A Data Strategy to Capitalize on a MultiโTrillion Dollar Opportunity
Will Greene, lead author of a new paper from the World Economic Forum urges policymakers, payers, and business leaders to see rare diseases as one of the greatest underappreciated opportunities in glo...
Will Greene, lead author of @weforum.org paper, discusses how smarter collection, sharing and analysis of rare disease data could unlock multi-trillion dollar gains in human healthโand what itโll take to rally global stakeholders. @globalgenes.bsky.social #RARECast
globalgenes.org/raredaily/a-...
27.02.2026 15:47 โ
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YouTube video by Life Sciences DNA Podcast
Orchestrating Scientists, Data, and AI to Discover New Drugs
Scotch McClure, CEO of Maxwell, explains why AI is becoming the engine of modern drug developmentโspeeding discovery, filtering noise in massive datasets, and taking on more of the scientific process under clear vision and guardrails.
www.youtube.com/watch?v=A9lE...
26.02.2026 20:04 โ
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Leukemia survivor and @columbiauniversity.bsky.social hematologist-oncologist Aaron Viny is reimagining blood cancer careโmoving from blunt chemo to precision strategies that rewire malignant cells, epigenetic programs, and their ecosystems.
thebioreport.podbean.com/e/reprogramm...
25.02.2026 17:48 โ
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When a Gene Echoes Through a Family
Rajani Aatre, senior genetic counselor at Michigan Medicineโs Frankel Cardiovascular Center, discusses the importance of cascade genetic testing, the ethical tension between honoring a patientโs priva...
Rajani Aatre of Michigan Medicine discusses the importance of cascade genetic testing, ethical tensions around its use, and the question of how to share life changing genetic information with relatives. @globalgenes.bsky.social #RARECast
globalgenes.org/raredaily/wh...
20.02.2026 17:41 โ
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Terns Pharmaceuticals' CEO Amy Burroughs discusses a strategic shift from metabolic disease to focus on the company's experimental therapy for chronic myeloid leukemia and how she is charting a clear path toward a broader oncology future.
thebioreport.podbean.com/e/a-strategi...
18.02.2026 15:51 โ
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Changing the Autoimmune Disease Playbook with RNA-Engineered CAR T Cells
Autoimmune diseases like myasthenia gravis have long forced patients to trade daily function for chronic immunosuppression, but [โฆ]
Cartesian Therapeutics Carsten Brunn discusses how the companyโs RNAโengineered CAR T cells target the root cause of autoimmune diseases, data from its study in myasthenia gravis, and the potential to expand indications. @globalgenes.bsky.social #RARECast
globalgenes.org/raredaily/ch...
13.02.2026 17:09 โ
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YouTube video by Life Sciences DNA Podcast
Scaling Practical AI Across Commercial Teams
Drew McCormick, head of data and analytics for Eversana, sits down with Nagaraja Srivatsan to trace the companyโs AI journey from classic predictive modeling in its data and analytics business to an enterprise-wide strategy. www.youtube.com/watch?v=eKuW...
12.02.2026 18:01 โ
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Ron Cooper, CEO of enGene, discusses the companyโs experimental gene therapy for nonโmuscle invasive bladder cancer, and how its dual payload is meant to activate both an innate and adaptive immune response in the bladder.
thebioreport.podbean.com/e/a-one-two-...
11.02.2026 16:29 โ
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Rewriting Rare Disease R&D with Foundation Models
Jean-Philippe Vert, co-founder and CEO of Bioptimus, discusses the inherent messiness of biology, the potential to transform rare disease drug development with an AI-driven foundation model, and how u...
Jean-Philippe Vert of Bioptimus discusses the inherent messiness of biology, the potential to transform rare disease drug development with an AI-driven foundation model, and how this could enable repurposing of existing drugs. @globalgenes.bsky.social #RARECast
globalgenes.org/raredaily/re...
06.02.2026 16:07 โ
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Michael Roberts, CEO of Adaptin Bio, discusses the companyโs platform technology that uses patient-derived T cells as delivery vehicles for targeted biologics that cross the blood-brain barrier to treat glioblastoma.
thebioreport.podbean.com/e/reprogramm...
04.02.2026 16:45 โ
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Resetting Aberrant Tregs Epigenetically to Treat Autoimmune Diseases
Michael McCullar, CEO of RegCell, discusses the role of dysfunctional Tregs in autoimmune diseases, the firmโs use of epigenetically modified regulatory T cells to treat these conditions, and how this...
Mike McCullar of RegCell discusses the firmโs use of epigenetically modified regulatory T cells to treat autoimmune conditions, and how this approach can suppress harmful immune responses without causing broad immunosuppression. @globalgenes.bsky.social #RARECast
globalgenes.org/raredaily/re...
29.01.2026 16:17 โ
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Xaira's Marc Tessier-Lavigne discusses applying end-to-end AI across target discovery, molecular design, and patient stratification; the companyโs $1 billion in funding, and how it seeks to enable a new generation of scientists.
thebioreport.podbean.com/e/a-billion-...
28.01.2026 16:28 โ
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Finding a Ready Treatment for a Newly Discovered, Ultra-Rare Disease
Caleb Bupp, pediatric geneticist at Corewell Health Helen DeVos Childrenโs Hospital in Grand Rapids, discusses the discovery of the ultra-rare neurodevelopmental condition known as Bachmann-Bupp syndr...
Caleb Bupp, pediatric geneticist at @corewellhealth.bsky.social discusses the discovery of the condition known as Bachmann-Bupp syndrome, how he and colleagues identified a potential treatment in an existing drug for chronic parasitic disease, and the path forward.
globalgenes.org/raredaily/fi...
22.01.2026 15:09 โ
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Faraz Choudhury of Immuto Scientific discusses the companyโs AI-enabled structural surfaceomics platform, how it allows drugs to selectively home in on diseased cells while sparing normal ones, and its plans to extend its science beyond cancer.
thebioreport.podbean.com/e/finding-ne...
21.01.2026 16:01 โ
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One Rare Motherโs Quest to Rewrite Her Sonโs Future with a Gene Therapy
Amber Freed, founder and CEO of SLC6A1 Connect, discusses how as a parent with no scientific background she catalyzed the development of the first experimental gene therapy for SLC6A1-related disorder...
Amber Freed of Slc6a1 Connect, discusses how she catalyzed the development of the first experimental gene therapy for SLC6A1-related disorder, mobilized scientists, and what other rare disease communities can learn from her journey. @globalgenes.bsky.social #RARECast
globalgenes.org/raredaily/on...
16.01.2026 15:52 โ
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Marc Iacobucci of NanOlogy discusses how the companyโs platform technology enables high-concentration dosing inside tumors that destroys cancer cells, stimulates immune responses, and spares patients the debilitating effects of systemic chemotherapy.
thebioreport.podbean.com/e/targeting-...
14.01.2026 16:58 โ
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Bio Usawa Receives Ghana FDA Approval for BioUcentaโข
Ghana follows Rwanda in approving BioUcentaโข, Africaโs first approved ranibizumab biosimilar...
BioUsawa announced a regulatory milestone in its mission to democratize access to high-quality, affordable biologic therapies across Africa with approval in Ghana of biosimilar to Lucentis.
www.globenewswire.com/news-release...
13.01.2026 19:10 โ
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Transforming TSC Epilepsy with a Precision Therapy
Allison Hulme, CEO of Aeovian Pharmaceuticals, discusses tuberous sclerosis complex, the problems with existing therapies for seizures related to the condition, and the opportunity for its next-genera...
Allison Hulme, CEO of Aeovian Pharmaceuticals, discusses tuberous sclerosis complex, the problems with existing therapies; and the opportunity for its next-generation, selective mTOR inhibitors to treat TSC and beyond. @globalgenes.bsky.social #RARECast
globalgenes.org/raredaily/tr...
09.01.2026 17:39 โ
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Valerie Daggett, CEO of AltPep, discusses the relationship between ฮฑ-sheet oligomers and the onset of Alzheimerโs disease, the companyโs synthetic peptides to target them, and the potential for its platform to treat a broader set of amyloid diseases.
thebioreport.podbean.com/e/an-effort-...
07.01.2026 18:15 โ
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Matt Wood @oxfordharrington.bsky.social discusses the challenges of rare disease drug development, the resources the center brings to address them, and the mechanisms it has established to accelerate therapeutic development. @globalgenes.bsky.social #RARECast
globalgenes.org?post_type=ra...
05.01.2026 16:30 โ
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Adam Feuerstein, senior biotech writer for STAT, joins us for our annual review-preview edition to discuss the noteworthy trends in the biotech sector in 2025, and whatโs in store at the upcoming JPMorgan Healthcare Conference and beyond in 2026.
thebioreport.podbean.com/e/the-year-i...
31.12.2025 16:37 โ
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Turning a Rare Cancer into a Call to Action
Floyd and Monique Stewart discuss how they navigated the healthcare system after Floyd's diagnosis with a rare cancer, the strain the experience put on their family, and why today they are rare diseas...
Floyd and Monique Stewart discuss navigating the healthcare system with a rare cancer diagnosis, the strain the experience put on their family, and why today they are rare disease advocates for families dealing with similar problems. @globalgenes.bsky.social #RARECast
globalgenes.org/raredaily/tu...
26.12.2025 16:16 โ
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Adam Gridley, CEO of Allay Therapeutics, discusses post-surgical pain, the need for alternatives to opioids, and the companyโs experimental, implanted, extended-release analgesic.
thebioreport.podbean.com/e/developing...
24.12.2025 17:01 โ
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Developing a New Approach to Cystic Fibrosis to Improve Outcomes
Mike Cloonan, CEO of Sionna, discusses its experimental therapy that stabilizes the misfolded protein at the heart of the disease, its exploration of combination therapies with existing and proprietar...
Mike Cloonan, CEO of Sionna, discusses the companyโs experimental therapy that stabilizes the misfolded protein at the heart of cystic fibrosis, its exploration of combinations with existing and proprietary therapies, and its $219M IPO. @globalgenes.bsky.social
globalgenes.org/raredaily/de...
19.12.2025 16:38 โ
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Kang Hsu, CMO of Canary Speech, discusses how its diagnostic tool works to diagnose neurological and psychiatric conditions using vocal biomarkers, how it is embedded invisibly into clinical workflows, and how the company is driving physician adoption.
thebioreport.podbean.com/e/leveraging...
17.12.2025 15:28 โ
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Tim Sweeney, CEO of Inflammatix, Inc., discusses the companyโs point-of-care test for sepsis, how it works; and how it is used in an ER setting to determine who needs antibiotics, ICUโlevel care, or a broader diagnostic workup.โ
thebioreport.podbean.com/e/determinin...
10.12.2025 15:07 โ
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Developing Targeted Therapies for a Rare Autoimmune Disease
Ben Zimmer, CEO of Priovant, discusses the company's experimental targeted therapy to treat the rare autoimmune condition dermatomyositis, how its relationship with its parent Roivant frees it from re...
Benjamin Zimmer, CEO of Priovant, discusses the company's experimental targeted therapy to treat the rare autoimmune condition dermatomyositis and how its relationship with its parent Roivant frees it from reliance on the capital markets. @globalgenes.bsky.social
globalgenes.org/raredaily/de...
05.12.2025 15:53 โ
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Atta Behfar, co-founder and CEO of Rion, discusses how the companyโs purified exosome products work, their potential as scalable and cost-effective regenerative therapies, and how they avoid the immune issues that have long hampered cell-based approaches.
thebioreport.podbean.com/e/separating...
03.12.2025 15:32 โ
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