Cell and Gene Therapy Business Outlook



The Best Way to Keep up with the Growing Cell and Gene Therapy Industry

From Science and Medicine Group, the company behind Instrument Business Outlook, Kalorama Information, SDi and other publications, comes a new publication: Cell and Gene Therapy Business Outlook.


With thousands of potential therapies on the market, cell and gene therapy promises future potential for pharmaceutical developers and those serving them.

  • A new twice-monthly publication dedicated to cell and gene therapy, Cell and Gene Therapy Business Outlook will offer the following:
  • Market Sizing and Forecasting of CGT Markets in Every Issue
  • Executive News Summaries – What is Happening in CGT Markets and Why It Matters
  • Deals Between CGT Companies Tracked in Every Issue
  • Important Science That Will Shape Tomorrow’s Business
  • Updates on Pipelines and Important Clinical Trials
  • Cell and Gene Therapy Tools, CMOs, Manufacturing Developments
  • Market Analysis of a Cell and Gene Therapy Segment in Every Issue




There are many websites, publications and sources on cell therapy. Cell and Gene Therapy Business Outlook differs from these sources in that it is created by market researchers and editors focused on business opportunity. Each issue will track the market size and potential for a key market segment.

Who Is Dealing with Whom? Tracking of Cell and Gene Company Deals In Every Issue.

There is a never-ending stream of activities in this market. How can you keep up? Each issue of Cell and Gene Therapy Business Outlook will keep track of mergers, investments, licensing, technology transfers and partnerships in the industry. Each issue of Cell and Gene Therapy Business Outlook contains an updated CGT Recent Deals Table with information on these important events.

Future issues will also analyze of the number of deals and increases or decreases in activity as a measure of business. You’ll never miss an important happening with Cell and Gene Therapy Business Outlook. Also, the Recent Deals Table is a great resource for tracking companies in the market.

The News That Matters

Edited by Blake Middleton, a professional CGT researcher and former Staff Research Associate at UCLA Department of Pharmacology, Cell and Gene Therapy Business Outlook is designed to provide the most relevant news. Included is news that could affect business decisions near-term. Cell and Gene Therapy Business Outlook also explains the relevant science.
With a focus on what the recent news of the day means for business, our curated news and news analysis means that you and your organization can be confident you won’t miss an important development in cell and gene therapy.




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Open up access: If more than one person will be reading, you can unlock access to other members of your organization. It’s easy to do: team subscription prices are as little as $4,995 annually for up to five readers. Larger team? Other licenses are available. Consult our website.  Convenient and Cost-Effective Seat-Based Pricing: Pricing depends on the number of users. Subscriptions can be as low as $2,200 annually for a limited one-person (single user) subscription.



MARKET SIZE: The global market for cell and gene therapy for oncology reached $1,582 million in 2020 and is expected to climb to $2,744 for 2021.

There are over 100 different types of cancer; some of the more prominent include lung, breast, brain, blood, prostate and colon cancer. The immune system plays a primary role in the body’s defense against malignancy. Although a tumor is derived from the body’s own cells and is expected to possess proteins that are recognized as self and nonantigenic, neoplastic cells can express antigens that are not recognized as self. These cells can often be eliminated by the immune system.

FORECAST: projected to increase to $7,391 in 2025; $17,490 million by 2030.

Treating cancer is difficult because it is not a single disease and because all the cells in a single tumor do not behave in the same way. Although most cancers are thought to be derived from a single abnormal cell, by the time a tumor reaches a clinically detectable size, the cancer may contain a diverse population of cells.

Market Forecast:  Strong increases in the CAR-T therapy market, increasing from just $16 million in 2017 to $1,081 million in 2020 and projected to increase to $7,391 in 2025; $17,490 million by 2030.  Blood cancers are the leading driver in the segment, representing 68% of total sales. This is expected to be the primary segment through the forecast, representing 80% of sales by 2025 and 80% in 2030.  The United States and Europe are the largest markets due to overall product approvals and cost associated with the therapies. The US market represented nearly 77%, while Europe represented 19% in 2020.  Gilead and Novartis combined represent 68% of the market for cell and gene therapy in oncology.  Industry refocuses on oncology cell and gene therapies in a post-pandemic arena, returning to pre-pandemic growth.



Adeno-associated viruses (AAV) are small human viruses which provoke only a mild immune response and are not known to cause any human disease. AAVs are quite simple in organization, possessing a small (4.7kb) single-stranded DNA genome with only two open reading frames (ORFs), rep and cap, flanked by short (145 base) inverted terminal repeats (ITRs). The rep ORF encodes multiple overlapping sequences for proteins required for replication, and the cap ROF does the same for capsid proteins, which are the proteins forming the outer viral protein coat. These genes alone are not sufficient for viral replication, and AAVs require co-infection with a second, helper virus (such as an adenovirus or HSV) to supply the remaining gene products for replication (hence the name adeno-associated virus).

Gene therapy AAV vectors are further modified to remove the rep and cap genes from the viral genome (along with their promoters and polyadenylation signal), replacing them with a therapeutic expression cassette. Production of recombinant AAV vectors in cell lines requires the rep and cap genes to be supplied by a plasmid transfected in trans, in addition to the genes supplied by the helper virus. None of these externally supplied viral genes are packaged into the final construct, so the resulting viral delivery vehicle consists only of the therapeutic cassette encased in an AAV capsid, without any viral genes present. The gene therapy vector is therefore incapable of replication, even with co-infection by a suitable helper virus.
In addition to their safety, AAV vectors possess many features which make them attractive gene therapy candidates. They have extremely low immunogenicity, they can infect both dividing and non-dividing cells, and they can persist outside the genome to offer stable, long-term expression without the risks associated with host genome integration.

AAV vectors also suffer from several shortcomings, however:
• Because of their wide distribution, many individuals have already been exposed to naturally occurring AAV serotypes and produce immune responses against them.
• AAV vectors cannot reach most tissues efficiently, and do not spread easily within those tissues if they do.
• Vectors will preferentially target some cell types but not others.
• Transduction efficiency is often extremely low.

Each of these shortcomings can be addressed by innovations in capsid structure. In addition to protecting the DNA payload, the capsid is responsible for binding to specific receptors on the target cell and safely delivering the DNA payload to the cell machinery that so will be transported to the nucleus. Viral packaging efficiency, host immunological response, tissue and cell type specificity, and transduction efficiency are all determined by the capsid serotype. Unfortunately, initial gene therapy experiments were restricted to a handful of natural AAV serotypes which had limited tropism in many human cell types. Common serotypes also present problems with pre-existing immunity (PEI), as up to 90% of the human population have already been exposed to at least one AAV serotype. For these reasons, novel capsid discovery is a current hotbed of gene therapy research.

More information on this topic can be found in the latest issue. SUBSCRIBE TODAY




With the 2022 Cell & Gene Meeting on the Mesa (#CGMesa22) underway, here’s a look at some notable recent developments in cell and gene therapy featured in the bimonthly newsletter, Cell and Gene Therapy Business Outlook.

  • Myrtelle, a clinical-stage gene therapy company based in Wakefield, MA, and Forge Biologics, a gene therapy contract development and manu-facturing organization (CDMO) based in based in Grove City, OH, have announced a manufacturing partnership to bring Myr-201, Myrtelle’s gene therapy for monogenic hearing loss, into Phase I/II clinical trials for the treatment of autosomal recessive nonsyndromic deafness 8 (DFNB8).  DFNB8 is caused by mutations in the TMPRSS3 gene encoding trans-membrane protease, serine 3, and Myr-201 uses a low-dose, locally administered adeno-associated virus (AAV) vector to deliver a functional copy of that gene.  Under the agreement, Forge will provide manufacturing services for research-grade and GMP-pathway plasmids as well as cGMP AAV process development and scale-up for Myr-201.  Development and cGMP manufacturing will take place in Columbus, OH, at The Hearth, Forge’s 200,000-square-foot gene therapy cGMP production facility, where Forge will use its platform process, including its proprietary Ingition HEK 293 suspension cell line and its pEMBR adenovirus helper plasmid.  Financial details were not disclosed.
  • Oncternal Therapeutics, a clinical-stage oncology company based in San Diego, CA, has announced that it has received IND clearance from the U.S. FDA for a Phase I/II dose escalation study of ONCT-808, an autologous chimeric antigen receptor (CAR) T cell therapy targeting ROR1 for the treatment of aggressive B cell non-Hodgkin’s lymphoma (B NHL).  ROR1 is a receptor tyrosine kinase that is highly expressed in many types of cancer (including both hematologic and solid tumors), but rarely expressed on healthy adult cells, and its expression has been associated with a survival advantage in tumor cells. Oncternal plans to begin the study in the next few months and present interim results in 2023.
  • MaxCyte, a cell engineering company based in Gaithersburg, MD, has announced the signing of a strategic platform license (SPL) with Vertex Pharmaceuticals, a biopharmaceutical company based in Boston, MA, to allow Vertex to use MaxCyte’s Flow Electroporation technology and ExPERT platform to develop exagamglogene autotemcel (exa-cel, formerly CTX001), its ex vivo gene therapy for the treatment of beta thalassemia and sickle cell disease. Under the agreement, Vertex obtains non-exclusive rights to use MaxCyte’s technologies in exchange for platform licensing fees and program related revenue.  Both beta thalassemia and sickle cell disease are genetic blood diseases caused by mutations in the HBB gene encoding β-globin, a major component of hemoglobin A (HbA), the most common form of hemoglobin in human adults.  Exa-cel is an autologous gene therapy in which a patient’s hematopoietic stem cells (HSCs) are harvested and edited to produce red blood cells expressing high levels of fetal hemoglobin (HbF) to complete with the defective HbA. Vertex is developing exa-cel in collaboration with Swiss gene editing company CRISPR Therapeutics, which used the same MaxCyte technology to develop the therapy under an agreement with MaxCyte. Vertex recently announced that the company had concluded discussions with the U.S. FDA and will submit exa-cel’s biologics licensing application (BLA) for the treatment of sickle cell disease and transfusion-dependent beta thalassemia for rolling review beginning in November 2022, and anticipates completing the submission in early 2023.  Vertex also plans to submit marketing applications for exa-cel to the European Medicines Agency (EMA) and the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) by the end of 2022.
  • Legend Biotech Corporation, a clinical-stage immune cell therapy company based in Somerset, NJ, has announced that that Japan’s Ministry of Health, Labour and Welfare (MHLW) has approved CARVYKTI (ciltacabtagene autoleucel, or cilta-cel) as a fourth-line treatment for adults with relapsed/refractory (R/R) multiple myeloma (MM). CARVYKTI, developed in collaboration with Janssen Biotech, is a genetically modified, autologous T-cell immunotherapy with a CAR design featuring two single-domain antibodies targeting B-cell maturation antigen (BCMA), which is primarily expressed on the surface of malignant multiple myeloma B-lineage cells, as well as late-stage B-cells and plasma cells.  CARVYKTI was approved by the U.S. FDA as a fifth-line treatment for adults with R/R MM in February 2022, and was granted conditional marketing authorization as a fourth-line treatment for adults with R/R MM by the European Commission (EC) in May 2022.  The therapy has received Breakthrough Therapy designation in the U.S. and China, and Orphan Drug designation in the U.S., EU, and Japan.
  • Scribe Therapeutics, a molecular engineering company based in Alameda, CA, and Sanofi, a multinational healthcare company based in Paris, France, have announced a strategic collaboration to use Scribe’s CRISPR by Design platform technology in Sanofi’s engineered natural killer (NK) cell therapies.  The agreement grants Sanofi a non-exclusive license to Scribe’s CasX-Editor (XE) genome editing technology to create ex vivo NK cell therapies for multiple oncology targets.  In exchange, Scribe will receive $25 million up front, and will be eligible for more than $1 billion in development and commercial milestone payments, as well as tiered royalties on any future sales.
  • ArsenalBio, a programmable immune cell therapy company based in South San Francisco, CA, has announced a multi-year collaboration with Genentech (a member of the Roche Group), also based in South San Francisco.  The two companies will combine their capabilities to evaluate effective T cell modifications and identify critical success circuits in T cell-based therapies, then apply those discoveries to the development of future therapeutic candidates. ArsenalBio’s proprietary technology platform combines non-viral genome editing, computational biology, and large-scale genetic screening to reprogram chimeric antigen receptor (CAR)-T cells to overcome cell exhaustion, improve memory, and enable persistence.  Under the agreement, ArsenalBio will receive $70 million up front in additions to research, development, and commercial milestone payments.
  • Pfizer, a multinational biotech and pharmaceutical company based in New York, NY, and Sangamo Therapeutics, a cell and gene therapy company based in Brisbane, CA, have announced plans to resume their Phase III study AFFINE evaluating giroctocogene fitelparvovec (PF-07055480) for the treatment of hemophilia A.  Hemophilia A is an X-linked inherited disorder resulting in a deficiency of coagulation factor VIII, and giroctocogene fitelparvovec uses an adeno-associated virus to deliver a functional factor VIII transgene.  The therapy was so effective that some participants in the trial were observed with factor VIII levels greater than 150%, levels which are high enough to carry an increased risk of blood clots.  Based on that risk, the U.S. FDA initialed a clinical hold to amend the protocol with guidelines to safely manage those elevated factor VIII levels.  The FDA lifted that clinical hold in March, but Pfizer and Sangamo decided to continue with a voluntary hold until now as they finalized study protocols and ensured all necessary study conditions had been met.
  • Emercell, an immunotherapy company based in Montpellier, France, and Cell-Easy, an analytics-driven contract development and manufacturing organization (CDMO) based in Toulouse, France, have announced a strategic agreement for the scale-up and manufacturing of NK-001, Emercell’s lead product. Emercell is developing a technology platform based on off-the-shelf natural killer (NK) cells, which can used alone as a monotherapy, in combination with therapeutic antibodies, or genetically engineered to create Chimeric Antigen Receptor (CAR)-NK cell therapies.  NK-001 consists of highly activated and alloreactive allogeneic NK cells derived from compatible pooled umbilical cord blood (UCB), to be used in combination with monoclonal antibodies (MAbs) for the treatment of refractory cancers such as lymphomas.  Emercell hopes to bring NK-001 to the clinic in 2023.
  • Verve Therapeutics, a clinical-stage gene editing company based in Cambridge, MA, has announced that its Clinical Trial Authorisation (CTA) application for its lead therapeutic candidate, VERVE-101, has been cleared by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) for the treatment of heterozygous familial hypercholesterolemia (HeFH).  VERVE-101 is an in vivo gene editing therapy engineered to knock out the PCSK9 gene encoding proprotein convertase subtilisin/kexin type 9, an enzyme involved the transport of cholesterol.  The therapy is comprised of a messenger RNA encoding an adenine base editor licensed from Beam Therapeutics and a guide RNA targeting the PCSK9 gene, packaged in a lipid nanoparticle (LNP) delivery system targeting the liver. A single A-to-G base change in the PCSK9 gene inactivates that gene, which has been shown to up-regulate low-density lipoprotein (LDL) receptor expression, leading to lower serum LDL cholesterol levels and a reduced risk for atherosclerotic cardiovascular disease (ASCVD).
  • Cellenkos, a clinical-stage cell therapy company based in Houston, TX, has announced that it has received IND clearance from the U.S. FDA to initiate a Phase I safety study followed by a Phase Ib randomized, double-blind, placebo-controlled trial of CK0803 for the treatment amyotrophic lateral sclerosis (ALS), a fatal neurological disease.  Cellenkos is developing allogeneic T regulatory (Treg) cell therapies for the treatment of inflammatory diseases and autoimmune disorders, and CK0803 is an allogeneic cell therapy product consisting of robust, activated Treg cells that carry neurotropic detection signals which direct them to inflammatory pockets inside the central nervous system.  The therapy is derived from clinical-grade umbilical cord blood units using Cellenkos’ proprietary CRANE process and can be administered by a simple intravenous infusion, without requiring any HLA matching, immune suppression, or lymphodepletion prior to administration.
  • Frontera Therapeutics, a clinical-stage gene therapy company based in Bedford, MA, has announced that it has received IND clearance from China’s Center for Drug Evaluation (CDE), National Medical Products Administration (NMPA), to evaluate FT-001, the company’s lead gene therapy candidate for the treatment of a rare genetic retinal disease.  Frontera is developing adeno-associated virus (AAV)-based gene therapies for indications in ophthalmology, hematology, neuromuscular diseases, and metabolic diseases.  FT-001 is an AAV-based gene therapy for the treatment of inherited retinal degenerations (IRDs) caused by a mutation in the RPE65 gene encoding retinoid isomerohydrolase.  The therapy is administered by a one-time injection into the subretinal space of the eye, where it delivers a functional copy of the RPE65 gene to the patient’s retinal cells.  FT-001 was previously granted IND approval from the U.S. FDA in April of this year.
  • Avectas, an immune cell engineering company based in Dublin, Ireland, and GenScript Biotech Corporation, a life sciences research tools and services company based in Piscataway, NJ, have announced a partnership to improve non-viral based cell therapy manufacturing technologies.  Avectas is developing SOLUPORE, a patented, non-viral, cell permeabilization technology designed for use with mRNA, DNA, and proteins.  The technology can efficiently delivery cell engineering payloads, including gene editing tools such as CRISPR, to primary T cells and natural killer (NK) cells for immuno-oncology and gene editing applications.  The partnership will combine Avectas’ cell engineering technology platform with GenScript’s synthetic long oligo expertise, to co-deliver Avectas’ GenCRISPR synthetic sgRNA and Cas9 protein ribonucleoprotein (RNP) complexes with its GenExact ssDNA homology-directed repair (HDR) templates to the cell nucleus.
  • The Center for Breakthrough Medicines (CBM), a cell and gene therapy contract development and manufacturing organization (CDMO) based in King of Prussia, PA, and jCyte, a clinical-stage cell therapy company based in Newport Beach, CA, have announced a multi-year manufacturing agreement that designates CBM as the primary manufacturer of jCyte’s intravitreal cell therapy, jCell.  Under the agreement, CBM will provide supplies for Phase III clinical studies of jCell, as well as commercial drug product following FDA approval the therapy. Administered by a minimally invasive intravitreal injection, jCell is designed to treat degenerative retinal disorders by providing sustained release of neurotrophic factors that have been shown to reduce photoreceptor cell death and improve the function of surviving photoreceptor cells. The therapy has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. FDA, and is currently in late-stage clinical development for the treatment of retinitis pigmentosa (RP).

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.

  • Kite Pharma, a Gilead Company based in Santa Monica, CA, has announced that the European Commission (EC) has granted approval for its chimeric antigen receptor (CAR) T cell therapy Tecartus (brexucabtagene autoleucel) for the treatment of adult patients 26 years of age and above with relapsed or refractory (R/R) B-cell precursor acute lymphoblastic leukemia (ALL).  Tecartus is an autologous, CAR T cell therapy targeting CD19, an antigen ubiquitously expressed on B cells.  Last year Tecartus was approved by the U.S. FDA for the same indication.
  • CellOrigin Biotech, an immune cell therapy company based in Hangzhou, China, and Qilu Pharmaceutical, a pharmaceutical company based in Jinan, China, have announced a collaboration to develop, manufacture, and commercialize off-the-shelf induced pluripotent stem cell (iPSC)-derived chimeric antigen receptor macrophages (CAR-iMAC) for cancer immunotherapy.  CellOrigin’s developmental pipeline currently includes an iPSC-derived natural killer (iNK) + monoclonal antibody therapy candidate for the treatment of hematological malignancies, a CAR-iNK cell therapy candidate for solid tumors, and several CAR-iMAC cell therapy candidates for solid tumors.
  • The University of Sydney has announced an unprecedented $478 million investment to build a leading biomedical precinct in New South Wales, its largest ever capital investment. The Sydney Biomedical Accelerator (SBA) will establish a 36,000 square-meter health, education, and research precinct, to be co-located at Royal Prince Alfred Hospital and the University’s Camperdown campuses.  The New South Wales Government contributed $143.3 million to the project, and other funding includes $73 million in philanthropy to the University of Sydney and a $20 million donation from the Susan and Isaac Wakil Foundation to establish The Isaac Wakil Biomedical Building.   The SBA will support over 1200 biomedical researchers and clinician scientists, including over 800 university laboratory researchers and PhD students, with occupation anticipated to begin in 2026.
  • Cytiva, a life sciences company based in Marlborough, MA, and Caring Cross, a 501(c)(3) non-profit organization focused on developing advanced medicines accessible to all patients, have announced a partnership to develop a chimeric antigen receptor (CAR)-T cell therapy for people with HIV in low-to-middle-income nations.  Cytiva will provide equipment and software packages, including the Sepax C-Pro, (a place of care instrument which can reduce CAR-T processing times by automatically isolating, concentrating, washing, and diluting cellular products) and the VIA Thaw and VIA Freeze instruments (which streamline freezing and thawing processes while minimizing the contamination risks associated with traditional water baths).  Caring Cross has developed a duoCAR-T cell therapy candidate targeting HIV which can both eliminate HIV-infected cells and protect CD4-T cells from HIV infection in in vitro studies and animal models, and the therapy is currently being evaluated in a Phase I/II clinical trial.  Caring Cross is also developing a stem cell gene therapy for the treatment of sickle cell disease (SCD) and beta-thalassemia.
  • Arbor Biotechnologies, a gene editing company based in Cambridge, MA, has announced that it has entered an agreement with Acuitas Therapeutics, a leader in the development of lipid nanoparticles (LNP) based in Vancouver, BC, to develop therapies for rare liver diseases.  Arbor specializes in discovering and developing novel gene editors using its machine learning/AI driven discovery platform, and has an established pipeline of rare liver disease programs in preclinical development.  The two companies plan to combine Acuitas’ optimized LNP delivery technology with Arbor’s proprietary CRISPR-based in vivo gene editing therapies targeting rare liver diseases to accelerate these programs into the clinic.
  • ElevateBio, a cell and gene therapy technology company based in Cambridge, MA, and the University of Pittsburgh have announced a long-term strategic partnership to develop cell and gene therapies.  The 30-year agreement will establish ElevateBio’s next BaseCamp process development and Good Manufacturing Practice (GMP) manufacturing facility at the Pitt BioForge BioManufacturing Center at Hazelwood Green.  The University of Pittsburgh created the Pitt BioForge biomanufacturing facility with a $100 million grant from the Richard King Mellon Foundation in November 2021.  The new BaseCamp facility will be equipped with gene editing and  induced pluripotent stem cell (iPSC) technologies, as well as cell, vector, and protein engineering capabilities, and is expected to generate more than 170 full-time jobs.
  • ElevateBio has also announced a partnership with the California Institute for Regenerative Medicine (CIRM) to advance regenerative medicine therapies through CIRM’s Industry Alliance Program.  For its part, ElevateBio will supply its induced pluripotent stem cells (iPSC) lines to academic and commercial beneficiaries of CIRM Discovery and Translational Grants, along with viral vector technology, process and analytical development, and Good Manufacturing Practice (GMP) manufacturing capabilities. CIRM has received $5.5 billion in funding from the state of California, and has already funded over 161 regenerative medicine research projects and 81 clinical trials.
  • BioCardia, a cell therapy company based in Sunnyvale, CA, has announced it has entered an agreement with BlueRock Therapeutics, an engineered cell therapy company (and subsidiary of Bayer AG) based in Cambridge, MA, to use BioCardia’s minimally invasive biotherapeutic catheter delivery systems to deliver BlueRock’s induced pluripotent stem cell (iPSC)-derived cell therapy product candidates locally to the heart for the treatment of heart failure. Under the time-limited agreement, BioCardia will receive an up-front payment, and BlueRock will have an option to negotiate a non-exclusive worldwide license to use BioCardia’s biotherapeutic delivery systems to deliver certain cell types for cardiac indications.
  • Gensaic, a gene therapy spinout from MIT based in Cambridge, MA, has announced a strategic collaboration agreement with Ovid Therapeutics, a gene therapy company based in New York, NY, to develop up to three genetic medicines for central nervous system (CNS) indications of interest to Ovid using its proprietary phage-derived particle (PDP) platform.  Under the terms of the agreement, Gensaic retains full rights to its PDP platform technology, while Ovid will have the right to license and develop any gene therapies resulting from the collaboration.  Ovid has also invested $5 million in Gensaic and retained rights to invest in future rounds.  Gensaic’s PDP platform is a modular system derived from the M13 bacteriophage (a filamentous virus which infects bacteria) and consists of three engineerable capsid proteins and a minimal phage DNA (mpDNA) genome capable of delivering of genetic payloads larger than 20 kb.  The platform combines phage display technology with directed evolution to engineer tissue-specific, immune-privileged, and redosable delivery vehicles that can be easily manufactured in bacterial culture.  Gensaic is currently focused on developing delivery systems  targeting muscle, respiratory, and CNS tissues.
  • Cambridge, MA-based bluebird bio has announced that the U.S. FDA has approved ZYNTEGLO (betibeglogene autotemcel, or beti-cel), its one-time gene therapy for the treatment of beta‑thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions.  Beta-thalassemia is a rare genetic blood disease caused by mutations in the HBB gene encoding β-globin, a major component of hemoglobin A (HbA) resulting in reduced or absent adult hemoglobin production. In Zynteglo therapy, a patient’s hematopoietic stem cells (HSCs) are harvested, then purified and transduced them with a lentiviral vector (LVV) carrying a modified form of the HBB gene encoding β-globin.  The cells are then re-infused back into the patient, where they engraft to the bone marrow and produce new blood cells expressing the modified HBB gene, enabling the cells to make normal or nearly normal levels of adult hemoglobin. The therapy is the first ex vivo lentiviral vector gene therapy to be approved in the U.S. for the treatment of beta-thalassemia. (See Cell and Gene Therapy Business Outlook vol. 2, issue 1, p. 13 for more on bluebird bio and Zynteglo, its gene therapy for beta-thalassemia.)    
  • GentiBio, a biotherapeutics company based in Boston, MA, has announced that it has entered a collaboration with Bristol Myers Squibb, based in New York, NY, to develop engineered regulatory T (Treg) cell therapies for the treatment of patients with inflammatory bowel diseases (IBD).  GentiBio specializes in engineered Treg cell therapies, which have the potential to treat autoimmune and inflammatory diseases such as IBD by re-establishing immune tolerance in a tissue-specific manner.  Under the multi-year collaboration, GentiBio will produce stable, disease-specific engineered Tregs against multiple targets utilizing its modular engineered Treg platform and scalable manufacturing process, and Bristol Myers Squibb will have the right to bring up to three programs resulting from the collaboration into the clinic.  In exchange, Bristol Myers Squibb has made an undisclosed cash payment to GentiBio up front, and GentiBio is eligible to receive up to $1.9 billion in development and sales milestones, plus royalties.