Roche Pharma AG

BioMarin will cut about 225 jobs on top of a previous round of layoffs affecting about 170 employees. The rightsizing at BioMarin continues to take its toll as CEO Alexander Hardy devises a new corporate strategy for the rare disease biotech.BioMarin plans to cut about 225 employees, or about 7% of its global workforce, the California biotech said in a securities filing (PDF) Wednesday.The latest reductions are separate from a previous set of layoffs affecting about 170 employees announced in May, a BioMarin spokesperson confirmed to Fierce Pharma. Employees impacted by the latest initiative were notified by Wednesday, and both rounds are expected to be “substantially completed” by the end of this year, according to the company.BioMarin chalked up the new cuts to “organizational redesign efforts” as well as the company’s narrowed focus for the hemophilia A gene therapy Roctavian and its decision to discontinue the development of BMN 293, a preclinical gene therapy for a genetically driven subtype of hypertrophic cardiomyopathy. The latter two moves were previously announced alongside BioMarin’s second-quarter report Aug. 5.“BioMarin is in the process of developing a new corporate strategy and a critical component of delivering on this strategy is to ensure that BioMarin is structured in a way that will position the company for continued success in the future,” the company spokesperson said in a statement. “As part of the new strategy, and because of changes related to our pipeline and business priorities going forward, our workforce was impacted.”The strategic rethink started as BioMarin brought on former Genentech CEO Hardy as its helmsman in late 2023. The company has yet to systematically share the new blueprint, and analysts are looking to an investor event that’s scheduled for Sept. 4 to gain more clarity.  Several products have already felt the impact from the revamp. The previous layoff round targeting 170 positions were associated with BioMarin’s termination of four candidates. Rather than a global launch, BioMarin has decided to focus Roctavian on its three existing territories where the gene therapy has secured reimbursement policies—the U.S., Germany and Italy.Amid the strategic review and pipeline makeover, BioMarin has also shuffled its executive team. The company is bringing on Amgen veteran Greg Friberg, M.D., to replace the retiring Hank Fuchs, M.D., as the new R&D chief. One of Hardy’s former colleagues at Roche, dealmaking expert James Sabry, M.D., Ph.D., will also join as chief business officer. Cristin Hubbard, who was Roche Pharma’s head of global product strategy, took on BioMarin’s chief commercial officer role in May.The BioMarin spokesperson didn’t specify whether the 225 layoffs involve any managerial roles. The company expects employee severance in this round will cost about $30 million to $35 million, of which the majority is likely to be incurred this year. The downsizing at BioMarin adds to a broader struggle among gene therapy developers. For Roctavian, BioMarin has to work through reimbursement hurdles and patients’ hesitance, especially when other effective therapies exist. The requirement to establish additional infrastructure to administer the one-time drug has also been an obstacle.CSL recently admitted that uptake of its hemophilia B gene therapy, Hemgenix, has been slower than expected, which the company blamed on the fragmented U.S. healthcare system.  Earlier this month, bluebird bio narrowed its 2024 expectations for its sickle cell disease gene therapy Lyfgenia to the lower end of its prior projection after slower-than-expected patient onboarding. As one of the earliest gene therapy players, bluebird had previously made some difficult decisions in Europe and has been relying on outside loans to stay afloat.In July, Pfizer unveiled a plan to cut 150 jobs at its dedicated gene therapy facility in North Carolina following the high-profile failure of—and the decision to discontinue—its Duchenne muscular dystrophy candidate.Last week, gene editor Tome Biosciences announced that it will disband nearly its entire workforce despite having raised $213 million in a combined series A and B round. In explaining the decision, the Massachusetts startup cited a dramatic shift in investor sentiment across the gene editing space, especially for preclinical companies. 

GenEdit is part of a wave of biotechs creating non-viral methods of gene therapy delivery. Image credit: Shutterstock/Vink Fan. Roche’s Genentech has partnered with biotech GenEdit to use the latter company’s non-viral delivery platform for gene editing therapies. Genentech will pay GenEdit $15m upfront as part of the multiyear collaboration and licence agreement. GenEdit is also in line for a further $629m if it meets certain milestones, according to a 23 January press release. Genentech has picked out GenEdit’s NanoGalaxy platform to advance its autoimmune pipeline. The platform uses non-viral, non-lipid hydrophilic nanoparticles (HNPs) for targeted in vivo delivery. As per the agreement, the two companies will seek out and develop HNPs that can be used to develop nucleic acid-based medicines for autoimmune indications. GenEdit will be tasked with discovering potential nanoparticle candidates while Genentech will oversee clinical and regulatory development, along with commercialisation duties. Roche Pharma Partnering’s global head James Sabry said: “Our collaboration with GenEdit will use their polymer nanoparticle library and platform with the potential to reshape treatment paradigms to help people with devastating and difficult-to-treat autoimmune diseases.” GenEdit was cofounded by CEO Kunwoo Lee in 2016. Lee previously worked with CRISPR pioneer and Nobel Prize winner Jennifer Doudna during his time at the University of California, Berkeley. Lee said: “This collaboration underscores the promise of GenEdit’s NanoGalaxy platform to deliver nucleic acid-based medicines via our hydrophilic nanoparticle technology.” GenEdit is part of a wave of biotechs creating non-viral methods of gene therapy delivery .  An adeno-associated virus (AAV) vector is one of the leading ways of delivering gene therapies, but with its widening use has come safety and durability concerns. In September 2021, the US Food and Drug Administration (FDA) held an advisory committee meeting to discuss the flagged issues. GenEdit aims to overcome the limitations and safety concerns of current approaches. It says its non-viral polymer nanoparticles offer payload flexibility and tissue selectivity, and the ability to re-dose while maintaining low immunogenicity. Genentech is not the only big pharma that has been attracted by GenEdit. Eli Lilly backed the biotech as part of a $26m Series A financing round in September 2021. In February 2022, Sarepta partnered with GenEdit in a $57m deal. The gene therapy maker combined its know-how with GenEdit’s NanoGalaxy platform for use in up to four neuromuscular indications. Earlier this week, Genentech returned global rights of two Alzheimer’s disease drug candidates to AC Immune following clinical failures. The termination of the agreement brought an 18-year relationship between the two companies to an end. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Cell and gene therapies: Pipe dream to pipeline The cell and gene industry is gaining momentum, with a new wave of therapies promising to transform the way doctors treat, and even cure, disease. In this report, Cytiva and GlobalData have collaborated to explore the rise of the cell and gene therapy industries, the current state of the market, present and future opportunities for advancement, and the challenges that lie ahead. By Cytiva Thematic By downloading this case study, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy

Remix to leverage REMaster™ platform to discover novel drugs for a diverse range of target classes Remix will receive $30 million upfront and is eligible to receive up to $12 million in near-term milestone payments and up to $1 billion in milestone payments and royalties WATERTOWN, Mass., Jan. 3, 2024 /PRNewswire/ -- Remix Therapeutics (Remix), a clinical stage biotechnology company developing small molecule therapies to modulate RNA processing and address underlying drivers of disease, today announced a collaboration and license agreement with Roche (SIX: RO, ROG; OTCQX: RHHBY) for the discovery and development of small molecule therapeutics that modulate RNA processing using Remix's REMaster drug discovery platform. Under the terms of the agreement, Remix will receive an upfront payment of $30 million and are eligible to receive up to $12 million in near-term milestone payments, as well as preclinical, clinical, commercial and sales milestones of up to $1 billion and tiered royalties. In exchange, Roche will have exclusive rights to specific targets. Remix will conduct discovery and preclinical activities with Roche, and Roche will be responsible for development and commercialization of any resulting products. "We're excited to partner with a team with strong expertise and dedication to the field of interfering with targets at the RNA level," said Peter Smith, Ph.D., President and Chief Executive Officer of Remix Therapeutics. "Our collaboration with Roche provides an opportunity to further leverage our REMaster drug discovery platform and translate our cutting-edge science into new medicines." "We have been impressed by Remix and their scientific accomplishments with their REMaster drug discovery platform," said James Sabry, Global Head of Roche Pharma Partnering. "This collaboration aligns well with our strategy to join forces with partners who pursue innovative approaches to discover and develop new medicines for patients in areas of high unmet medical need." About Remix Therapeutics Remix Therapeutics is a clinical stage biotechnology company developing novel small molecule therapies designed to reprogram RNA processing and treat disease. The REMaster™ technology platform facilitates RNA processing pattern identification, leveraging these learnings to modulate gene expression. Remix's innovative therapeutic approach has the potential to alter the way genes are read from the genome, to correct, enhance, or eliminate the gene message, thereby addressing disease drivers at their origin. For more information visit . SOURCE Remix Therapeutics