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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