Base to Base Biotech

por Jim Cornall

Base to Base biotech podcast 63: Cell and gene therapy logistics, and turning back the Parkinson’s clock

This week, we have a conversation with Mark Sawicki, president and CEO of Cryoport Systems, and we chat with Nick Manusos, CEO of Kenai Therapeutics. Times: 03:22 Cryoport Systems 22:24 Kenai Therapeutics Cryoport Systems Cryoport Systems provides temperature-controlled supply chain solutions for the life sciences industry. The company supports the transportation, storage and management of sensitive biological materials used in biopharma development, reproductive medicine and animal health. Its services are designed to protect products and materials that require tightly controlled environments, including cell and gene therapies, biologics and reproductive specimens. The company offers an integrated platform that combines specialised shipping systems, logistics and transportation services, cryopreservation, biostorage and bioservices capabilities, and consulting support. Its solutions cover a range of temperature requirements, from cryogenic conditions below -150°C to refrigerated and controlled room-temperature environments. Cryoport Systems also provides monitoring and traceability technologies intended to maintain the integrity of materials throughout the supply chain. Cryoport Systems is part of Cryoport, Inc., a publicly traded company headquartered in Brentwood, Tennessee. Founded in 1999, Cryoport, Inc. serves customers globally through a family of companies that includes Cryoport Systems, MVE Biological Solutions and CRYOGENE. The organisation supports clinical and commercial programmes worldwide, with a particular focus on the temperature-controlled supply chain requirements of advanced therapies. Kenai Therapeutics Kenai Therapeutics recently announced a major milestone with the dosing of the first patient in its phase 1 clinical trial of RNDP-001. This approach is fundamentally disease-modifying, moving beyond symptom control to address the root cause of Parkinson's disease, and has the potential to be manufactured in large batches and readily available for more patients across the globe. Current Parkinson’s treatments focus almost entirely on managing the symptoms caused by the loss of dopamine-producing neurons. RNDP-001 represents a significant shift by aiming to replace those lost neurons, repair damaged neural circuits, and potentially restore motor function. Unlike patient-specific (autologous) therapies, RNDP-001 is an allogeneic (donor-derived), cryopreserved product built on a proprietary iPSC platform. This is a critical practical advancement, allowing the product to be manufactured in large batches and readily available for more patients, which is essential for global access. The therapy has been granted Fast Track designation by the U.S. FDA, acknowledging the urgent need for new treatments and the potential of RNDP-001 to address a serious unmet medical need. To get in touch with guest suggestions, or to sponsor or advertise on the podcast, please email jim@deeptechdigest.com

E63

12 jun 2026

48:54

Base to Base biotech podcast 62: A new approach to strokes

This week, we have a conversation with Revalesio president Greg Archambeau about tackling strokes, and other conditions, with a novel method. Revalesio While the pharmaceutical industry often focuses on complex chemical compounds, Revalesio is pioneering a novel drug class rooted in a surprising field: fluid physics. Revalesio is also focusing on stroke, a condition in need of new therapies. Revalesio’s breakthrough therapy, RNS60, is unlike traditional treatments. It uses a proprietary process to create an oxygen-supersaturated saline that harnesses physical forces to fundamentally improve cellular health. By boosting mitochondrial biogenesis and ATP production, the therapy addresses the core of cellular decline. Revalesio is currently at the frontier of human health with a primary focus on ischemic stroke, where they have already demonstrated game-changing results, with their phase 2 clinical trials showing a 50% reduction in brain loss and a significant reduction in disability for stroke patients. With a yearly global economic burden of stroke estimated at $890bn, RNS60 has the potential to unlock a new standard of care and drastically reduce hospital stays (from 10.8 days to 6.0 days in high-dose groups). The platform technology is also being explored for traumatic brain injury (TBI), Alzheimer’s disease, and broader healthy aging and health-span applications. Led by an executive team with deep roots at Eli Lilly, AbbVie, and Amgen, Revalesio is now moving toward phase 3 trials. To get in touch with guest suggestions, or to sponsor or advertise on the podcast, please email jim@deeptechdigest.com

E62

5 jun 2026

26:14

Base to Base biotech podcast 61: Making tumours visible, and taking on tough challenges

This week, we speak with John Friend, CEO of Kazia Therapeutics; and have a conversation with PureTech Health CEO Robert Lyne and Eric Elenko, president and co-founder of PureTech and acting CEO of Gallop Oncology. Times: 03:23 Kazia Therapeutics 31:56 PureTech Health PureTech Health PureTech Health plc is a hub-and-spoke biotherapeutics company dedicated to giving life to science and transforming innovation into value. The company recently announced positive topline data from its phase 1b clinical trial of LYT-200, a first-in-class, fully human anti-galectin-9 monoclonal antibody, in heavily pretreated patients with relapsed/refractory (R/R) high-risk (HR) myelodysplastic syndrome (MDS) and R/R acute myeloid leukaemia (AML). Based on the results, PureTech's founded entity, Gallop Oncology, has selected a recommended phase 2 dose (RP2D) and intends to engage with the U.S. Food and Drug Administration (FDA) to discuss the design of a subsequent trial that could potentially support registration of LYT-200 in R/R HR-MDS. PureTech’s strategy has produced dozens of therapeutic candidates, including three that have received U.S. FDA approval. Kazia Therapeutics About 1 in 8 women in the U.S. will develop breast cancer in their lifetime. While many patients benefit from advances in treatment, some of the most aggressive forms are still very difficult to treat and often stop responding to therapy. A growing focus in oncology is how to overcome that resistance by changing how tumours behave. Kazia Therapeutics is advancing a strategy centred on reprogramming cancer cells. Instead of just blocking growth, the goal is to make tumours less aggressive and more visible to the immune system. Their lead candidate, paxalisib, is an oral therapy currently being studied in advanced breast cancer. In metastatic triple-negative breast cancer, one of the most treatment-resistant tumour types, early data suggests that combining paxalisib with standard therapies may begin to address both immune resistance and metastatic drivers simultaneously. To get in touch with guest suggestions, or to sponsor or advertise on the podcast, please email jim@deeptechdigest.com

E61

29 may 2026

1:01:08

Base to Base biotech podcast 60: Delivery method reduces prostate cancer tumour size

This week, we have a conversation with the founder, director and chief scientific advisor of Alessa Therapeutics, Pamela Munster. Alessa Therapeutics Alessa Therapeutics recently announced positive preliminary phase 1 safety and efficacy data for its prostate cancer treatment Enolen. This was the first study to demonstrate that the FDA-approved prostate cancer compound enzalutamide can be safely and locally administered to the prostate via sustained drug eluting implants. Recent key data presented by researchers at the NCI at the European Association of Urology Congress in London included: All 20 patients enrolled in the initial cohort were successfully implanted. These implantations achieved very high intraprostatic enzalutamide levels with minimal systemic drug exposure and resulted in no delay to surgery. Pre-radical prostatectomy MRI’s conducted for 18/20 patients (2 pending) showed a reduction in tumour volume in 84% of the lesions over an average duration of 35 days. There were no reported effects on testosterone levels or negative effects on sexual function. To get in touch with guest suggestions, or to sponsor or advertise on the podcast, please email jim@deeptechdigest.com

E60

22 may 2026

24:43

Base to Base biotech podcast 59: DIA helping biotech thrive, and Gorlin syndrome

This week, we have a conversation with DIA chief science and regulatory officer, Maria Vassileva, and Medicus Pharma CEO, Raza Bokhari. Times: 02:53 Medicus Pharma 29:13 DIA DIA DIA (Drug Information Association) is a global organisation that assists life science professionals from across all areas of expertise to engage with patients, peers and thought leaders in a neutral environment on the issues of today and the possibilities for tomorrow. Starting with the controversy over the drug Thalidomide, DIA’s founders - a group of 30 pharmaceutical professionals, medical writers, and academics - came together to create a platform for necessary global communication and collaboration to solve a healthcare threat to unborn children worldwide. Today, professionals from 80 countries engage with DIA through its membership network, educational offerings, and professional development opportunities. Medicus Pharma Medicus Pharma Ltd. has submitted an Orphan Drug Designation (ODD) application to the U.S. Food and Drug Administration for SkinJect (D-MNA) for the treatment of basal cell carcinoma (BCC) in patients with Gorlin syndrome, a rare genetic disorder characterised by the development of multiple, recurrent skin cancers. There are no FDA-approved therapies specifically for BCC in Gorlin Syndrome. The submission represents a strategic expansion of the SkinJect programme into a high unmet need orphan indication, where current treatment options are limited and often involve repeated surgical procedures associated with cumulative morbidity and disfigurement. Medicus believes SkinJect can address this through a localised, repeatable, non-surgical treatment approach. It is a doxorubicin-containing dissolvable microneedle array designed for direct intradermal delivery into BCC lesions. SkinJect has been evaluated in phase 1 and 2 clinical studies in patients with basal cell carcinoma. To get in touch with guest suggestions, or to sponsor or advertise on the podcast, please email jim@deeptechdigest.com

E59

15 may 2026

1:05:07

Base to Base biotech podcast 58: Congenital myotonic dystrophy

This week, we have a conversation with congenital myotonic dystrophy patient advocacy consultant Lisa Harvey-Duren, who was the founding Myotonic Dystrophy Foundation executive director in 2008, and Michael Snape, CEO and CSO of AMO Pharma, which is developing a treatment for the disease. AMO Pharma AMO Pharma is a clinical-stage specialty biopharmaceutical company working to identify and advance promising therapies for the treatment of serious and debilitating diseases in patient populations with significant areas of unmet need, including rare and severe childhood onset neurogenetic disorders with limited or no treatment options. In addition to developing AMO-02 for congenital myotonic dystrophy, the company is also progressing AMO-02 as a clinical stage treatment for arrhythmogenic right ventricular cardiomyopathy. AMO-01 is being investigated for treatment of Phelan-McDermid syndrome and AMO-04 as a clinic-ready potential medicine for Rett syndrome and related disorders. To get in touch with guest suggestions, or to sponsor or advertise on the podcast, please email jim@deeptechdigest.com

E58

8 may 2026

44:59

Base to Base biotech podcast 57: Keeping drugs cool, and treatment resistant depression

This week, our conversations are with Uwe Diegel, CEO of Lifeina, about their cooling technologies, and with Dirk Beher, CEO of FundaMental Pharma, about treatment resistant depression. Times: 02:37 FundaMental Pharma 23:56 Lifeina FundaMental FundaMental Pharma is a preclinical biopharmaceutical company focused on developing new treatments for treatment‑resistant depression (TRD). The company’s platform centres on proprietary dual‑acting modulators of the N‑methyl‑D‑aspartate receptor (NMDAR). These molecules simultaneously disrupt the pathological complex formed between NMDAR and the TRPM4 ion channel, while also acting as potent NMDAR antagonists. This dual mechanism is designed to intervene upstream in the neurobiological processes associated with excitotoxicity and impaired neuronal signalling, offering a differentiated strategy compared with existing antidepressant classes. FMP374 is the company’s lead candidate and has demonstrated strong antidepressant‑like effects at low nanomolar concentrations in preclinical proof‑of‑concept studies. Importantly, these studies have not shown dissociation‑related behaviours, ataxia or hyperactivity at efficacious doses, supporting the potential for an at‑home therapeutic profile. The molecule is orally bioavailable and is progressing toward IND‑enabling studies. The company’s goal is to deliver new therapeutic options for patients with severe unmet medical needs, where current treatments often fail to provide adequate relief. Lifeina Lifeina is a French health‑tech company focused on portable, connected medical‑device solutions for people who rely on temperature‑sensitive medications. Its flagship product, LifeinaBox, is a compact, battery‑powered mini‑fridge designed to keep biologics such as insulin, growth hormones and certain injectable therapies within a safe temperature range during travel or daily use. The device pairs with a mobile app that monitors temperature, battery status and usage patterns, providing alerts if the medication is at risk. The company positions its technology as a way to give patients greater independence and mobility. Lifeina works with patient groups, clinicians and pharmaceutical partners to refine its devices and expand compatibility with a wider range of biologic medicines. To get in touch with guest suggestions, or to sponsor or advertise on the podcast, please email jim@deeptechdigest.com

E57

1 may 2026

46:21

Base to Base biotech podcast 56: GRIN genes and relapsing MS

This week, our conversations are with Bruce Leuchter, co-founder, president, CEO of Neurvati Neurosciences and GRIN Therapeutics; and Daniel Vitt, CEO of Immunic. Times: 02:54 Immunic 24:04 Neurvati Neurosciences/GRIN Therapeutics GRIN Therapeutics GRIN Therapeutics Inc. is dedicated to the research and development of precision therapeutics for neurodevelopmental disorders with the goal of bringing hope to patients and caregivers. In 2024, GRIN Therapeutics reported promising topline data from a phase 1b/2a clinical trial evaluating investigational radiprodil in GRIN-related neurodevelopmental disorder (GRIN-NDD) in patients with gain-of-function variants, leading to the decision to advance to the global pivotal phase 3 Beeline trial. The company has an additional ongoing clinical trial to evaluate radiprodil for the potential treatment of tuberous sclerosis complex (TSC) and focal cortical dysplasia type II (FCDII). GRIN Therapeutics is an affiliate of Neurvati Neurosciences. Immunic Immunic Inc. is a late-stage biotechnology company pioneering the development of novel oral therapies for neurologic diseases. The company’s lead development programme, vidofludimus calcium (IMU-838), is currently in phase 3 clinical trials for the treatment of relapsing multiple sclerosis, for which top-line data is expected to be available by the end of 2026. It has already shown therapeutic activity in phase 2 clinical trials in relapsing-remitting multiple sclerosis, progressive multiple sclerosis and other diseases. Vidofludimus calcium combines neuroprotective effects, through its mechanism as a first-in-class nuclear receptor-related 1 (Nurr1) activator, with additional anti-inflammatory and anti-viral effects, by selectively inhibiting the enzyme dihydroorotate dehydrogenase (DHODH). The company’s development pipeline also includes earlier-stage programmes, including IMU-856 and IMU-381, aimed at building a broader therapeutics platform addressing neurodegenerative, chronic inflammatory, and autoimmune-related diseases. To get in touch with guest suggestions, or to sponsor or advertise on the podcast, please email jim@deeptechdigest.com

E56

24 abr 2026

1:03:28

Base to Base biotech podcast 55: Beating transplant challenges and a circular supply chain

This week, our conversations are with Steve Perrin, president and CSO of Eledon Pharmaceuticals, and Jason Bock, CEO and co-founder of CTMC. Times: 03:18 CTMC 35:46 Eledon Pharmaceuticals CTMC CTMC is a joint venture between Resilience and the MD Anderson Cancer Center. It works like an internal manufacturing group, adapting to change in real time without charge ups and allowing for better control as a company develops their research process. With all of the resources in one circular supply chain (patients, clinicians, manufacturing, and regulatory), CTMC is executing the manufacturing and analytical processes while also supporting active engagement with MD Anderson, preparing to launch trials as soon as the FDA approves an IND. CTMC provides biotech partners with access to MD Anderson's clinical trial network, allowing therapies to be tested in patients without the biotech having to coordinate the trials themselves. The recent Link announcement of $60m Series A financing led by Johnson & Johnson highlights the success of the partnership between CTMC and Link for advancing the development of CAR T therapies for solid tumours into the clinical stage. Eledon Pharmaceuticals There are major challenges in transplantation, such as a global donor organ shortage and patients often needing repeat transplants. Eledon Pharmaceuticals is the only company developing an immunosuppression drug that targets the CD40 ligand (CD40L) pathway in organ transplantation – a well-validated immune system communication pathway. This anti-CD40L antibody aims to reduce or eliminate the toxicities associated with today's standard of care drugs and help organs last longer, which may also free up organs for new patients, reduce repeat transplants, and help alleviate the global donor organ shortage. Eledon plans to move into a phase 3 trial in kidney transplantation this year after discussions with the FDA. The company is also active in islet cell transplants resulting in insulin independence in patients with previously near-uncontrollable type 1 diabetes. To get in touch with guest suggestions, or to sponsor or advertise on the podcast, please email jim@deeptechdigest.com

E55

17 abr 2026

1:02:37

Base to Base biotech podcast 54: Parkinson’s disease and peptides

This week, because April 11 is World Parkinson’s Day, we chat with Dieter Willbold, CSO of Priavoid, and Antje Willuweit, director preclinical development of Priavoid, and Karsten Eastman, CEO and co-founder of Sethera Therapeutics, about the company’s work on peptides. Times: 02:52 Sethera Therapeutics 22:35 Priavoid Priavoid Priavoid, a company advancing novel disease-modifying approaches for neurodegenerative disorders, recently presented preclinical proof-of-concept data for its candidate PRI-101 in Parkinson’s disease (PD) and related synucleinopathies. The results show that PRI-101 inhibited aggregation of α-synuclein (α-syn) and actively disassembled α-syn-fibrils across in vitro and in vivo models relevant to PD. α-syn aggregates play a key role in the disease pathology of Parkinson’s, multiple system atrophy (MSA), and other synucleinopathies. In PD mouse models, PRI-101 was associated with prolonged median survival versus placebo. Short- and long-term treatment also led to a dose-dependent reduction of α-syn aggregates in the brain, accompanied by significant improvements in behavioural performance. PRI-101 is orally available all-d-peptide candidate based on Priavoid’s proprietary detangler platform. By binding to α-syn, PRI-101 aims to promote the conversion of the toxic and self-replicating neurodegenerative aggregates back towards native, non-toxic α-syn-monomers, thereby counteracting disease-driving aggregation processes. Sethera Therapeutics A team of researchers from the University of Utah and Sethera Therapeutics have uncovered a new way to build more stable and drug-like peptides, opening the door to medicines that could target diseases long considered “undruggable.” The team studied a natural enzyme, PapB, that can ‘staple’ peptides into circular structures known as macrocycles. What makes PapB so unusual is that it combines flexibility and precision: it works on many different building blocks—including those that biology usually rejects—yet still creates a single, predictable bond. In a single gentle step, it transforms linear peptides into sturdy, ring-shaped molecules that are more stable, more resistant to degradation, and better suited for drug development. Many peptide drugs are stabilized with disulfide bonds, which break down in the body, or rely on complicated, costly, and time-consuming chemical methods to achieve the same effect. PapB streamlines the process, creating durable “stapled” peptides that drug developers can program with unprecedented ease. This opens vast new chemical space for peptide medicines, including scaffolds associated with better cell penetration and oral dosing—two qualities essential for advancing peptide therapeutics. To get in touch with guest suggestions, or to sponsor or advertise on the podcast, please email jim@deeptechdigest.com

E54

10 abr 2026

40:43

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