Methylmalonic Acidemia

Six months after launching with a mega-round of financing, Seaport Therapeutics has raised $225 million more to advance a lead drug candidate that could overcome some limitations of currently available treatments for depression. Seaport’s platform technology, called Glyph, designs oral drugs that leverage the lymphatic system to deliver the medicine in a way that makes more of the active pharmaceutical ingredient available in the body to confer its therapeutic effect. The Boston-based company also says its drugs reduce complications such as elevated liver enzyme levels, a sign of drug toxicity that can limit the use of some molecules. Lead Seaport program SPT-300 is a prodrug, a compound that converts inside the body into the active drug allopregnanolone. This neurosteroid modulates the GABA-A receptor to spark effects on the central nervous system. Seaport is advancing the drug into Phase 2b testing as a potential treatment for major depressive disorder with or without anxious distress. Seaport says the new capital will go toward advancing its pipeline through important clinical milestones. The company also plans to further advance the capabilities of its Glyph technology platform. presented by Health IT Accelerating Claim Processing: Strategies to Shorten the Life of a Claim These strategies and practices can significantly shorten the life cycle of claims, leading to quicker resolutions and improved financial outcomes. By Greenway Health Seaport was spun out of PureTech Health, a company that forms startups around platform technologies. In April, Seaport launched, revealing its science, its lead drug candidate, and $100 million in funding. That Series A round was led by Arch Venture Partners and Sofinnova Investments. General Atlantic led Seaport’s new Series B financing. Other participants in the latest round included T. Rowe Price Associates, Foresite Capital, Invus, Goldman Sachs Alternatives, CPP Investments, and other unnamed new investors. Founding investors Arch and Sofinnova also joined the round, as did Third Rock Ventures and PureTech Health. Seaport is part of a bevy of life science companies that have secured new capital. Here’s a recap of recent biotech financing news: —Radiopharmaceuticals developer Alpha-9 Oncology closed a $175 million Series C round to support its pipeline, including A9-3202, which began a Phase 1 test earlier this year in advanced melanoma. Lightspeed Venture Partners and Ascenta Capital led Alpha-9’s new financing. —AvenCell Therapeutics has $112 million in Series B funding to continue Phase 1/2 testing of AVC-101, a CAR T-therapy that can be switched “on” or “off” to address safety and efficacy challenges of currently available cell therapies, which are always “on.” AvenCell was formed in 2021 by Intellia Therapeutics and Cellex Cell Professionals. The company’s latest financing was led by Novo Holdings. Sponsored Post The Future of Hospitals and Pharma Companies Will Depend on Strength of Healthcare Analytics Insights PurpleLab® stands out from others in this sector by providing its data analytics services to several different groups of users across healthcare and pharma companies. By Stephanie Baum —Tolerance Bio launched with $17.2 million to support development of allogeneic stem cell-based cell therapies. These therapies are intended to preserve the thymus, the organ that trains T cells to defend against threats and preserve autoimmunity. The Philadelphia-based biotech’s seed financing, led by Columbus Venture Partners, will be used to advance toward the clinic with potential therapies for immune diseases. —March Biosciences, spun out of the Center for Cell and Gene Therapy, unveiled $28.4 million in Series A financing. Lead program MB-105 is a CAR T-therapy designed to target CD5, a protein expressed on cancerous T cells. A Phase 1 test is underway enrolling patients with refractory T cell lymphoma and leukemia. Mission BioCapital and 4BIO Capital led March Bio’s financing. —Be Biopharma closed $82 million in financing as the cell therapy developer moves into the clinic with BE-101, a potential treatment for hemophilia B. The biotech’s therapies are made by engineering B cells to produce therapeutic proteins. Be Bio last raised money in 2022, a Series B financing that totaled $130 million. —Terray Therapeutics, a biotech that uses generative AI to discover and develop small molecule drugs, unveiled a $120 million Series B financing. Los Angeles-based Terray will use the capital to advance internal programs into clinical trials for indications that were not disclosed. The financing was led by new investor Bedford Ridge Capital and earlier investor NVenture, the investment arm of NVIDIA. Here’s more about Terray and its technology platform, tNova, which it unveiled in 2022. —Genetic medicines have been difficult to deliver to the kidney. Judo Bio is developing oligonucleotide drugs that target a particular receptor to reach the organ, and the Cambridge-based startup unveiled $100 million in seed and Series A financing to advance its research toward the clinic. The Series A round was co-led by Atlas Venture, TCG, and Droia Venture. —Purespring Therapeutics, a developer of gene therapies for kidney disease, raised £80 million (about $105 million). The London-based company’s therapies target the podocyte, a special cell implicated in 60% of renal diseases. Proceeds will be used to advance lead program PS-002 into a Phase 1/2 clinical trial for immunoglobulin A nephropathy, a rare disease currently treated with chronically administered therapies. Sofinnova Partners led Purespring’s Series B financing. —Artificial intelligence biotech company Basecamp Research revealed a $60 million Series B financing to scale its collection of data and strengthen its AI capabilities. The London-based startup also announced a multi-year collaboration with David Liu of the Broad Institute of MIT and Harvard. Basecamp said this partnership will research new approaches to programmable genetic medicines with potential applications in a range of diseases. —City Therapeutics launched with $135 million to develop next-generation RNA interference (RNAi) drugs. The company says its engineering platform can design therapies in smaller sizes that have novel cellular mechanisms and improved potency, efficiency, and specificity. The Cambridge-based biotech, co-founded by scientists involved in the first generation of RNAi therapies, expects to enter the clinic by the end of 2025. Arch Venture Partners led City’s Series A round. —Resolution Therapeutics raised £63.5 million to advance to Phase 1/2 testing with RTX001, an engineered autologous macrophage cell therapy that could offer anti-fibrotic and anti-inflammatory effects in patients with end-stage liver disease. The company, which maintains operations in London and Edinburgh, Scotland, said this trial will be conducted in the United Kingdom and Spain. Syncona led the Series B round, which will also support research in other inflammatory fibrotic conditions such as graft-versus-host disease and lung fibrosis. —Triveni Bio raised $152 million to support a pipeline whose most advanced program, TRIV-573, is a bispecific antibody that blocks two targets associated with atopic dermatitis. The Watertown, Massachusetts-based biotech aims to file an investigational new drug application for this program in the first quarter of 2025. Triveni launched last year supported by a $92 million Series A financing. Goldman Sachs Alternatives led the company’s Series B. —Kailera Therapeutics launched with $400 million to support its GLP-1 and GIP receptor agonist drugs for metabolic indications, including obesity. Kailera’s four programs were licensed from China-based Jiangsu Hengrui Pharmaceuticals. The Series A financing was co-led by Atlas Venture, Bain Capital Life Sciences, and RTW Investments. —Radiopharmaceutical developer Aktis Oncology unveiled $175 million in financing to support development of a pipeline led by AKY-1189, an alpha radiotherapy that targets Nectin-4, a tumor-associated antigen found in a range of solid tumors. The Series B round was led by RA Capital Management and co-led by RTW Investments and Janus Henderson Investors. Boston-based Aktis emerged from stealth in 2021 backed by $72 million in Series A financing. —Genespire raised €46.6 million (about $52 million) to advance to Phase 1/2 testing with GENE202, an in vivo gene therapy in development for methylmalonic acidemia, a rare inherited disorder that impairs the metabolism of certain amino acids and fats. The Milan, Italy-based startup’s Series B round was co-led by Sofinnova Partners, XGEN Venture, and CDP Venture Capital. —Vaccines developer Vicebio unveiled $100 million in financing as it begins a Phase 1 test of VXB-241, a bivalent vaccine designed to address both respiratory syncytial virus (RSV) )and human metapneumovirus (hMPV). Data are expected in mid-2025. The next Vicebio program is VXB-251, a trivalent vaccine for RSV, hMPV, and parainfluenza virus 3. The London-based company’s Series B round was led by TCGX. —GC Therapeutics launched with $65 million in financing. The biotech’s next-generation cell therapies come from a platform technology called TFome, which programs induced pluripotent stem cells. GC’s initial therapeutic areas of focus include gastrointestinal, neurological, and immunological diseases. Cormorant Asset Management led Cambridge, Massachusetts-based biotech’s Series A financing. —Neuroscience startup Nura Bio extended its Series A financing by $68 million to proceed to Phase 2 testing with an oral small molecule designed to block SARM1, an enzyme associated with axonal damage. It faces potential competition from Eli Lilly, which is in early clinical development with a drug that goes after the same target. Nura Bio’s founding investor The Column Group led the startup’s new financing. —F2G raised $100 million for olorofim, which is in late-stage development to treat aspergillosis and other invasive fungal infections. Olorofim is from a novel class of antifungal agents that work by inhibiting the pyrimidine synthesis pathway. The cash will enable the Manchester, U.K.-based company to complete late-stage clinical development, submit regulatory applications, and prepare for U.S. commercialization of the drug. The new financing was led by new investor AMR Action Fund and co-led by ICG. —T cell engagers first reached patients as cancer treatments. Candid Therapeutics aims to bring such drugs to autoimmune disorders, and its efforts are backed by more than $370 million in financing. Candid’s drug candidates came from acquiring and merging two biotechs, Vignette Bio and TRC 2004. The Candid pipeline includes two T cell engagers for cancer now being repositioned for development in autoimmune diseases. These therapies are designed to selectively deplete B cells that drive disease. —Startup creator Flagship Pioneering unveiled its newest company, Mirai Bio. Mirai’s platform technology is applied to the development of genetic medicines for the company’s partners working across a range of therapeutic areas and therapeutic modalities. It says its platform technology unlocks delivery of a drug to any tissue and cell type, optimize design of the drug cargo, and facilitates manufacturing. Like other startups that come from Flagship, Mirai is backed by the customary $50 million in financing. —Superluminal Medicines, a company developing small molecule drugs that target elusive G protein-coupled receptors (GPCRs), closed $120 million in financing. The Boston-based startup says its drugs target membrane receptors that mediate cell signaling and response processes fundamental to human physiology. These small molecule come from a generative biology and chemistry platform technology. The new capital will be used to advance toward the clinic in undisclosed indications. RA Capital Management led the Series A financing. —Riding strong investor interest in metabolic disorder drugs, OrsoBio closed $67 million to advance a pipeline that includes programs for type 2 diabetes, obesity, and the fatty liver disease MASH. The Menlo Park, California-based startup’s drugs address pathways associated with energy metabolism. The Series B round, led by Ascenta Capital and Woodline Partners, comes less than a year after the company unveiled $60 million in Series A financing. Photo by Flickr user Massachusetts Office of Travel & Tourism via a Creative Commons license

Genespire patched together a €46.6 million (about $52 million) funding round to get its lead gene therapy into the clinic in a few years. The Milan-based biotech, which employs nine people, said Wednesday that the Series B will fuel its mission to create a lentiviral vector-based gene therapy for children with methylmalonic acidemia, a rare genetic disease marked by disruptions to the breakdown of certain fats and amino acids. Also known as MMA, it can lead to muscular weakness, seizures, developmental delays, organ damage and even death. Other gene therapy startups have attempted but then abandoned efforts to develop treatments for the disease, including AstraZeneca’s LogicBio and Selecta Biosciences . There are no disease-modifying drugs, and treatment is currently addressed by diet modifications or sometimes liver transplants. Genespire’s work is based on research out of the San Raffaele Telethon Institute for Gene Therapy. The four-year-old biotech recently recruited former Lysogene CEO Karen Aiach-Pignet as its new chief executive after Lysogene shut down. It’s “a new page for me at a time when there have been a lot of ups and downs in the gene therapy space,” she said in an interview. “Probably more downs over the recent years.” Several gene therapy makers, including bluebird bio — which saw its sickle cell gene therapy Lyfgenia get approved last year — are struggling after interest in the once-bustling field has waned amid the broader biotech downturn and questions about the profitability of making these therapies. That said, new biotechs are still emerging, with plans to focus on neurodegenerative and CNS diseases , cancers and other areas. Aiach-Pignet joined Genespire after bringing Lysogene from the lab to Phase 3 with a gene therapy that missed the bar on improving cognitive development in Sanfilippo syndrome type A, a serious inherited neurodegenerative lysosomal storage disorder. Aiach-Pignet said a patient group called Blue Daisy has acquired the program. Sofinnova Partners, which had backed Lysogene, co-led Genespire’s Series B. XGEN Venture, CDP Venture Capital and Indaco Venture Partners SGR are also investors in Genespire. Aiach-Pignet said it is too soon to commit to a time frame for the Phase 1/2 study, though it is likely more than a year away, she said. The company still needs to coordinate the study with regulators and patient advocacy groups, according to the CEO. “Something that I really pay great attention to is the viewpoints, the experience, the expertise of patient groups,” Aiach-Pignet said. “The reason why I wanted to include them in the process is because we need to have a very robust clinical protocol, but we also need to have a protocol that makes sense for the patients and will make sense for the payers down the line.” Moderna is aiming for approval of its own experimental medicine for MMA by 2028. The biotech is in Phase 1/2 with an mRNA infusion treatment candidate known as mRNA-3705. Meanwhile, a BridgeBio subsidiary called CoA Therapeutics terminated a Phase 1 of its oral drug BBP-671 last year. Editor’s note: This story was updated to include additional information on Blue Daisy acquiring Lysogene’s gene therapy.

Genespire’s GENE202 was developed using the company’s Immune Shielded Lentiviral Vector (ISLV) platform that allows a patient’s liver to produce the therapy throughout its lifetime. Image credit: jack8 / Shutterstock. Italian biotech Genspire is set to launch a Phase I/II clinical trial of its lead gene therapy, GENE202, as a treatment for a group of rare, inherited diseases – methylmalonic acidemia. The company will be able to fund the trial after it received €46.6m ($52.1m) in a Series B financing round. The financing will also help to fund discovery and preclinical work for other gene therapy candidates. Investors included European investment firms such as Sofinnova Partners, XGEN Venture and CDP Venture Capital. Genspire is a spinout from Milan-based San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), and has previously raised €16m in Series A financing in 2020. Methylmalonic acidaemia is a group of rare genetic disorders where an individual is unable to metabolise proteins and fats properly. This results in high levels of acid in the blood (acidaemia) and body tissues. Methylmalonic acidaemia often manifests within the first few months of life and its severity can vary from mild disease to life-threatening. According to the US National Organization for Rare Disorders (NORD), methylmalonic acidaemia usually affects 1 in 50,000 to 1 in 100,000 live births. The disease is usually managed through dietary modifications to limit certain proteins and fats. See Also: Biologic API Manufacturing Companies (Protein and Peptide) in Contract Manufacturing for the Pharmaceutical Industry Bavarian Nordic gains US contract for additional mpox vaccine GENE202 is an off-the-shelf, one time gene therapy developed using the company’s Immune Shielded Lentiviral Vector (ISLV) platform. ISLV-based therapies are designed to allow a patient’s liver to produce the therapy throughout its lifetime. Gene therapies are a growing area of interest , with multiple gene therapies gaining approval from the US Food and Drug Administration (FDA) in recent years. In recent months, multiple companies have entered the gene therapy market by acquiring or merging with specialist companies and pipelines. Last year, Alexion AstraZeneca Rare Disease signed a definitive agreement, acquiring and licensing Pfizer ’s early-stage gene therapy portfolio for rare diseases . The deal included a $1bn payment along with net sale royalties from the acquired therapies. Despite the recent interest, the uptake of gene therapies is limited due to their high price tags . Furthermore, not all gene therapies make it to market, especially for rare diseases, as their high cost and limited commercial market can deter companies from investing. In some cases, academic institutes and non-profit organisations have ‘rescued’ these gene therapy programmes. Another therapy in development for methylmalonic acidaemia is Moderna ’s mRNA therapy, mRNA-3705. The therapy is being investigated in the Phase I/II Landmark study (NCT04899310) as a treatment for a specific type of methylmalonic acidaemia, one due to CoA mutase (MUT) deficiency. In June 2024, mRNA-3705 was included in a Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot programme by the US Food and Drug Administration (FDA). The START programme is aimed at expediting the development of novel drugs and biological products for rare diseases. 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. Thank you. You will receive an email shortly. Please check your inbox to download the Whitepaper. By Cytiva Thematic By downloading this Whitepaper, 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