BiotechTV - News

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 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.

From Syncona's Capital Markets Day, Founder and CEO Catherine Pickering walks us through the science of roginolisib and describes the current landscape for uveal melanoma. She discusses her phase 2, which is approaching a key readout.

CEO Michael Parini describes the science behind how Spur's gene therapies have two key differences compared to others, including a proprietary capsid. He discusses phase 1/2 data that has already been announced, and how he thinks about the phase 3.

From Syncona's Capital Markets Day, CEO Lance Baldo describes the trial's design and the degree to which Beacon will be trying to restore vision for these patients.

He talks about Syncona's listed fund, and clarifies the details of a return of capital strategy for some shareholders, and he describes the strategy of the firm's new private fund. Plus, how Syncona thinks about opportunities in life sciences today.

He his take on topics such as how the Baby KJ news is allowing investors and companies to think about scaling bespoke therapies, how AI might start to impact the clinical trial side of our business, and why he sees pharma adoption of AI as being important.

Co-Founder & CEO Cyriac Roeding and Co-Founder & CSO David Suhy describe the science behind this approach and explain how AI is helping they move closer to the clinic. The first IND filing is scheduled to be late '26 or early '27.

He describes learnings from Afrezza's commercialization, why the company acquired scPharmaceuticals, an update on their partnership with United Therapeutics in PAH, and a key IPF asset that is in the pipeline.

He comments on Abivax, Corvus, Iovance, Legend, Terns, and more.

He argues that investors are too focused on each percentage of weight loss and not focused enough on the tolerability of new options. He discusses the timeline for the phase 3 that will likely start later this year, and how it might look different than the phase 2 study that just read out.

Maneet Singh says that pharma companies need to keep buying, and have the financial wherewithal to do it. He also gives thoughts on which areas within biotech could attract the most interest.

CEO Greg Mayes describes the science behind luvesilocin, which is a prodrug of 4-OH-DiPT, a psilocybin-like compound, and discusses the company's plans for a phase 3 study. Plus, comments on two other indications the company is pursuing.

He gives his take on two questions being discussed the most: 1) is AXPAXLI approvable on one study and 2) how might physicians think about using it in practice.

He highlights pipeline programs targeting ENPP3, CLDN6, CLDN18.2, TL1A (including TL1A x IL23p19), CD20 x CD3, and CD19xCD3.