Beyond Biotech - the podcast from Labiotech Labiotech

Duchenne muscular dystrophy (DMD) is a genetic disorder with progressive muscle degeneration and weakness due to changes in the protein dystrophin, which helps keep muscle cells intact. 
There are few treatments for DMD, although recent advances mean that males – who are primarily affected by DMD – can now sometimes survive into their 30s.


More drugs are being developed in the fight against DMD, and this week we had a conversation with Frank Gleeson, CEO of Satellos Bioscience, about recent work on addressing DMD, including the company’s own work in the field.

00:47-02:01: About Satellos Bioscience
02:01-04:33: What is Duchenne muscular dystrophy?
04:33-05:43: What are the challenges in treating Duchenne muscular dystrophy?
05:43-09:08: What are the new treatments for Duchenne muscular dystrophy?  
09:08-11:20: What is your treatment for Duchenne muscular dystrophy?
11:20-13:42: Are there different approaches to treating Duchenne muscular dystrophy?
13:42-14:06: How is your treatment delivered?
14:06-17:32: How important is early intervention?
17:32-18:56: Where is Satellos at with clinical trials?
18:56-20:41: Preclinical trial results
20:41-21:44: Outreach to the Duchenne muscular dystrophy community
21:44-23:12: Is a cure for Duchenne muscular dystrophy possible?

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This week, we have a conversation about clinical trial design with Gen Li, the president and founder of Phesi, a global provider of patient-centric data analysis. 
Earlier this year, Phesi’s global analysis of all clinical trials conducted in 2023 revealed that more than a quarter (28%) of trials were cancelled during phase II – above the average attrition rate of 20% before 2020. 

00:39-01:31: About Phesi
01:31-01:49: Is your database global?
01:49-03:08: How successful are the different phases of clinical trials?
03:08-04:29: What are the biggest challenges for clinical trials currently?
04:29-06:23: Are clinical trials improving?
06:23-08:14: How can data improve clinical trials?
08:14-10:47: How does artificial intelligence affect clinical trial design?
10:47-12:53: Can clinical trial costs be reduced?
12:53-15:15: Can clinical trial times be shortened? 
15:15-18:21: Can data help with clinical trial diversity?
18:21-19:46: How can you fill knowledge gaps?
19:46-22:01: Do you have less data for rare diseases?
22:01-23:28: How does your company help with clinical trial design?
23:28-25:12: What kind of solutions can you provide?

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RTW is a global multi-strategy investment firm focused on supporting innovative biotech and biopharma companies across the US, Europe, and China. 
The company invests in both public and private companies across their entire life cycle, with a focus on those addressing next-generation gene and RNA therapies, rare diseases, targeted oncological, cardiovascular, and neurological disorders, and more.

To get an overview of what’s happening in financing for biotech companies currently, we had a conversation with Stephanie Sirota, chief business officer of RTW Investments.

01:11-04:18: About RTW Investments
04:18-06:45: What kind of biotech companies do you invest in?
06:45-08:31: How do you evaluate biotech companies?
08:31-11:36: How hands-on are you with companies you invest in?
11:36-14:08: How can companies raise funds currently?
14:08-16:05: Are there any undervalued sectors in biotech?
16:05-17:52: How important are M&As?
17:52-18:30: Is there a trend with M&As?
18:30-19:46: What is the state of the market with IPOs?
19:46-21:21: What are the major trends in biotech in 2024?
21:21-22:17: How could the US elections affect biotech funding?
22:17-24:41: What is the royalties market?
24:41-26:12: Are there regional differences?
26:12-27:52: Getting good biotech deals today

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Doloromics is a precision neuropharmaceuticals company. It was set up four years ago, to use newer technologies, particularly in the omics space, to take advantage of different single-cell transcriptomic technologies or proteomic technologies to better understand the progression of neurological diseases like chronic pain.
The Doloromics pipeline is built upon a proprietary discovery and validation platform DOLOReS. 


Jackson Brougher, chief scientific officer and co-founder of Doloromics, is our guest on this week’s Beyond Biotech podcast.

00:46-04:41: About Doloromics
04:41-07:09: What is chronic pain?
07:09-08:30: How challenging is the subjectivity of pain?
08:30-11:04: What is nociception?
11:04-12:23: The challenges of treating pain
12:23-16:35: Is chronic pain a disease?
16:35-19:57: What is VX-548?
19:57-22:17: Monoclonal antibodies and CGRPs
22:17-26:22: How have recent advances in genomic technologies advanced the understanding of pain?
26:22-31:03: What is the DOLOReS platform?
31:03-32:37: Delivery methods
32:37-33:55: Keeping costs low
33:55-34:58: Next steps


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Based in Austin, Texas, biotech Colossal Biosciences is at the forefront of de-extinction, critically endangered species protection and the repopulation of critical ecosystems. 
Its mission is to use CRISPR technology to bring back extinct animals, such as the mammoth, the thylacine, and the dodo.


Since DNA was recovered in 2021 from mammoths frozen in the Arctic tundra, the company has been working on splicing bits of the recovered DNA into the genome of its closest relative, the Asian elephant, as it shares 99.6% of its DNA. 

Ben Lamm, co-founder and CEO of Colossal Biosciences spoke with us about the company’s plans.

01:03-02:57: Introduction to Ben Lamm
02:57-04:26: About Colossal Biosciences
04:26-09:35: What techniques are used to tackle de-extinction?
09:35-13:48: What are the biggest challenges?
13:48-14:57: What tools do you need to ‘build’ an extinct species? 
14:57-19:17: How do you address not adversely affecting ecosystems?
19:17-22:22: How do you take on negativity over your de-extinction work?
22:22-23:26: Reintroducing existing species into previous habitats?
23:26-24:06: Does this extend to critically endangered species?
24:06-25:54: Why is there a lot of interest in de-extinction?
25:54-28:10: How do you ensure we don’t repeat the same patterns of extinction?
28:10-29:38: Can you address the loss of current species?
29:38-30:35: How can you help solve the loss of species?
30:35-33:48: Addressing the challenges of introducing different species?
33:48-35:22: Transparency through documentaries
35:22-37:27: 2028 goal for mammoth calves

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It would appear that 2024 is shaping up to be a big year for the development of treatments for schizophrenia, with several clinical trials under way.
Recently, Boehringer Ingelheim and Sosei Group Corporation (Sosei Heptares) announced a global collaboration and exclusive option-to-license agreement. This is to develop and commercialize Sosei Heptares’ portfolio of first-in-class GPR52 agonists, a novel G protein-coupled receptor (GPCR) target. The goal is to improve patient outcomes by addressing the positive, negative, and cognitive symptoms of schizophrenia.


Clíona MacSweeney, Sosei Heptares’ GPR52 program leader, spoke with us about that partnership, as well as other developments in the field of schizophrenia treatments.

00:57-03:44: What is schizophrenia?
03:44-04:52: What are the current schizophrenia treatments?
04:52-06:27: Why is schizophrenia challenging to treat?
06:27-07:34: Why is 2024 a big year for schizophrenia treatments?
07:34-08:56: Seratonin and dopamine modulation
08:56-10:46: How does muscarinic modulation work?
10:46-12:35: Excitatory and inhibitory tone pipeline 
12:35-14:12: Digital therapeutic and epigenetic modulation
14:12-16:12: GPR52 agonists
16:12-17:53: How are GPR52 agonists different?
17:53-19:03: Are there lots of schizophrenia treatments coming?
19:03-20:10: Will some new schizophrenia treatments be used in conjunction with existing treatments?
20:10-20:49: Is a schizophrenia cure possible?
20:49-21:22: Treatment frequency
21:22-22:21: Where is Sosei Heptares with clinical trials for schizophrenia?
22:21-22:45: What else is in Sosei Heptares’ pipeline?
22:45-23:35: Sosei Heptares becoming Nxera
23:35-24:42: Schizophrenia conferences

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