12 Recent Developments in Cell and Gene Therapy as of April 22, 2022

There have been a number of recent developments in cell and gene therapy, as detailed in our bimonthly newsletter, Cell and Gene Therapy Business Outlook.

1. In public offering news, Massachusetts-based Selecta Biosciences has announced a $38.7 million public offering to continue developing ImmTOR, its antigen-specific immune tolerance platform.
2. Leading the private financing news, Cambridge-based cell therapy company Be Biopharma has raised $130 million in Series B financing to support its innovative B cell-based therapeutic protein delivery platform, bringing the company’s total to over $180 million, while vision-focused Aurion Biotech has raised $120 million with a corneal cell therapy leading its pipeline.
3. Cimeio Therapeutics, based in Boston, MA, and Basel, Switzerland, has emerged from stealth with a novel cell-shielding technology and a $50 million Series A investment, and French DNA analysis company Genomic Vision has announced a new financing line worth up to $30 million.
4. Finally, Scottish macrophage-based cell therapy Resolution Therapeutics has announced a $13 million extension to its $35 million Series A financing, Florida-based allogeneic cell supplier Gift of Life Biologics closed a $6 million funding round, and Belgian orthopedic cell therapy company Bone Therapeutics has signed a $5.4 million convertible bonds facility.
5. Selecta Biosciences, a clinical-stage biotech company based in Watertown, MA, has announced that it has agreed to sell 27,428,572 shares of common stock at $1.41 per share (and accompanying warrants to purchase up to 20,571,429 shares of common stock, with an exercise price of $1.55 per share). The offering was expected to close on April 11, 2022, with gross proceeds of approximately $38.7 million.  SVB Leerink is sole bookrunner for the offering, with Canaccord Genuity acting as lead manager.  Selecta is developing ImmTOR, a nanoparticle-based system that delivers an immunosuppressant along with an antigen to promote antigen-specific immune tolerance.  The company’s candidate SEL-212 is being developed and commercialized for the treatment of chronic refractory gout through an out-licensing partnership with Swedish Orphan Biovitrum, and is currently being evaluated in in a Phase III clinical study. SEL-212 uses Selecta’s ImmTOR platform to deliver Pegadricase, which is a PEGylated version of urate oxidase derived from the yeast Candida utilis, designed to treat gout by reducing levels of serum uric acid.  While the PEGylation process blocks immunogenic epitopes on the Pegadricase enzyme, as a foreign protein it can still provoke a strong immune response.  By delivering an immunosuppressant drug called rapamycin alongside Pegadricase, ImmTOR promotes immune tolerance toward the foreign enzyme.  Selecta is also beginning a Phase I/II clinical trial of SEL-302 for the treatment methylmalonic acidemia (MMA), a rare metabolic disease that affects the body’s ability to metabolize certain amino acids and fats. SEL-302 uses the ImmTOR platform to deliver MMA-101, an AAV8-based gene therapy that delivers a functional copy of the MMUT gene to treat MMA.  ImmTOR delivery promotes immune tolerance toward the AAV vector, increasing the duration of its effectiveness and allowing it to be re-dosed.  The company is currently developing treatments for multiple indications in the fields of biologics, gene therapy, and autoimmune disease.
6. Be Biopharma, based in Cambridge, MA, has announced the closing of a $130 million Series B financing bringing the total raised by the company to over $180 million. The financing was led by ARCH Venture Partners joined by new investors including Bristol Myers Squibb and existing investors including Atlas Venture, RA Capital Management, Alta Partners, Longwood Fund, and Takeda Ventures.   Be Biopharma is developing autologous and allogeneic Engineered B Cell Medicines (BeCM), based on research by co-founders Richard James, PhD,  and David Rawlings, MD, at Seattle Children’s Research Institute and the University of Washington School of Medicine.  The company’s BeCM platform takes advantage of B cells’ intrinsic ability to produce large quantities of protein, co-opting them to become programmable medicine factories with a broad range of applications.  Be Biopharma’s initial pipeline is centered on rare diseases and cancer, but the company plans to add additional therapeutics in areas such as infectious disease, neurological conditions, and autoimmune disease.
7. Aurion Biotech, a clinical-stage vision-based biotech company based in Seattle, WA, with operations in Boston, MA, and Tokyo, Japan, has announced a $120 million financing round. The financing was led by Deerfield Management, and included existing investors Petrichor Healthcare Capital Management, Flying L Partners, Falcon Vision, and Visionary Ventures, with additional participation by Alcon. The funding will be disbursed based on the achievement of certain milestones.  Aurion is developing therapies for eyesight disorders (Falcon Vision and Visionary Ventures both specialize in ophthalmology-related investment), and its first candidate is a cell therapy for the treatment of corneal edema secondary to endothelial dysfunction.  The condition is treatable with corneal transplant therapy, however a severe shortage of donor corneas and the complex surgical procedure required limit that treatment’s availability to patients.  Aurion’s platform cultures healthy cells from donor corneas in a proprietary and patented process that enables a single cornea donor to treat more than 100 recipient eyes with a simple injection.
8. Cimeio Therapeutics, based in Boston, MA, and Basel, Switzerland, has emerged from stealth with a $50 million Series A investment from Versant Ventures’ Ridgeline Discovery Engine in Basel. The company is developing Shielded-Cell & Immunotherapy Pairs, which combine cell-shielding technology with precisely paired immunotherapies, with the goal of improving hematopoietic stem cell (HSC) transplant and adoptive cell therapy (ACT) outcomes.  Cimeio’s proprietary immunotherapies target diseased cells, while healthy transplanted cells are protected by its cell-shielding technology.  The immunotherapies can be safely administered post-transplant as well.  The technology uses gene editing to alter cell surface receptors, inserting novel protein variants which prevent antibody binding while retaining receptor function.  In preclinical studies, the company has been able to protect its edited cells from attack by antibodies, T-cell engagers, antibody-drug conjugates (ADCs), and chimeric antigen receptor (CAR)-T cells.  Cimeio plans to have its first candidates in clinical development in 2023.
9. Genomic Vision, a DNA analysis company based in Bagneux, France, has announced a new financing line with Winance for a maximum of 15 financing tranches of €2 million each, for a total of up to €30 million (approximately $32.5 million), which is sufficient to finance the company’s projected activities and development for the next three years. The company has developed a propriety Molecular Combing technology platform which automatically stretches single DNA molecules onto specially treated glass surfaces. The discrete DNA stands can then be analyzed at the single molecule level using the company’s Genomic Morse Code platform, which utilizes fluorescent probes of different colors and sizes designed to recognize a selected region of interest in the DNA and generate a code which can be used to identify changes to that DNA sequence, such as amplifications, deletions, repeats, inversions, and translocations.
10. Resolution Therapeutics, a cell therapy company based in Edinburgh, Scotland, has announced the completion of a £10 million ($13 million) extension to the £26.6m ($34.75 million) Series A financing by Syncona, which was announced in December 2020. The company specializes in macrophage cell therapies for the treatment of inflammatory organ damage, based on clinical research by founders Stuart Forbes, PhD, at the University of Edinburgh, and John Campbell, PhD, at the Scottish National Blood Transfusion Service. Resolution is developing an autologous macrophage cell therapy for the treatment of chronic liver disease, and the financing will be used to continue to develop its autologous macrophage therapy pipeline and expand that development into allogeneic (“off-the-shelf”) macrophage cell therapies.
11. Gift of Life Biologics, a GMP-compliant supplier of allogeneic cellular starting material based in Boca Raton, FL, has announced that it has closed a $6 million funding round, led by a world-renowned pharmaceutical company and Three Bridges Private Capital. The company is backed by the Gift of Life Marrow Registry and operates a 32,000 square-foot facility in South Florida which includes a donor center, a high-capacity apheresis collection facility, and a cellular therapy laboratory and biobank.  Gift of Life’s donor registry has a genetically diverse pool of more than 400,000 characterized donors and is accredited by the World Marrow Donor Association (WMDA), its apheresis center is accredited by the Association for the Advancement of Blood & Biotherapies (AABB), and its cellular therapy laboratory’s moderate and high complexity testing services are Clinical Laboratory Improvement Amendments (CLIA) accredited.
12. Bone Therapeutics, an orthopedic cell therapy company based in Gosselies, Belgium, has announced that it has signed a €5 million ($5.4 million) convertible bonds (CBs) facility arranged by ABO Securities. The financing will be issued in seven tranches (€1.5 million, €1 million, and six tranches of €500,000) and will be used for the continuing clinical development of Bone Therapeutics’ lead candidate ALLOB. ALLOB is Bone Therapeutics’ off-the-shelf, allogeneic bone cell therapy derived from ex vivo cultured human bone marrow mesenchymal stromal cells (MSCs) from healthy adult donors, and is currently being evaluated in a Phase IIb clinical trial for the treatment of high-risk (delayed-union) tibial fractures, and in a Phase IIA trial evaluating ALLOB administration in lumbar spinal fusion.