Developing treatments for rare pediatric diseases

Beyond Biotech - the podcast from Labiotech

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All About Change

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Professional football player Jonathan Jones: Mentorship and Making an Impact in Your Community 22:49

för 9 dagar sedan22:49

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Jonathan Jones is an NFL cornerback for the Washington Commanders who rose from the undrafted ranks to become two-time Super Bowl champion with the New England Patriots, a businessman, philanthropist, and licensed pilot. In 2019, Jonathan founded the Jonathan Jones Next Step Foundation in 2019, a platform dedicated to empowering youth through education, professional development, and mentorship. The foundation works to alleviate food insecurity, promote women in stem and sports, and to promote professional development in the communities where he lives. Jay and Jonathan talk about investing in the communities they live in, acknowledging the people who helped you become the person you are, and paying that same investment forward to the next generation. Episode Chapters 0:00 intro 1:24 Building local connections 4:25 Jonathan’s mentors and mentees 10:54 Jonathan’s pride in his mentees’ successes 13:04 how Jonathan chooses his causes 14:08 Jonathan’s support for girls and young women 17:19: Jonathan’s passion for flying 19:40 The Next Step Foundation 20:29 Goodbye For video episodes, watch on www.youtube.com/@therudermanfamilyfoundation Stay in touch: X: @JayRuderman | @RudermanFdn LinkedIn: Jay Ruderman | Ruderman Family Foundation Instagram: All About Change Podcast | Ruderman Family Foundation To learn more about the podcast, visit https://allaboutchangepodcast.com/ Looking for more insights into the world of activism? Be sure to check out Jay’s brand new book, Find Your Fight , in which Jay teaches the next generation of activists and advocates how to step up and bring about lasting change. You can find Find Your Fight wherever you buy your books, and you can learn more about it at www.jayruderman.com .…

för ungefär ett år sedan 29:17

MP3•Episod hem

Rare diseases affect around 1 in 2,000 people. Many of the thousands of rare diseases are difficult to diagnose. Of the known rare diseases, about 80% have genetic causes.

Leukodystrophies are a group of rare genetic disorders for which there is currently no curative therapy.

This week on the podcast we are discussing novel treatments of rare pediatric diseases in a conversation with Dan Williams, CEO, executive director and co-founder of Synaptix Bio. Synaptix Bio is working on developing treatments for severe leukodystrophies.

The company has partnered with the Children’s Hospital of Philadelphia to take a TUBB4a leukodystrophy therapy to the clinic and beyond.

01:41-05:08: About Synaptix Bio
05:08-07:07: What are rare diseases?
07:07-09:36: How do you develop treatments for rare diseases?
09:36-11:15: How many rare diseases have treatments?
11:15-13:32: What is the space looking like for companies looking to develop rare disease treatments?
13:32-14:10: How does the fact that many rare diseases are genetic affect drug development?
14:10-16:21: Do the low numbers of people with rare diseases affect funding and research?
16:21-17:55: Are the drugs being developed useful in treating other diseases?
17:55-22:44: What is H-ABC?
22:44-23:25: Are there any treatments for H-ABC?
23:25-25:15: Where is Synaptix Bio with clinical trials?
25-15-27:40: How do you design clinical trials for H-ABC with the number of patients being low?

This week’s podcast was sponsored by KBI Biopharma. Visit their facility in Geneva, Switzerland, on June 27, to join the discussion on solving biotherapeutic challenges. Register at kbibiopharma.com/conferences.

Interested in being a sponsor of an episode of our podcast? Discover how you can get involved here!

Stay updated by subscribing to our newsletter

Kapitel

1. Developing treatments for rare pediatric diseases (00:00:00)

2. About Synaptix Bio (00:01:41)

3. What are rare diseases? (00:05:08)

4. How do you develop treatments for rare diseases?  (00:07:07)

5. How many rare diseases have treatments?  (00:09:36)

6. What is the space looking like for companies looking to develop rare disease treatments? (00:11:15)

7. How does the fact that many rare diseases are genetic affect drug development?  (00:13:32)

8. Do the low numbers of people with rare diseases affect funding and research? (00:14:10)

9. Are the drugs being developed useful in treating other diseases?  (00:16:21)

10. What is H-ABC?  (00:17:55)

11. Are there any treatments for H-ABC?  (00:22:44)

12. Where is Synaptix Bio with clinical trials?  (00:23:25)

13. How do you design clinical trials for H-ABC with the number of patients being low? (00:25:15)

143 episoder

#News #Tech News #Labiotech

Developing treatments for rare pediatric diseases

Beyond Biotech - the podcast from Labiotech

published för ungefär ett år sedan

Dela

MP3•Episod hem

Rare diseases affect around 1 in 2,000 people. Many of the thousands of rare diseases are difficult to diagnose. Of the known rare diseases, about 80% have genetic causes.

Leukodystrophies are a group of rare genetic disorders for which there is currently no curative therapy.

The company has partnered with the Children’s Hospital of Philadelphia to take a TUBB4a leukodystrophy therapy to the clinic and beyond.

Interested in being a sponsor of an episode of our podcast? Discover how you can get involved here!

Stay updated by subscribing to our newsletter

Kapitel

1. Developing treatments for rare pediatric diseases (00:00:00)

2. About Synaptix Bio (00:01:41)

3. What are rare diseases? (00:05:08)

4. How do you develop treatments for rare diseases?  (00:07:07)

5. How many rare diseases have treatments?  (00:09:36)

6. What is the space looking like for companies looking to develop rare disease treatments? (00:11:15)

7. How does the fact that many rare diseases are genetic affect drug development?  (00:13:32)

8. Do the low numbers of people with rare diseases affect funding and research? (00:14:10)

9. Are the drugs being developed useful in treating other diseases?  (00:16:21)

10. What is H-ABC?  (00:17:55)

11. Are there any treatments for H-ABC?  (00:22:44)

12. Where is Synaptix Bio with clinical trials?  (00:23:25)

13. How do you design clinical trials for H-ABC with the number of patients being low? (00:25:15)

143 episoder

#News #Tech News #Labiotech

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Beyond Biotech - the podcast from Labiotech

1
How nanobodies will revolutionize immunotherapy 41:13

för 12 dagar sedan41:13

41:13

From llamas and camels to humans worldwide. Nanobodies are enjoying success in the clinic and experts are hailing these camelid-derived biologics as one of the next big things in immunotherapy. Kristian Reich is co-founder and CSO of MoonLake Immunotherapeutics. After a long and distinguished career as a clinician and researcher, Kristian helped to launch MoonLake in 2021 and quickly helped move their lead candidate to the clinic. Now, on the verge of Phase III trials, he joins 'Beyond Biotech' to talk nanobodies, innovation, serendipitous science, and the power that comes from never stopping to dream. 01:04 Meet Kristian Reich 05:26 Excited by the science of nanobodies 07:13 MoonLake’s mission 13:12 Milestones at MoonLake 15:24 Introduction to nanobodies 17:55 Serendipitous science 19:46 How nanobodies work 23:18 Global prevelance of immune conditions 24:31 The impact on patients 27:09 Nanobodies in the clinic 31:19 Manufacturing challenges, regulatory challenges 34:04 Working with top pharma 35:55 The future of nanobodies 37:42 The future of MoonLake 39:10 Don’t stop dreaming Interested in being a sponsor of an episode of our podcast? Discover how you can get involved here! Stay updated by subscribing to our newsletter…

Beyond Biotech - the podcast from Labiotech

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för 19 dagar sedan39:38

39:38

Cell therapies are at the cutting edge of personalized medicine but as demand for these therapies grows, how will the industry scale manufacturing to meet patient needs? Fabian Gerlinghaus is co-founder and CEO of Cellares. He and his team have developed the Cell Shuttle, an advanced cell manufacturing technology that is already demonstrating the potential to 10x cell yields while halving costs for pharmaceuticals companies and innovators. Is this the future for smart cell manufacturing? 0:52 A starting point in aeronautical engineering 2:43 First steps into biotech 5:25 Co-founding Cellares 6:30 The problem that Cellares addresses 11:02 Best practice bio manufacturing 17:14 Differentiation in automation 18:15 What does success look like for Cellares 19:38 Smart factories 21:58 Inside the Cellares smart factory 26:34 Manufacturing CAR-T and stem cell therapies 27:54 Mass production vs personalized medicine 31:39 Global ambitions and regional regulators 35:07 Positioning Cellares in the market Interested in being a sponsor of an episode of our podcast? Discover how you can get involved here! Stay updated by subscribing to our newsletter…

Beyond Biotech - the podcast from Labiotech

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för 26 dagar sedan33:48

33:48

Spinal cord injuries impact millions of people worldwide and, unlike many chronic injuries, they usually arrive with no forewarning, impacting the young and old alike. Brian Culley is CEO of Lineage Cell Therapeutics and his company is now in the clinic with a stem cell therapy for spinal cord injuries. Learn why stem cells could be promising for these devastating injuries and how Brian and his team plan to overcome the challenges of manufacturing at scale and succeeding in a highly regulated space. 00:57 Meet Brian Culley 02:15 Lineage Cell Therapeutics and its mission 04:56 Spinal cord injuries 06:40 Costs beyond the Individual 08:15 Current treatment options 10:48 The place for stem cell therapies 12:57 Lineage in the clinic 16:27 The DOSED program 18:37 The advantages of DOSED 20:29 Timelines for results 21:35 Scaling manufacturing 24:28 Partnering with pharma on stem cell therapies 26:38 Other applications for stem cell therapies 28:49 Mainstreaming stem cell therapies 30:52 Challenges ahead 32:43 The future for Lineage Cell Therapeutics Interested in being a sponsor of an episode of our podcast? Discover how you can get involved here! Stay updated by subscribing to our newsletter…

Beyond Biotech - the podcast from Labiotech

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för 5 weeks sedan37:47

37:47

Mitochondria are famously the powerhouse of the cell, but will mitochondrial therapeutics power the next big mainstream medical breakthrough? Klaus Dugi is CEO of Vandria, a biotech that develops mitophagy inducers that rejuvenate cells to treat age-related and chronic diseases. He joins 'Beyond Biotech' to talk cell mitophagy, biotech agility, and innovation in tackling neurodegenerative disease. 00:42 Introducing Klaus Dugi 02:51 Lessons learned in top pharma 05:10 The gap that Vandria seeks to fill 06:21 Mitochondrial therapeutics 07:45 Mitophagy inducers and how they work 14:53 Comparing approaches to treating neurodegenerative diseases 17:20 Aging, longevity, and healthy life years 25:26 Challenges ahead 27:31 Performance enhancement and resilience 31:15 Towards the mainstream? 34:35 Looking forward for Vandria 36:02 Developments to watch out for in mitochondrial therapeutics Interested in being a sponsor of an episode of our podcast? Discover how you can get involved here! Stay updated by subscribing to our newsletter…

Beyond Biotech - the podcast from Labiotech

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för 6 weeks sedan41:51

41:51

Regeneration Biomedical has developed a new technique to address neurodegenerative diseases like Alzheimer's using stem cell therapy. Dr. Christopher Duma, has long been an innovator in neurosurgery using a gamma knife radiosurgery to treat glioblastoma. Now with Regeneration Biomedical, Duma is taking on neurodegenerative diseases with a first-in-human clinical trial where a patient's own stem cells are injected directly into their brain. On the podcast this week, we talk with Duma about his career in neurosurgery, the impetus for launching his biotech, the promise of stem cell therapy, and the challenges innovators face moving new therapies through the clinic. 00:38 Introducing Dr Christopher Duma 02:44 Advances and breakthroughs in neurosurgery 05:33 The vision and impetus for Regeneration Biomedical 10:45 Regeneration Biomedical’s R&D pipeline 14:12 Milestones ahead 14:46 Advancing beyond Alzheimer’s Disease 15:52 Stems cells from fat cells 19:42 Bypassing the blood-brain barrier 22:45 Patient recruitment 24:13 The treatment process 27:10 The state of stem cell research in the US 29:29 The challenge of financing innovative research 32:15 Global stem cell research landscape 33:14 Controversy in stem cell research 34:00 Future horizons for Regeneration Biomedical 37:23 Partnering with top pharma companies 37:48 Advice for researchers and entrepreneurs Interested in being a sponsor of an episode of our podcast? Discover how you can get involved here! Stay updated by subscribing to our newsletter Learn more about the latest research in Alzheimer's disease treatment !…

Beyond Biotech - the podcast from Labiotech

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[Rebroadcast] How AI immune system mapping can boost drug discovery 28:53

för 7 weeks sedan28:53

28:53

We’re taking a short break this week and, while we’re away, we invite you to enjoy one of our favorite episodes. We will return with a brand-new episode next week! Immunai is mapping the immune system at unprecedented scale and granularity. The map, paired with machine learning, looks at how the immune system will respond to drug targets, offering an affordable way to prevent expensive drug failures. The ultimate goal is to market immune treatments for diseases like cancer faster than ever before. In this week’s conversation, Noam Solomon, CEO and co-founder of Immunai, covers the data gap in drug discovery and how machine learning (ML) can solve it, how to de-risk early-stage drug discovery, predictions for AI, and more. 00:49-01:13: About Immunai 01:13-01:45: Why map the immune system? 01:45-02:44: Are you taking a step back to study the problem in order to move forward? 02:44-03:49: How difficult is it to map the immune system? 03:49-05:29: What is your AMICA platform? 05:29-07:24: Where does your data come from? 07:24-09:09: How do you account for differences between patients? 09:09-11:35: What are the biggest challenges to drug development? 11:35-14:07: How can AI improve drug development? 14:07-14:55: Will AI advances speed up drug development? 14:55-16:06: Is the use of AI applicable in all diseases and conditions? 16:06-17:48: What sets your approach apart from other companies using AI? 17:48-18:54: What partnerships does Immunai have? 18:54-20:24: What are pharma companies looking for from Immunai? 20:24-23:17: How can AI help with clinical trials? 23:17-24:32: Can AI help with preventative care? 24:32-26:30: Google Maps for the immune system 26:30-27:18: What will we see from AI in drug discovery in the short term? 27:18-28:06: What are the next steps for Immunai? Interested in being a sponsor of an episode of our podcast? Discover how you can get involved here! Stay updated by subscribing to our newsletter…

Beyond Biotech - the podcast from Labiotech

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för 8 weeks sedan26:42

26:42

Sweden's fastest growing private biotech, Anocca, is making plans for clinical trials in pancreatic cancer, with the TCR-T cell therapy trials called VIDAR-1. The CEO, Reagan Jarvis, when a researcher, pitched the company idea to a leading Swedish industrialist, who became co-founder, and provided the initial financing. The company has raised more than €100m to date. On the podcast this week, we have a conversation with Jarvis about TCR-T cell therapies, creating off-the-shelf products and partnerships with EmendoBio and Shinobi Therapeutics. 00:43-02:10: About Anocca 02:10-04:16: About TCR-T cell therapies and their use in treatments 04:16-06:02: Anocca’s approach to T-cell biology, cutting-edge biotechnologies and integrated software 06:02-07:58: A different approach to other cell and gene therapy companies 07:58-09:16: Facing challenges 09:16-10:48: Addressing conditions and diseases 10:48-11:54: Pancreatic cancer 11:54-13:40: What represents success for Anocca? 13:40-14:09: What is VIDAR-1? 14:09-16:08: Partnerships 16:08-18:05: Scaling up and addressing costs 18:05-20:07: How is TCR-T therapy evolving? 20:07-23:11: The impact of artificial intelligence 23:11-25:16: Anocca timeline 25:16-25:57: Closing comments ` Interested in being a sponsor of an episode of our podcast? Discover how you can get involved here! Stay updated by subscribing to our newsletter…

Beyond Biotech - the podcast from Labiotech

1
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för 9 weeks sedan29:56

29:56

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Beyond Biotech - the podcast from Labiotech

1
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för 10 weeks sedan21:24

21:24

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Beyond Biotech - the podcast from Labiotech

1
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för 11 weeks sedan32:32

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In the fall of 2024, Haya Therapeutics, based in Switzerland and San Diego, landed a $1bn deal with Eli Lilly to apply its long non-coding RNA (ncRNA) technology to obesity. The company’s lead candidate, HTX-001, is making progress towards the clinic in cardiomyopathy. CEO Samir is one of the first researchers to publish on lncRNA – which is also known as the Dark Genome This week, our podcast guest is Samir Ounzain, CEO and co-founder of Haya Therapeutics. Ounzain was one of the first researchers to publish on lncRNA, also known as the ‘dark genome.’ 00:55-03:47: About Haya Therapeutics 03:47-06:37: What is the dark genome? 06:37-09:29: What is the connection between lncRNA and disease? 09:30-13:22: How can disease be addressed via the dark genome? 13:22-16:45: Which diseases are you tackling? 16:45-18:02: What is the EchoHAYA platform? 18:02-21:07: How does your lead candidate, HTX-001, work? 21:07-22:00: Is the objective stopping fibrosis or reversing it? 22:00-23:41: Other companies in the field 23:41-24:52: Working with Eli Lilly 24:52-27:15: A new frontier in medicine? 27:15-28:43: Cost effectiveness 28:43-30:07: Clinical trials 30:07-31:48: Applications to other diseases Interested in being a sponsor of an episode of our podcast? Discover how you can get involved here! Stay updated by subscribing to our newsletter…

Beyond Biotech - the podcast from Labiotech

1
Can Tumor Activated Therapy kill solid tumors? 31:22

för 12 weeks sedan31:22

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Solid tumors present a big challenge for current treatments. However, a new approach might provide the answers - Tumor Activated Therapy. By targeting specific proteins common in all solid tumor micro-environments, Seekyo scientists think this can induce self-destruction of the tumor itself. Seekyo’s lead therapy, SKY01, can only be activated within the tumor, making it a highly-targeted solution. This differentiated approach overcomes the limits of existing treatments, such as small molecule or antibody-drug conjugates. It could also be more cost effective than many advanced, far cell and gene therapy alternatives. Seekyo is looking for clinical entry to target four of the more challenging solid cancers - pancreas, TNBC, colorectal and lung, in an umbrella PhI/IIa. This will demonstrate safety and initial efficacy readings whilst at the same time defining the lead indication to progress to later-stage development. This week, our guest is Seekyo Therapeutics’ CEO, Oury Chetboun. 00:38-01:57: About Seekyo Therapeutics 01:57-03:17: The challenges of treating solid tumors 03:17-06:08: What is Tumor Activated Therapy? 06:08-07:46: What is SKY01? 07:46-08:10: What happens to the cancer cells? 08:10-09:52: Does the tumor vanish? 09:52-10:16: How is it delivered? 10:16-11:30: Are other companies working on tumor-activated therapies? 11:30-13:09: What treatment options are being worked on for solid tumors? 13:09-13:47: Long-lasting treatment 13:47-15:33: Clinical trials 15:33-16:22: Timelines 16:22-18:08: Does the treatment have other potential applications? 18:08-19:59: Cost effectiveness 19:59-21:59: Is the goal to cure people? 21:59-22:46: Treatment frequency 22:46-24:00: The impact of tumor size 24:00-25:23: Seekyo Therapeutics’ pipeline 25:23-27:27: Fund-raising 27:27-30:33: Reaction to the therapy Interested in being a sponsor of an episode of our podcast? Discover how you can get involved here! Stay updated by subscribing to our newsletter…

Beyond Biotech - the podcast from Labiotech

1
J.P. Morgan Annual Healthcare Conference 2025: trends and highlights you may have missed 19:57

för 13 weeks sedan19:57

19:57

This week, we take a look at some of the highlights from the 43rd Annual J.P. Morgan Healthcare Conference, which took place in San Francisco recently. While there weren’t necessarily many financial blockbusters, there were plenty of announcements and discussions on everything from artificial intelligence to the rising influence of Chinese companies and research, to the potential ramifications of the incoming Trump administration in the U.S. To help us navigate some of the highlights of the event, we spoke with Orca Bio’s co-founder and CEO, Ivan Dimov, and also Parabilis Medicines’ chief business officer, Greg Miller. 02:01-03:53: About Orca Bio 03:53-05:43: JPM highlights 05:43-07:26: Stand-out deals and announcements 07:26-09:01: Were any sectors more prominent than others? 09:01-12:38: About Parabilis Medicines 12:38-13:58: JPM highlights 13:58-16:33: Stand-out deals and announcements 16:33-17:53: Were any sectors more prominent than others? 17:53-19:17: Was there an optimistic mood? Interested in being a sponsor of an episode of our podcast? Discover how you can get involved here! Stay updated by subscribing to our newsletter…

Beyond Biotech - the podcast from Labiotech

1
Venture capital co-creation: The next big thing in biotech investment? 37:22

för 14 weeks sedan37:22

37:22

Jessica Owens and Iana Dimkova are co-founders of Initiate Ventures—a female-led venture capital firm and studio that launched recently with a $45 million debut fund. Initiate Ventures is shaking up the traditional venture capital model by blending investments in healthcare, life sciences, and technology with a company creation platform. The company is tackling some of the most pressing challenges in healthcare, and their innovative approach offers fresh insights into what it takes to scale transformative startups. Owens was the co-founder of GRAIL (acquired by Illumina for $8bn) and was a partner at Kleiner Perkins, where she helped shape some of the most successful health tech startups. Dimkova is a former healthcare technology investor at GE Ventures, who also scaled a company that raised more than $700m and built one of the largest radiation therapy networks in the US. Their approach is reflected in their dual model: funding existing companies while also co-creating ventures alongside founders in their startup studio. They helped launch groundbreaking companies like Macro Trials, a precision clinical research platform, and Persana, a leader in oncology diagnostics. This week on the podcast, we have a conversation with one of the co-founders of the company, Jessica Owens. 00:40-05:19: About Initiate Ventures 05:19-08:33: What sets Initiate Ventures apart from other venture funds? 08:33-10:59: Revitalizing healthcare 10:59-13:46: Determining realistic company visions 13:46-15:15: Initiate Ventures and Initiate Studios 15:15-19:50: Working with companies 19:50-21:36: Working with new companies 21:36-23:00: Evaluating potential success 23:00-24:40: How hands on is Initiate Ventures? 24:40-28:24: Is there a change in what attracts capital? 28:24-30:02: Infectious disease – a neglected area 30:02-33:08: How important are ESG considerations? 33:08-35:42: Trends for 2025 35:42-36:42: Goals for 2025 Interested in being a sponsor of an episode of our podcast? Discover how you can get involved here! Stay updated by subscribing to our newsletter…

Beyond Biotech - the podcast from Labiotech

1
Developing non-opioid pain drugs 33:01

för 15 weeks sedan33:01

33:01

Brixton Biosciences, a clinical-stage life sciences company spun out from Massachusetts General Brigham, has been awarded $2m from the NIH HEAL Initiative to support the development of non-opioid, injectable pain relief solutions. As part of the NIH's efforts to tackle the U.S. opioid crisis, the funding will advance research into safer, drug-free alternatives to opioid pain management. Brixton’s Neural Ice is an injectable, drug-free pain management technology designed to provide long-lasting relief from chronic and post-operative pain. On the podcast this week, we have a conversation with Sameer Sabir, CEO and co-founder of Brixton Biosciences, to dive deeper into pain treatment, the opioid crisis, and Brixton’s mission. 01:05-02:47: About Brixton Biosciences 02:47-03:39: Brixton’s pipeline 03:39-07:46: The opioid crisis 07:46-08:41: The extent of the crisis 08:41-12:00: Current alternatives to opioids 12:00-12:40: Managing pain 12:40-17:19: How was Neural ice developed? 17:19-18:33: Duration of treatment 18:33-21:01: Suitability for different levels of pain 21:01-21:31: Speed of effect 21:31-22:37: Side effects 22:37-24:18: Opioids vs. non-opioids 24:18-25:33: Are there different responses to Neural Ice? 25:33-26:29: Standardized doses 26:29-27:42: Pricing 27:42-28:49: Other work on alternatives to opioids 28:49-30:45: Challenges to developing new pain treatments 30:45-31:57: Other priorities at Brixton 31:57-32:16: Timeline for Neural Ice Interested in being a sponsor of an episode of our podcast? Discover how you can get involved here! Stay updated by subscribing to our newsletter…

Beyond Biotech - the podcast from Labiotech

1
[Rebroadcast] The power of natural killer cells 28:59

för 17 weeks sedan28:59

28:59

While our podcast takes a brief break, Labiotech invites you to enjoy some of our favorite episodes. We will return with a brand-new episode on January 3rd, 2025! Have an awesome holiday season! On this podcast , 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 2023, 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. Interested in being a sponsor of an episode of our podcast? Discover how you can get involved here! Stay updated by subscribing to our newsletter…

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Liknande Beyond Biotech - the podcast from Labiotech

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Beyond Biotech - the podcast from Labiotech « » Developing treatments for rare pediatric diseases

Kapitel

1. Developing treatments for rare pediatric diseases (00:00:00)

2. About Synaptix Bio (00:01:41)

3. What are rare diseases? (00:05:08)

4. How do you develop treatments for rare diseases? (00:07:07)

5. How many rare diseases have treatments? (00:09:36)

6. What is the space looking like for companies looking to develop rare disease treatments? (00:11:15)

7. How does the fact that many rare diseases are genetic affect drug development? (00:13:32)

8. Do the low numbers of people with rare diseases affect funding and research? (00:14:10)

9. Are the drugs being developed useful in treating other diseases? (00:16:21)

10. What is H-ABC? (00:17:55)

11. Are there any treatments for H-ABC? (00:22:44)

12. Where is Synaptix Bio with clinical trials? (00:23:25)

13. How do you design clinical trials for H-ABC with the number of patients being low? (00:25:15)

Developing treatments for rare pediatric diseases

Kapitel

1. Developing treatments for rare pediatric diseases (00:00:00)

2. About Synaptix Bio (00:01:41)

3. What are rare diseases? (00:05:08)

4. How do you develop treatments for rare diseases? (00:07:07)

5. How many rare diseases have treatments? (00:09:36)

6. What is the space looking like for companies looking to develop rare disease treatments? (00:11:15)

7. How does the fact that many rare diseases are genetic affect drug development? (00:13:32)

8. Do the low numbers of people with rare diseases affect funding and research? (00:14:10)

9. Are the drugs being developed useful in treating other diseases? (00:16:21)

10. What is H-ABC? (00:17:55)

11. Are there any treatments for H-ABC? (00:22:44)

12. Where is Synaptix Bio with clinical trials? (00:23:25)

13. How do you design clinical trials for H-ABC with the number of patients being low? (00:25:15)

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Välkommen till Player FM

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Liknande Beyond Biotech - the podcast from Labiotech

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Beyond Biotech - the podcast from Labiotech « »
Developing treatments for rare pediatric diseases

4. How do you develop treatments for rare diseases?  (00:07:07)

5. How many rare diseases have treatments?  (00:09:36)

7. How does the fact that many rare diseases are genetic affect drug development?  (00:13:32)

9. Are the drugs being developed useful in treating other diseases?  (00:16:21)

10. What is H-ABC?  (00:17:55)

11. Are there any treatments for H-ABC?  (00:22:44)

12. Where is Synaptix Bio with clinical trials?  (00:23:25)

4. How do you develop treatments for rare diseases?  (00:07:07)

5. How many rare diseases have treatments?  (00:09:36)

7. How does the fact that many rare diseases are genetic affect drug development?  (00:13:32)

9. Are the drugs being developed useful in treating other diseases?  (00:16:21)

10. What is H-ABC?  (00:17:55)

11. Are there any treatments for H-ABC?  (00:22:44)

12. Where is Synaptix Bio with clinical trials?  (00:23:25)