Allogene Therapeutics Inc., of South San Francisco, reported preclinical data on its Turbocar technology, designed to allow cytokine activation signaling to be engineered selectively into CAR T cells. After Turbocars mimicking signaling from multiple cytokines were generated, screened and selected based on CAR T-cell manufacturability and in vitro serial killing activity, a range of functional assays showed improvements in activity for the Turbocar T cells compared to parental CAR T cells. The data are slated for presentation in a poster session at the virtual American Society of Gene & Cell Therapy annual meeting on May 12.

Almac Discovery Ltd., of Belfast, U.K., a member of the Almac Group, said it formed a research collaboration with Merck & Co. Inc., of Kenilworth, N.J., focusing on the generation of small-molecule inhibitors against specified deubiquitinase targets to treat neurodegenerative and other diseases. Initially, the companies will collaborate in a two-year joint research program, with Merck assuming responsibility for lead optimization, preclinical and clinical development and commercialization. Almac is set to receive an up-front license fee and research funding and is eligible for payments based on predetermined research, development and commercial milestones as well as tiered royalties on product sales.

The Wistar Institute and Batavia Biosciences Inc., of Woburn, Mass., said they established a strategic collaboration to streamline the clinical-grade manufacture and global distribution of Wistar’s rubella vaccine seed stock commonly known as RA 27/3. In December 2019, Wistar was awarded a $1 million grant from the Bill & Melinda Gates Foundation to support that global access-focused international collaboration.

Beam Therapeutics Inc., of Cambridge, Mass., reported preclinical data on its gene editing programs to treat sickle cell disease (SCD), scheduled for presentation during the American Society of Gene and Cell Therapy annual meeting in May. Using its adenine base editors (ABEs), Beam’s hereditary persistence of fetal hemoglobin (HbF) program to treat SCD aims to recreate single-base changes in the gamma globin gene promoters HBG1 and HBG2 that disrupt repressor binding, leading to increased HbF expression. After sorting human erythroid cells from the bone marrow of mice 16 weeks post-engraftment, an increase in gamma globin levels of more than 65% was seen compared to unedited cells, which showed less than 1.5%. Beam replicated those findings with a second donor at 18 weeks post-engraftment, achieving similar results. In addition, CD34-positive cells from a homozygous sickle patient were successfully edited, showing greater than 60% HbF levels with a concomitant decrease in hemoglobin S (HbS) levels in vitro. Beam also used its ABE platform to edit CD34-positive hematopoietic stem and progenitor cells isolated from donors with the sickle cell trait, achieving editing levels of the HbS mutation of greater than 70% following in vitro erythroid differentiation (IVED). Beam subsequently developed an ultra-high-performance liquid chromatography assay to detect the naturally occurring globin variant HbG-Makassar, which showed conversion of sickle HbS to the asymptomatic HbG-Makassar at levels near 70%, with simultaneous reduction of HbS globin to 30% of control levels in edited IVED cells from a homozygous SCD patient.

Catalent Inc., of Somerset, N.J., said it will increase manufacturing capacity and prep for large-scale commercial production at its 875,000-square-foot facility Bloomington, Ind., location to manufacture New Brunswick, N.J.-based Johnson & Johnson Inc.’s COVID-19 vaccine candidate. Catalent plans to hire about 300 additional employees at the site for the program. Under an accelerated timeline, J&J plans to initiate a phase I trial in September, with clinical data on safety and efficacy expected by year-end. That could allow vaccine availability for emergency use in early 2021, the company added.

Cytrx Corp., of Los Angeles, said that Orphazyme A/S, of Copenhagen, is on track to file an NDA in the first half of 2020 for arimoclomol in Niemann-Pick disease. The company also said it plans to treat its first patient in May through its early access program and plans to file an MAA with the EMA in the second half of 2020. Arimoclomol amplifies production of heat-shock proteins, which can rescue defective misfolded proteins, clear protein aggregates and improve the function of lysosomes, Cytrx said. In 2011, Cytrx agreed to sell and transfer certain data, intellectual property rights and other assets, including contractual rights and obligations relating to a portfolio of chemical compounds, including arimoclomol, to Orphazyme, which included conditional future payments to Cytrx contingent on achieving specified clinical/regulatory and sales milestones as well as single- and double-digit royalty payments, depending on the indication.

New data from Edigene Inc., of Cambridge, Mass., and Beijing, showed significant enzyme α-L-iduronidase (IDUA) enzymatic activity with long duration in mucopolysaccharidosis type I (MPS I) patient-derived cells. Edigene’s program uses an RNA base editing technology. Oligonucleotides of various design, called arRNA, were developed to produce precise and sequence-specific A-to-I conversion in the mRNA sequence for codon 402, leading to generation of the wild-type IDUA mRNA and protein. MPS I is a rare and potentially fatal lysosomal storage disorder that can affect organs and tissue throughout the body. It is a genetic condition that results in a deficiency of the IDUA. As a result, metabolic waste accumulates in cells and cause progressive damage.

Editas Medicine Inc., of Cambridge, Mass., said it initiated IND-enabling activities for EDIT-201, an allogeneic natural killer (NK) cell medicine for treating solid tumor cancers. The company is also advancing several other programs using CRISPR editing to treat solid and liquid tumors, including engineered induced pluripotent stem cell-derived NK cells and engineered gamma-delta T cells.

Fate Therapeutics Inc., of San Diego, said it will present a new off-the-shelf, induced pluripotent stem cell (iPSC)-derived, CAR-targeted cell-based cancer immunotherapy program at the American Society of Gene & Cell Therapy’s virtual meeting in May. The preclinical program targets MHC class I-related proteins A (MICA) and B (MICB) and is supported by an exclusive license from the Dana-Farber Cancer Institute to intellectual property covering antibody fragments binding MICA/B for iPSC-derived cellular therapeutics. MICA and MICB are stress proteins selectively expressed at high levels on many solid tumors. Proteolytic shedding of the α1 and α2 domains of MICA/B expressed on tumor cells is a common mechanism of NK cell evasion, the company said.

Haima Therapeutics LLC, of Cleveland, said it was awarded a $300,000 Phase I Small Business Innovation Research grant from the National Heart, Lung, and Blood Institute of the NIH to support development of its hemostatic nanotechnology to mitigate bleeding in thrombocytopenic patients. Thrombocytopenia, characterized by low platelet counts, affects more than 150,000 people each year in the U.S. Patients with severe thrombocytopenia are at high risk of excessive, uncontrollable bleeding. The current standard of care is transfusion with donor platelet products. Haima’s technology mimics platelet’s hemostatic functions while providing advantages of large-scale production and sterilizability to reduce contamination risk and ensure long shelf-life, the company said.

Heat Biologics Inc., of Durham, N.C., said it continues to advance its COVID-19 vaccine program. The firm’s gp96 COVID-19 effort is focused on providing prophylactic protection to elderly patients and those with underlying health conditions. New data suggest that T-cell and antibody immunity are required for effective prophylactic protection against COVID-19. Heat's vaccine is being designed to clear virus-infected cells by promoting long-term cellular immunity, the company noted.

Innovative Cellular Therapeutics Inc., of Rockville, Md., said two abstracts on its novel Coupledcar solid tumor CAR T technology for treating colorectal and thyroid cancers were selected for oral presentation at the American Society of Gene & Cell Therapy annual meeting in May. They describe two heavily pretreated patient cohorts with colorectal cancer and thyroid cancer, respectively, that achieved near complete remission or partial remission after treatment with Coupledcar.

Preveceutical Medical Inc., of Vancouver, British Columbia, offered an update on its non-addictive analgesic (dynorphin pain peptide) program. Following the identification and shortlisting of a panel of peptides showing promising cell-based activity and extended stability in two distinct types of biological media, the program has identified its first peptide with clear in vivo activity in a rat model of inflammatory pain. The peptide has demonstrated effectiveness in alleviating moderate inflammatory pain after administration of a single dose, the company said. Other shortlisted peptides from the panel will be tested.

Proteogenix SA, of Strasbourg, France, disclosed the first human immune COVID-19 library for the fast discovery of antibodies against the virus. The library, created using blood samples from dozens of recovered patients, is adapted for screening with phage display and strives to meet the rising demands for effective antibodies for a broad range of applications. Its diversity (1.2 x 1010 different clones) allied to the fast turnaround time and sensitivity of the screening technology are intended to fast-track the discovery of antibodies with the strongest affinity, specificity and viral blocking activity, the company said.

Restorbio Inc., of Boston, said it entered a definitive merger agreement with Menlo Park, Calif.-based Adicet Bio Inc. that will create a combined publicly traded company focused on the development of Adicet’s off-the-shelf allogeneic gamma delta T-cell therapies for oncology and other indications. The company’s lead candidate, ADI-001, is a gamma delta CAR T-cell therapy targeting CD20 being developed for non-Hodgkin lymphoma. The all-stock transaction sees Adicet merge with a wholly owned subsidiary of Restorbio with the equity holders of Adicet holding 75% of Restorbio’s outstanding common stock at the close of the deal, expected later this year. The combined entity will operate under the name Adicet Bio with its shares expected to trade on Nasdaq under a new ticker symbol that is yet to be determined. The combined company will also continue the development of RTB-101, Restorbio’s small-molecule product candidate that is an inhibitor of target of rapamycin complex 1 (TORC-1), for a COVID-19-related indication, with clinical data expected by the first quarter of 2021. Under the terms of their merger agreement, a contingent value right (CVR) will be distributed to Restorbio stockholders entitling CVR holders to receive net proceeds from the commercialization, if any, received from a third-party commercial partner of the product candidate RTB-101. At market close April 29, Restorbio’s shares (NASDAQ:TORC) were trading at $1.54, up 26%.

Revolution Medicines Inc., of Redwood City, Calif., said details of the antitumor effects of a SHP2 inhibitor through modulation of key elements of the immune system in preclinical cancer models has been published in Cancer Research. The data demonstrate that inhibition of SHP2, a cellular protein that plays a central role in cell survival and growth, may exert therapeutic antitumor effects by modulating multiple arms of the immune response to the tumor in addition to reducing oncogenic signaling within tumor cells themselves. Importantly, the data indicate that those two mechanisms may be additive in their antitumor impact. The company is evaluating RMC-4630, its orally bioavailable investigational small molecule designed to selectively inhibit the function of SHP2, in a multicohort phase I/II program in patients with advanced tumors. The company and its collaboration partner, Paris-based Sanofi SA, intend to expand that clinical program to include a study evaluating a combination of RMC-4630 with an anti-PD-1 antibody.

Rubius Therapeutics Inc., of Cambridge, Mass., said it will present preclinical data from its oncology pipeline of red cell therapeutics at the American Society of Gene & Cell Therapy virtual meeting in May. The company plans to present preclinical data from its lead artificial antigen-presenting cell program, RTX-321, for the treatment of HPV16-positive tumors. The data demonstrate that antigen-specific T cells for tumor-specific immunotherapy can be activated and expanded in vitro.

Supernus Pharmaceuticals Inc., of Rockville, Md., said it entered a definitive agreement to acquire the CNS portfolio of US Worldmeds, of Louisville, Ky., that will bring in-house three established and marketed products in the U.S. market and a product candidate in late-stage development. They include: Apokyn (apomorphine hydrochloride) injection to control body movements in people with Parkinson’s disease (PD) when they experience an off episode; Myobloc (rimabotulinumtoxinB), a botulinum toxin type B injectable indicated for the treatment of cervical dystonia to reduce the severity of abnormal head position and neck pain associated with cervical dystonia in adults, and the treatment of chronic sialorrhea in adults; Xadago (safinamide) tablets, a monoamine oxidase type B inhibitor indicated as a daily adjunctive treatment to levodopa/carbidopa in patients with PD experiencing off episodes; and, an apomorphine infusion pump for the continuous treatment of motor fluctuations in PD patients whose motor control is unsatisfactory with oral levodopa and at least one other noninvasive PD therapy. A new drug application submission for the product is expected in the second half of the year with potential launch, if approved by the FDA, in the second half of 2021.

Venatorx Pharmaceuticals Inc., of Malvern, Pa. and the Geneva-based Global Antibiotic Research and Development Partnership (GARDP) said they are collaborating to accelerate the development of, and access to, cefepime-taniborbactam (formerly cefepime/VNRX-5133), an investigational combination of the fourth-generation antibiotic cefepime with taniborbactam, a broad-spectrum beta-lactamase inhibitor that restores the activity of cefepime against carbapenem-resistant Enterobacterales and carbapenem-resistant Pseudomonas aeruginosa. Venatorx will receive support from GARDP to complete the development of cefepime-taniborbactam, which includes a phase III complicated urinary tract infection trial, which is in progress.

Verndari Inc., of Sacramento, Calif., said it plans to start preclinical testing of a potential COVID-19 coronavirus vaccine, which will be administered using Vaxipatch, a microneedle array dermal patch.

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