Beyond Biotech - the podcast from Labiotech Labiotech

German-headquartered Isarna Therapeutics is developing a portfolio of antisense therapies targeting an emerging therapeutic field in human biology – transforming growth factor beta (TGF-B) signaling.

Precise modulation of TGF-B pathways using antisense therapy may result in safer and more effective treatment options for a broad range of indications.
The company’s lead compound, ISTH0036, is in clinical development in ophthalmology.
On the podcast this week we chat with René Rückert, chief operating officer at Isarna Therapeutics, who explains TGF-B and antisense oligonucleotides’ role in tackling disease.

00:59-02:45: About Isarna Therapeutics
02:45-05:05: What is transforming growth factor beta?
05:05-06:16: Why is TGF a good target?
06:16-09:00: What are antisense oligonucleotides?
09:00-10:02: What are antisense oligonucleotides useful for treating?
10:02-11:21: What advantages do antisense oligonucleotides have?
11:21-13:00: How can antisense oligonucleotides be delivered?
13:00-14:58: Are antisense oligonucleotides still being researched?
14:58-16:31: Are there challenges with using antisense oligonucleotides?
16:31-16:56: Are many companies working on antisense oligonucleotides?
16:56-18:06: Are antisense oligonucleotides best used as a monotherapy or with other treatments?
18:06-20:42: What is Isarna’s pipeline?
20:42-22:47: How does your AMD treatment work?
22:47-23:50: What is the timeline for your treatment?
23:50-26:34: What can patients expect?

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Cytokinetics is a late-stage, specialty cardiovascular biopharmaceutical company focused on discovering, developing and commercializing first-in-class muscle activators and next-in-class muscle inhibitors as potential treatments for debilitating diseases in which cardiac muscle performance is compromised.
This week, we have a conversation with the CEO of Cytokinetics, Robert Blum, about the company’s aficamten, its next-in-class cardiac myosin inhibitor, prior to results from a phase 3 clinical trial in obstructive hypertrophic cardiomyopathy. 

00:54-02:32: About Cytokinetics
02:32-04:11: What are the current treatments for cardiovascular conditions?
04:11-05:44: About cardiovascular disease
05:44-08:59: What is aficamten?
08:59-09:12: How is it administered?
09:12-10:04: Where is aficamten on the path to commercialization?
10:04-12:06: What does this mean for patients?
12:06-12:42: What will you be presenting in Portugal?
12:42-16:06: Could aficamten have other applications?
16:06-16:50: What is CK-136?
16:50-18:02: What is omecamtiv mecarbil?
18:02-20:26: Cytokinetics’ business model

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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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