Selecta Biosciences, Inc.

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.

The new funding will be used to take ProGly-Uricase into a phase 1 trial of patients with elevated uric acid levels. GRO Biosciences has ended the week with an extra $60.3 million in the bank, which the protein therapeutics-focused biotech will use to push its lead gout treatment into clinical trials. Gout occurs when high levels of uric acid in the blood cause crystals to form and accumulate in and around a joint, leading to pain and swelling. Horizon Therapeutics’ Krystexxa remains the only approved biologic to treat uncontrolled gout. But some patients who receive this uricase enzyme therapy soon develop anti-drug antibodies (ADAs) that clear out the enzyme, according to GRO Bio. GRO Bio’s pitch is that its own uricase enzyme therapy, ProGly-Uricase, can prevent the emergence of ADAs, allowing patients to keep control of their serum uric acid levels for the long term. The new funding will be used to take ProGly-Uricase into a phase 1 trial of patients with elevated uric acid levels, as well as to “broaden the GRObio pipeline, and to expand its genomically recoded organism (GRO) platform for scalable production of therapeutics,” per the company. The series B was co-led by new investors Atlas Venture and Access Biotechnology, the biopharma investment arm of Access Industries. Atlas partner Kevin Bitterman, Ph.D., and Access Biotechnology’s Managing Director Dan Becker, M.D., Ph.D., both joined GRO Bio’s board as part of the financing arrangements. Previous investors Redmile Group, Digitalis Ventures and Innovation Endeavors were also back for the series B, along with Leaps by Bayer, which led GRO Bio’s $25 million series A in 2021. Altogether, GRO Bio has now raised over $90 million in capital to date, the biotech pointed out. The Cambridge, Massachusetts-based company, which aims to “leverage synthetic biology to expand the amino acid alphabet,” also has plans to use its tech to treat autoimmune diseases without broadly suppressing the immune system by conferring highly specific tolerance to disease-causing autoantigens. “Having proved our therapeutic approach preclinically and demonstrated scalability of our GRO platform, we have assembled the ideal team to advance GRObio to a clinical-stage company,” CEO Dan Mandell, Ph.D., said in the release. “This financing enables us to acquire valuable clinical efficacy data in gout while expanding our platform to demonstrate the first scalable production of proteins with multiple NSAAs, including simultaneous incorporation of drug, immune recruitment, and tissue-targeting payloads,” Mandell added. GRO Bio isn't the only company looking to take on Krystexxa's crown. For example, Selecta Biosciences and Sobi produced phase 3 data last year that suggested their SEL-212 candidate ImmTOR can match the efficacy of Horizon's mainstay, despite being administered less frequently.

Cartesian Therapeutics shared plans to raise $130 million. Cartesian Therapeutics has claimed a phase 2b win for its BCMA-directed CAR-T therapy in generalized myasthenia gravis (gMG). But the biotech’s victory came on a primary endpoint it changed in May and in a population that excluded the 25% of participants treated at community clinics. Investigators randomized 36 patients to receive Descartes-08 or placebo. Cartesian restricted its primary endpoint analysis to 26 people enrolled at academic medical centers who received at least one dose and completed at least one post-baseline assessment of MG Composite (MGC) score. The decision removed nine patients treated at community clinics from the primary analysis. Another subject dropped out. The biotech switched its primary endpoint from the MG-ADL symptom scale to MGC in May. Argnex, Johnson & Johnson and UCB looked at MG-ADL for the primary endpoints of their pivotal gMG studies.  In Cartesian’s primary efficacy population, 10 out of the 14 recipients of Descartes-08 had a five-point or greater improvement in MGC after three months. Three of the 12 participants on placebo had such an improvement. The difference in response rates, 71% versus 25%, was enough for the clinical trial to meet the MGC endpoint in the modified intent-to-treat efficacy population. The biotech said a three-point change in MGC is generally seen as clinically meaningful. Cartesian used the “more stringent endpoint of five points” based on the responses seen in an earlier study. The press release lacks details of how Descartes-08 compared to placebo using a three-point response threshold.  Cartesian did share results from a per-protocol population that included 31 patients. In that population, the MGC response rates in the Descartes-08 and placebo arms were 69% and 33%, respectively, resulting in a p-value of 0.048. The biotech assessed change from baseline in MG-ADL, the original primary objective, as a secondary endpoint. Recipients of Descartes-08 had a mean 5.6-point improvement on the MG-ADL symptom scale after three months. MG-ADL scores in the placebo arm kept pace with the treatment cohort over the first month but then reversed. The placebo improvement was less than two points after three months.  Cartesian reported a significant imbalance in the proportion of people with thymoma, a type of cancer associated with gMG. While 42% of people on placebo had thymoma, none of their counterparts in the Descartes-08 arm had the cancer. There were numeric imbalances on other variables, with more people on placebo having received complement inhibitors or FcRN drugs and had their thymus removed.  The readout is the first big milestone for Cartesian since it joined the Nasdaq through a reverse merger with Selecta Biosciences. Cartesian emerged from the deal with $110 million to test its belief that mRNA can make CAR-T viable in autoimmune disorders by reducing the toxicity associated with DNA cell therapies and by eliminating the need for preconditioning chemotherapy. Cartesian ended March with almost $105 million, a sum it said would support operations into the second half of 2026. The biotech shared plans to raise $130 million through a private investment in public equity alongside news of the phase 2b results.