023. Surani talks to CEO Michelle Werner about the inception of Alltrna, the company's tRNA therapeutic thesis and her journey to the role. She also talks about the company's plans to study its technology in rare genetic liver diseases and the potential to accelerate clinical trials with the use of basket trials. Dr David Weinstein, a rare diseases pediatrician and owner of Weinstein Rare Disease and Clinical Development Consulting also joins the podcast to give his take on the potential of tRNA therapeutics to serve thousands of diseases. He gives his thoughts on the company's mission, potential clinical trial challenges ahead and how the therapeutics might fit into the treatment paradigm.Timestamps:01:58 - Background of Alltrna and tRNA therapeutics for rare genetic diseases06:00 - Company formation under Flagship and Michelle's personal backstory11:35 - Focussing on stop-codon diseases of the liver for first clinical trials12:55 - Dr Weinstein talks about tRNA therapies for rare genetic diseases16:55 - The use of basket trials to accelerate clinical trials 18:30 - Efficacy and safety considerations & regulatory pathway21:45 - How tRNA therapies would fit into the treatment paradigm24:05 - Potential for tRNA in other genetic diseases beyond the liver 26:00 - Alltrna's future pipeline and business strategy For any comments, questions, feedback or suggestions you can connect directly with Surani Fernando on LinkedIn or email: raisingbiotech@gmail.comMusic composed by: Yrii Semchyshyn (Coma Media)

In this episode of Raising Biotech, Surani delves into Arialys and its mission to revive an autoimmune encephalitis drug from the grave after big pharma no longer wanted it. The company founders are a bunch of venture capitalists (Avalon Ventures, MPM Capital & Catalys Pacific) who saw potential in pursuing the preclinical drug candidate -- raising a $58 million seed financing in September 2023 to take it into human testing.Surani speaks to CEO Jay Lichter (Avalon) about his background, success stories, how he stumbled across Astellas' drug (now ART5803) and why he was so impressed with preclinical data in non-human primate marmoset models. Marty Jefson, CEO of Pinteon Therapeutics, a veteran neuropsychiatry drug development executive, talks about the drug's potential to fit in the treatment paradigm and what convinced him to join the company's clinical advisory board. Also joining the podcast to give medical context on the drug's potential is Dr Leon Henderson MacLennan, medical advisor and co-founder, InThought. The experts discuss the drug's safety and efficacy considerations, clinical trial hurdles and potential for the drug to expand to other autoimmune psychosis conditions including schizophrenia. Jay talks about clinical trial plans ahead, his preferences when it comes to a future IPO vs M&A and what he thinks will be the key value driver for a company exit.Timestamps:01:56 - Jay's background & history investing in novel technologies03:30 - Astellas looks to sell CNS assets04:00 - What exactly is anti-NDMA-receptor encephalitis (ANRE)?05:15 - Avalon Ventures team up with MPM Capital and Catalys Pacific for asset sale07:10 - Preclinical trials and impressive data in marmoset models09:15 - Marty (Pinteon) discusses ANRE burden and ART5803's clinical potential12:10 - Leon (InThought) discusses unmet need for targeted approach13:55 - Marty and Leon talk about first impressions of preclinical data15:05 - Experts discuss human trial considerations and Jay talks clinical trial plans19:15 - Safety considerations and historical challenges developing CNS drugs22:00 - Market opportunity of ANRE and other autoimmune indications like schizophrenia26:10 - Arialys' financing plans, long-term visions and potential future exitFor any comments, questions, feedback or suggestions you can connect directly with Surani Fernando on LinkedIn or email: raisingbiotech@gmail.comMusic composed by: Yrii Semchyshyn (Coma Media)

In this episode of Raising Biotech, Surani delves into Synchron and its mission to develop a potential first-in-class implantable brain computer interface (BCI) that allows people with motor impairment to communicate their thoughts through a device. Founded in 2012, Synchron has raised $145 million since inception, which has taken the implant into clinical trials and the company is gearing up for its final clinical trial.Surani speaks to Dr Tom Oxley, about his origins as a neurologist in Australia and his inspiration for Synchron's BCI. He talks about his struggles to finance the company in Australia, which led Synchron to the US. Dr Elad Levy, Professor and Chair, Department of Neurosurgery, University at Buffalo gives his view of the technology's potential, particularly its favorable risk profile and straightforward implant procedure versus more invasive competitor BCIs. Dr David Putrino, Director of Rehabilitation Innovation, Mount Sinai Health System speaks about the BCI's utility, its potential to improve patient quality of and reduce overall healthcare costs. Kurt Haggstrom, CCO talks about navigating Synchron through reimbursement and commercial considerations and building Synchron into a bigger brand. Tom outlines next-step clinical trial and financing plans, thoughts on IPO vs a sale and ultimate visions for the company.Timestamps:01:46 - The crux of the Synchron's BCI03:00 - Back story of Tom and the "light bulb" for Synchron05:52 - Learning the hard way: Australian investors vs US investors07:23 - Synchron vs BCI competitors (including Elon Musks's Neuralink)11:10 - Published clinical trial data so far, current risk/benefit profile12:23 - Dr Levy (UB) gives his view on the potential impact of Synchron's BCI14:57 - Dr Putrino (Mount Sinai) talks about Synchron's impressive progress16:43 - Dr Levy and Dr Putrino give thoughts on clinical data, safety and efficacy19:20 - Kurt and Tom talk about designing the pivotal trial with reimbursement in mind23:24 - Expert insights on potentially acceptable pricing of a BCI25:36 - Market reach with tech boom and "less complex" implant procedure27:58 - Future financing plans, IPO possibilities, and building Synchron into a bigger brand30:58 - Tom's thoughts on acquisition and comments on Elon Musk's BCI vision32:15 - Future vision for Synchron and BCI capabilitiesFor any comments, questions, feedback or suggestions you can connect directly with Surani Fernando on LinkedIn or email: raisingbiotech@gmail.comMusic composed by: Yrii Semchyshyn (Coma Media)

In this episode of Raising Biotech, Surani Fernando speaks with eGenesis CEO Mike Curtis on its mission to create pig donor organs for humans waiting for a transplant. The company has raised $263 million over the last 6 years and is now raising further funds to start human clinical trials.Surani speaks to Mike about the company's history overcoming past Xenotransplantation challenges and using CRISPR Cas9 to finalize their platform -- which conducts 69 edits in the pig genome for optimal compatibility and safety in humans. After recent data (Nature) showed a maximum survival of a monkey surviving with a pig kidney for over 2 years, the company is talking to regulators to enter clinical trials and in parallel is talking to investors and potential partners to take its programs forward. As the company enters unchartered territory, all future strategic business options are on the table. Dr Adam Griesemer, associate professor of surgery at Grossman School of Medicine, NYU Langone Health, gives his take on the unmet need and progress of Xenotransplanation and eGenesis' scientific thesis of 69 gene edits. He also discusses the academic debate around the number of genetic edits needed and logistical considerations for implementing and scaling the availability of pig donor organs for human transplantation.Timestamps:02:00 - CEO Mike Curtis describes organ shortage issue02:57 - The history of xenotransplantation04:57 - Creating the perfect pig donor organ: 69 gene edits & using CRISPR Cas906:40 - Founding of eGenesis with Harvard scientists George Church and Luhan Yang07:30 - Financing so far, raising $263 million in three financing rounds09:02 - Nature paper showing two-year survival of monkey with pig kidney10:46 - Dr Adam Griesemer (NYU) paints unmet need and eGenesis' scientific thesis15:07 - Thoughts on recent results and academic debate on number of genetic edits16:45 - Talks with regulators for clinical path forward in kidney transplant18:07 - Target patient population for first US human trials and Japanese opportunity20:45 - Logistical and implementation considerations if finally approved21:45 - Choosing pigs as ideal donors and upscaling considerations25:05 - Societal impacts: patient advocacy and animal welfare sentiment27:00 - Future financing plans and potential partnerships with global players29:30 - eGenesis' 10 year vision of donor organs without immunosuppressionFor any comments, questions, feedback or suggestions you can connect directly with Surani Fernando on LinkedIn or email: raisingbiotech@gmail.comMusic composed by: Yrii Semchyshyn (Coma Media)

In this episode of Raising Biotech, Surani speaks with CEO and co-founder Jack O'Meara of Ochre Bio, a company dedicated to tackling the large and unsolved problem of complex and chronic liver diseases. Founded in 2019, Ochre raised an impressive $30 million after only three years in start-up mode and is now gearing up to take some of its drug candidates into the clinic.Surani speaks to Jack about his individual journey and the moment he met and decided to partner up with co-founder Dr Quin Wills. Jack discusses their light bulb idea of setting up a company to study the liver and develop novel and targeted gene therapies using human donor organs as preclinical models instead of mice. Jack talks about Ochre's fundraising journey in Europe vs US, his own experiences raising funds as a relatively young biotech founder and how the company successfully set up an organ "ICU" to conduct its research.Dr Scott Friedman, Dean of therapeutic discovery and Chief of the division of liver diseases at the Icahn School of Medicine, Mount Sinai, joins the podcast to share his thoughts on Ochre's thesis and mission with siRNA gene therapies and its "deep phenotyping" approach. With 40 years of experience in the field, he's seen a lot of failure, so discusses what types of challenges Ochre will have to think about as they progress into human testing.Jack discusses next steps as the company moves towards the clinic -- which involves partnership talks, a Series B fundraising and narrowing down the first indication to test its medicines.Timestamps:02:00 - CEO Jack O'Meara gives Ochre Bio's liver disease thesis04:08 - Rewinding to Jack's back story and connecting with co-founder Dr Quin Wills05:30 - Generating the "light bulb" moment of using discarded human donor organs for research07:05 - Why liver disease, particularly NASH has seen so many catastrophic failures (Intercept)08:38 - Quin and Jack search for investors and take roadshow to more "risk-friendly" US investors10:25 - What it was like for Jack being such a young biotech entrepreneur and how that impacted fundraising13:40 - Dr Scott Friedman (Mount Sinai) gives his take on Ochre's scientific thesis and mission15:00 - After some initial logistical hiccups, Ochre builds its own "organ" ICU centre for testing17:36 - Use of siRNA therapy and deep phenotyping approach to generate lead drug candidates20:52 - A compelling strategy to de-risk trials, but potential challenges ahead to consider24:45 - Partnership discussions and Series B plans to get drugs into the clinic25:08 - Prioritizing "smaller" disease to tackle in first clinical trials28:17 - Jack's visions in building Ochre into a large multi-product company in Europe.For any comments, questions, feedback or suggestions you can connect directly with Surani Fernando on LinkedIn or email: raisingbiotech@gmail.comMusic composed by: Yrii Semchyshyn (Coma Media)

A new narrative podcast series hosted by journalist Surani Fernando that explores biotechs raising impressive funds to develop ambitious medical breakthroughs. Surani speaks with CEOs and founders as well as medical and industry experts for a comprehensive view of each unique endeavor.