BiotechTV - News

Co-Founder, President & CEO Greg Verdine describes the toxicity issues associated with the immuosupressive drugs that transplant recipients currently receive, and explains how LIFE-001 is different. Plus, given how the therapy is designed to safely prevent T-cell activation and proliferation, there could also ultimately be an autoimmune use case as well.

Dr. Berger, who founded Ariad Pharmaceuticals here in 1991, describes Kendall's transition from candy factories, to Polaroid, to what it is today.

Jim Olaghere describes signing up for a gene editing treatment clinical trial for sickle cell disease and how it has enabled him to climb mountains and Bob Coughlin recounts the moment when a therapy became available for his son's cystic fibrosis.

Alexis Borisy recounts the story of Revolution Medicines, which delivered the pancreatic cancer breakthrough this year, and talks about the health of the founder led side of the biotech sector. Mathai Mammen recently took Parabilis public in the largest IPO in biotech history, and describes the science that made it possible.

He describes how science 'keeps you young' because it always based on what you don't know. He urges scientists to stay humble when talking about science to the public because things can change, and he shares stories from his career and offers advice on how to keep the United States globally competitive.

Interviewed by HSBC Innovation Banking's Katherine Andersen, Najat Khan discusses AI, her priorities upon taking the role of CEO at Recursion earlier this year, encouraging employees to speak more than just one language of expertise, and women in leadership.

Moderated by BiotechTV's Chloe Kirk, Gabriel Astorga (@GoodOlGab), Maynard Okereke (@HipHopScienceShow), and Alex Dainis (@AlexDainis) describe how the got started posting about science on social media and how they grew their audiences to what they are today. Plus, advice for companies and scientists about how to resonate the best.

The combined following of the group reaches approximately 3 million people on Instagram and TikTok.

He describes the format and goals of the event.

Moderated by MassBio CEO Kendalle Burlin O'Connel, Kymera Therapeutics CEO Nello Mainolfi, CRISPR Therapeutics CEO Samarth Kulkarni, and Ginkgo Bioworks CEO Jason Kelly discuss their companies' innovations (protein degradation, CRISPR gene editing, and autonomous labs), how the U.S. can increase its competitiveness, and the 'Why' that motivates them each day. 

Dr. Zerhouni, the Founder and Chair of ModeX Therapeutics and Former NIH Director, talks about investments in science that started with Vannevar Bush, current day NIH funding, adding a political element to the grant making process, science communication, and more.

The lead two programs, which target TLR7/8 and PARP1, have data at ASCO and were licensed with the idea of laying the foundation for clinical expertise at Eikon. Another clinical program, a WRN helicase inhibitor, is the first to entirely come out of Eikon's platform. Plus, Dr. Perlmutter's take on being a public company, and his thoughts on China as a medicine cabinet to the world.

Dr. Mark Goldsmith of Revolution Medicines describes the benefit seen by advanced patients who received the pancreatic cancer pill daraxonrasib vs those in a control group of investigator's choice standard-of-care chemotherapy, and he discusses the medicine's side effect profile. Plus, next steps for this treatment in pancreatic cancer and beyond.

Co-Founder & CEO Natalie Mount discusses the raise, and explains why she believes gamma delta T-cell engagers have advantages over CD3s. After presenting preclinical data at the recent AACR annual meeting, the lead EGFR program is preparing to go into the clinic.

CEO Abe Ceesay describes Rapport's precision neuroscience approach of targeting the AMPA receptor specifically through TARPγ8, and explains why the company took the extra step of tracking an electrographic biomarker in its phase 2a study in addition to seizure frequency. Plus, next steps and additional indications.

Philip Astley-Starke describes the timeline of development of this oncolytic immunotherapy, his impression of FDA's review process and decisions around it, and the status of where the treatment stands today.

CSO Robert Allen describes Invivyd's approach and why antibodies can be a good approach, especially as it relates to being resistant to viral evolution. He walks us through the science and some potential use cases.

Co-Founder & CEO Michel Maharbiz describes the idea behind this brain-computer interface (BCI), which is not yet in human trials. While cautioning that much work and learning lies ahead, he sees the possibility that such a system could also improve a patient's rehab and might potentially have further uses in diseases of cognitive decline.

He describes his work in tropical disease, and how climate change has been elevating it in areas like the Gulf Coast of the United States. Plus, the importance of elevating scientists, what he has found works best for communicating with the public, and why he disagrees with the idea of 'hunkering down' until a day that science funding might rebound in the U.S..

He shares his thoughts on the data, the competitive profile of this IL-2 Treg therapy vs JAK inhibitors, and how the overall platform looks for other I&I conditions.

Co-Founder and CEO Serge Saxonov walks us through the platform, which can process up to 800 whole-transcriptome samples per year (1 cm² each) on a single instrument, scaling to thousands of samples per year for targeted assays, and has single-cell level sensitivity.

Plus, discussions on key trial readouts outside of obesity: eplontersen, Lp(a), oral IL-23, and milvexian.

He discusses Praxis, Insmed, Ionis, PTC Therapeutics, Rapport, and United Therapeutics.

After experiencing a setback last year with Korro's LNP delivered alpha-1 antitrypsin deficiency program, he describes learnings from that experience and how Korro has new momentum with a galNAc delivered approach targeting urea cycle disorders, hepatic encephalophy, and new alpha-1 program. The company recently closed a $85 private placement.

He describes both the biomarker and functional benefits seen in the December data for the company's next-generation exon 51 skipping therapy.

He reminds us of the unmet need for this type of therapy and discusses the planning that Vera has been doing ahead of the decision date.

He describes physician, patient, and payor feedback from this closely watched launch. Plus, praliciguat, and why Akebia bought a targeted complement inhibitor from Q32 Bio.

He discusses the INHBE data and the market reaction to it, which he believes is due to misconceptions about the patient population, and the next steps for this program. Plus, an AATD presentation that will happen next month at American Thoracic Society.

CEO Alice Reicin walks us through recent data and guides for what is next in this patient population. Plus, an upcoming healthily volunteer readout for a HHT program and how it might translate to patients in future trials.

CEO James Helliwell describes Eupraxia's platform and how it differs from other extended release technologies. Plus, Eosinophilic Esophagitis, and how the company thinks about other uses.

Co-Founder and CEO Jeffrey Finer describes how Septerna is able to screen for GPCR targets in ways that have not previously been possible. Plus, an overview of programs targeting MRGPRX2, PTH1R, and a collaboration with Novo on obesity.

He discusses Tyra, Sionna, Alumis, Nuvalent, and Monopar.

CEO Ajit Singh describes the idea behind the new products, and highlights that $100M has been raised to fund the development of them.

CEO Cameron Turtle walks us through the data, shares his opinion on its translatability to part B of the current study, and tells us what else is ahead for Spyre.

He discusses the Biogen deal, and how it is a sign of increased activity Alloy sees in their genetic medicines business. Plus, monoclonals vs multispecifics, and the state of discovery work today.

He describes how Stoke aims to up-regulate healthy genes in cases where diseases are caused by autosomal dominant haploinsufficiencies, like Dravet. He discusses the phase 1/2 data, a phase 3 that is scheduled to read out in 2027, and the regulatory history of the program.

He describes the multiple variables that factor in to designing new medicines with AI, how Iambic's platform has allowed it to shorten the time to get a medicine into the clinic versus traditional discovery work, and how he thinks AI will augment the work of human scientists.

He explains how Chai-2 works, and how it compares to other antibody building / binding AI models.

The first patient was dosed in DOMMINO-1, a Phase 2 open-label, single-arm study enrolling 100 adult patients across clinical sites in the UK and U.S.. It could potentially offer an accelerated approval opportunity if it reaches a high enough response rate.

He describes the science behind Coya's combination therapies, and programs targeting ALS (currently in a phase 2 study), FTD, and Alzheimer's.

He discusses Insmed, IDEAYA, Bicycle, Compass Pathways, Revolution Medicines, Cytokinetics, and CG Oncology

He describes data presented at the Muscular Dystophy Association Annual Meeting which showed biopsy and safety data for 20 patients who have been followed for 90 days and 3 who are out to a year. Plus, explaining the three FDA meetings that are key ffor the company.

Michal Preminger will lead BioMed X Venture labs as CEO, and it will partner with pharma, foundations, and VCs to build mission driven companies.

He discusses the the data and how he compares it to Dupixent and other medicines on the market, how translatable the data might be to a phase 3 that will be up and running later this year, and what other data the company will have in the near future.

MassBio President & CEO Kendalle Burlin O'Connell moderates a discussion with Zag Bio CEO Jason Cole and Altido Therapeutics CEO Chip Clark about running early stage companies and what advice they have for new CEOs. They offer advice about what it will take to keep our ecosystem globally competitive.

From Syncona's Capital Markets Day, CEO Jim MacDonald describes the learnings Yellowstone has gained from looking at cured AML patients from the Oxford biobank and how it is translating that into new medicines.

From Syncona's Capital Markets Day, he describes Slingshot and how he believes it differs from other early stage VC investors. Once an asset is ready for the clinic, Slingshot will either partner it or build its own company.

From Syncona's Capital Markets Day, CEO Haseeb Ahmed describes the science behind the platform, and make the case why IgAN, a condition that has seen a bevy of new therapies in development, is the right indication for a gene therapy.

CEO Amir Hefni describes the ability of macrophages to reduce inflammation and reverse fibrosis in conditions like liver disease. An interim analysis for the clinical trial will read later this year.

From Syncona's Capital Markets Day, ALTx founder and Francis Crick researcher Simon Boulton describes ALTs and the science behind how the company plans to target them.