Is there a connection between worms being eradicated from the gut in the early 20th Century and an increase in allergies and autoimmune disease?  

This week, we have a conversation with Holoclara CEO Andrea Choe, who not only believes there is a connection, but also sees secretions from worms as a way to tackle such diseases. 

01:31-06:05: About Holoclara
06:06-08:26: From discovery to setting up a company
08:26-10:40: Was this a symbiotic relationship?
10:40-12:30: As we are losing species for a variety of reasons, are there other species that may also be beneficial?
12:30-14:03: What are roundworms secreting?
14:03-15:28: How do you take these secretions and advance that to creating a drug?
15:28-15:44: Do you need the worms to make your product?
15:44-16:02: What conditions are you looking to address?
16:02-16:43: Are you looking to cures or disease maintenance?
16:43-16:58: How far down the path are you with this?
16:58-17:26: How important are partnerships and investment?
17:26-18:12: What is reaction like to the concept of using worms to tackle diseases?
18:12-19:25: What are the next steps?
19:25-20:44: Are there other cures to be discovered from other species?

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Jennifer Schranz is senior vice president and global head of rare diseases in the R&D division at Ipsen.

On the podcast this week, she discusses a breakthrough treatment for primary biliary cholerangitis and cholestatic liver disease.

00:56-00:49: Background information
01:44-03:21: What are primary biliary cholerangitis (PBC) and cholestatic liver disease?
03:21-03:50: How rare is PBC?
03:50-04:45: How easy is PBC diagnosis?
04:45-05:45: What are the current treatments for the condition?
05:45-07:22: What are the challenges to innovation?
07:22-08:27: What is elafibranor?
08:27-08:37: How is it administered?
08:37-09:18: Is elafibranor taken for life?
09:18-11:08: What is the ELATIVE trial, and what were the results you presented at the AASLD Liver Meeting?
11:08-13:09: What was the reaction at the meeting?
13:09-13:55: A patient-centric approach
13:55-14:44: Is a cure for PBC a possibility?
14:44-17:20: How important are acquisitions to Ipsen’s pipeline?
17:20-18:33: Working on multiple diseases
18:33-19:58: How much impact can you have on liver diseases?
19:58-21:04: What are the next steps for elafibranor?

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On this week’s podcast we have a conversation with Jordi Mata-Fink, co-founder and chief executive officer of the newly-launched U.S. company Gate Bioscience.

00:45-01:06: About Gate Bioscience
01:06-02:09: What are disease-causing extracellular proteins?
02:09-04:44: How have diseases caused by extracellular proteins been treated in the past?
 04:44-05:47: Have there been diseases that haven’t been addressed?
 05:47-07:05: Tackling the different issues created by extracellular proteins
 07:05-09:41: What are molecular gates?
 09:41-12:56: How does your technique distinguish between good and bad extracellular proteins?
12:56-16:24: How did the idea become a company?
16:24-17:24: Which diseases will you focus on?
17:24-18;47: How do you address costs?
18:47-19:11: How will this be given to patients?
19:11-20:01: Disease cure or disease management?
20:01-21:35: How was the company created?
21:35-24:18: The rise of small molecules
24:18-25:58: What are the next steps?

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Among other things, UK-headquartered company Phenotypeca has been working with the Bill & Melinda Gates Foundation to produce albumin for low and middle-income countries to reduce the costs of vaccines.

The company’s CEO, Johnny Cordiner, and research and development director, Professor Ed Louis, tell us about the work, as well as the company and its other projects.

01:01-11:57: About Phenotypeca
11:57-14:04: What is recombinant protein technology?
14:04-16:39: What are the issues around the cost of albumin?
16:39-20:48: How can you help improve affordability?
20:48-26:55: What is QTL technology?
26:55-30:18: What are the benefits of this technology?
30:19-34:22: Partnering with the Bill & Melinda Gates Foundation
34:22-36:37: What’s the timeline?
36:37-39:45: What else are you working on?

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French company CellProthera has seen some remarkable results treating people who have had a heart attack.  On this week’s podcast, the company’s CEO Matthieu de Kalbermatten tells us how it works.

01:00-03:29: About CellProthera
03:30-06:30: What are ProtheraCytes?
06:30-09:02: Is this a cure?
09:02-10:24: Is prevention the best option?
10:25-11:49: Is this therapy useful to treat other conditions?
11:50-13:55: How quickly do you need to treat a patient?
13:56-16:39: The field of regenerative therapy
16:39-17:52: Is regenerative therapy a growing sector?
17:53-18:26: Clinical trials
18:26-19:22: Upcoming phase 3 trials
19:22-20:36: The regulatory pathway
20:37-23:25: Cost and scale
23:25-24:20: The need for partnerships
24:21-24:57: Next steps
24:57-28:30: Is there excitement in the medical field for this treatment?

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On today’s podcast, Jean-Philip Piquemal, the chief scientific officer and co-founder of Qubit Pharmaceuticals, explains how the company’s Atlas platform is leading the way to more effective and safer drugs.

00:54-01:52: The history of Qubit Pharmaceuticals

01:52-04:04: What is the Atlas software?

04:04-05:18: Failing quickly is the key

05:19-06:53: What are digital twins?

06:54-09:00: Improving safety, reducing costs, and boosting drug discovery

09:00-10:44: Areas of interest

10:44-13:21: Recent successes

13:23-14:57: Preparing for future pandemics

14:58-17:03: Staying ahead of other companies

17:04-18:29: Enough diseases to go around

18:30-20:11: Upgrading Atlas software

20:13-21:59: How Qubit works with pharma companies

21:59-24:41: The Quantum for Bio program

24:42-27:58: Looking to the future, and helping patients
 

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This week, our guest on the podcast is Jason C. Foster, CEO of Ori Biotech.

Ori is a London, U.K. and New Jersey, U.S. based manufacturing technology company pioneering flexible process discovery with translation and scalable commercialization of cell and gene therapies (CGT). 

Ori has developed a proprietary, full stack manufacturing platform that closes, automates and standardizes CGT manufacturing allowing therapeutics developers to develop and bring their products to market at commercial scale. 

The promise of the Ori platform is to fully automate CGT manufacturing to increase throughput, improve quality and decrease costs by combining proprietary hardware, software, data and analytics.

Ori was founded in 2015 by Dr Farlan Veraitch from University College London.

The company also produces a monthly report on its website, which covers the latest in CGT approvals, regulations, trials and industry news.

This week, our guest on the podcast is Mark Kotter, founder of newly-created company clock.bio.

clock.bio, a sister company to bit.bio, which Kotter also founded and is the CEO of, aims to extend and improve quality of life by reversing the harmful effects of time in our cells, harnessing the regenerative capabilities of human pluripotent stem cells (hiPSCs).

These hiPSCs have the ability not only to stay young but also to rejuvenate when forced to age.

Clock.bio’s approach is to harness this power of ‘resetting the clock’ in hiPSCs to identify new approaches to treating age-related diseases. It has developed an aging model that ‘force ages’ hiPSCs and triggers their self-rejuvenation mechanism. Unbiased CRISPR screens on large samples of these cells allows for the identification of gene candidates that are causally relevant for cell rejuvenation.

This enables a decoding of rejuvenation biology and corresponding IP; the resulting knowledge will be used to create novel treatment approaches.

The company’s mission is to extend health span by 20 years based on biomarkers of aging in a phase 3 trial by the end of this decade. 

Clock.bio recently published a white paper for rejuvenation therapies and a blueprint for clock.bio.

This week, we speak to Artiva Biotherapeutics’ CEO, Fred Aslan, M.D.

The company has demonstrated the potential for efficacy and safety with natural killer (NK) cells in two cancer clinical trials. In August, the company announced FDA clearance for an IND for lupus – marking a first for an allogeneic, off-the-shelf NK or CAR-T cell therapy in autoimmune disease.

AlloNK (also known as AB-101) is a non-genetically modified, cord blood-derived, allogeneic, cryopreserved, ADCC-enhancing NK cell therapy candidate for use in combination with monoclonal antibodies or innate-cell engagers in the out-patient setting. 

Artiva is investigating AlloNK in a phase 1/2 multicenter clinical trial to assess the safety and clinical activity of AlloNK alone and in combination with the anti-CD20 monoclonal antibody, rituximab, in patients with relapsed or refractory B-cell-non-Hodgkin lymphoma (B-NHL). Artiva is also investigating the safety and clinical activity of AlloNK in combination with rituximab in patients with lupus nephritis. 

In addition, Artiva is collaborating with Affimed in a phase 2, open-label, multi-center, multi-cohort study, testing a combination therapy, comprised of AlloNK and the innate cell engager AFM13, for the treatment of patients with relapsed/refractory CD30-positive lymphomas. Artiva selects cord blood units with the high affinity variant of the CD16 receptor and a KIR-B haplotype for enhanced product activity. 

Using the company’s cell therapy manufacturing platform, Artiva can generate thousands of doses of pure, cryopreserved, infusion-ready NK cells from a single umbilical cord blood unit while retaining the high and consistent expression of CD16 and other activating NK receptors, without the need for engineering. AlloNK is being administered in the outpatient setting over multiple doses and multiple cycles.

Artiva’s pipeline also includes AB-201, an anti-HER2 CAR-NK cell therapy candidate for the treatment of HER2-overexpressing tumors, such as breast, gastric, and bladder cancers, and for which an IND has been allowed by FDA, and a pipeline of CAR-NK candidates targeting both solid and hematopoietic cancers. Artiva has also entered into therapeutic NK cell collaborations with Merck Sharp & Dohme. 

Compared to the smallest mRNA molecule, which is 300 nucleotides long, the largest transfer RNAs (tRNA) is less than a third of the size at 76 nucleotides. 

Scientists at Alltrna are harnessing the unique biology of tRNAs to engineer a single tRNA medicine that could treat thousands of rare diseases that share the same genetic mutation. This year, Alltrna presented at ASGCT the first demonstration that an engineered, modified tRNA could recognize and correct, in vivo, a flawed mRNA instruction no matter where it occurred in the genome.

Alltrna, which was founded in 2018 by Flagship Pioneering, recently announced it had raised $109 million in a Series B financing to advance the company’s platform and first drug candidates towards the clinic for a first indication in Stop Codon Disease.  

Stop Codon Disease encompasses ​​thousands of rare and common diseases that arise from premature termination codons (PTC), also known as nonsense mutations, where the code for an amino acid has been mutated into a premature “stop” codon. This results in a truncated or shortened protein product with no or altered biological activity that causes disease. Approximately 10% of all people with a genetic disease have Stop Codon Disease, representing approximately 30 million people worldwide. Alltrna’s tRNA medicines can read these PTC mutations and deliver the desired amino acid, thereby restoring the production of the full-length protein.

The company’s platform incorporates artificial intelligence and machine learning tools to ‘learn’ the tRNA language and deliver diverse, programmable molecules with broad therapeutic potential. 

This week, our conversation is with Alltrna CEO, Michelle Werner.

To learn more about the topic, we invite you to read the following article:  Will tRNA therapy be the next big thing in genetic disease treatment?