Highlights & Summary
Intro
July stands out for its wealth of news on company growth - read below for some of the many financing rounds, acquisitions, and partnerships the next-gen space saw in the last month.
Cell Therapy
- Vertex acquires ViaCyte and doubles down on T1D | Acquisitions
- MD Anderson and Obsidian ready to begin Phase 1 on TIL therapy | Clinical Trials
Gene Therapy
- Freeline Therapeutics demonstrates sustained factor IX levels in hemophilia B patients | Clinical Trials
- VBL Therapeutics’ AAV therapy flunks PhIII for ovarian cancer | Clinical Trials
Oligo Therapy
- Roche will license and advance asset into a Phase 3 clinical study for immunoglobulin A nephropathy (IgAN) | Clinical Trials
- Verve Therapeutics Doses First patient with its In Vivo Base Editing Medicine in Heterozygous Familial Hypercholesterolemia | Clinical Trials
Cell Therapy
Atara stock dips after phase 2 progressive MS candidate has inconclusive results | Clinical Trials
An interim analysis of Atara’s Phase 2 study of a multiple sclerosis drug showed inconclusive results, causing their stock price to tumble. However, the data review showed no safety issues and enrollment has since been completed, with a final data readout expected in late 2023.
Vertex acquires ViaCyte and doubles down on T1D | Acquisitions
Vertex’s latest acquisition of ViaCyte expands their type-1 diabetes portfolio with additional stem cell-based treatments in development as well as other intellectual property and GMP facilities. This cements their position as the frontrunner in the T1D market.
MD Anderson and Obsidian ready to begin Phase 1 on TIL therapy | Clinical Trials
MD Anderson and Obsidian have received IND clearance by the FDA for their new therapy, an engineered tumor-infiltrating lymphocyte with membrane-bound IL15. The initial patient population will be melanoma patients who have failed prior therapies, including immune checkpoint blockade.
With 800 products in development for 5 of the most common blood cancers, the development pipeline for blood cancer therapies is getting crowded and will make it more challenging for biosimilars and newcomers to succeed.
Astellas launches a west coast cell and gene therapy hub with a $70M price tag | Financing
Astellas is planning a West Coast Innovation and Research Center in San Francisco, adding to its hub in Massachusetts and headquarters in Tokyo. This addition is likely to increase its penetration in markets across the US, making Astellas an even stronger player in cell therapy.
Gene Therapy
Freeline Therapeutics demonstrates sustained factor IX levels in hemophelia B patients | Clinical Trials
In an NEJM publication, Freeline Therapeutics reports results from their Ph1-2 trial of a treatment for hemophilia B. Patients demonstrated sustained factor IX levels at 26 weeks and are now enrolled in a long-term followup study.
Roche wades deeper into AAV therapies with biotech partnership | Partnership
Roche has entered a partnership with Avista Therapeutics for the development of novel gene therapies for ophthalmic diseases. Avista’s platform will be used to develop novel AAVs targeted towards retinal cells, delivered via intravitreal injection. Roche will conduct clinical studies of candidates and will retain licensing rights.
VBL Therapeutics’ AAV therapy flunks PhIII for ovarian cancer | Clinical Trials
VBL Therapeutics’ AAV candidate showed no survival benefit in an interim analysis of a Ph3 study in platinum-resistant ovarian cancer, leading to early termination of the trial.
AavantiBio latest company to announce layoffs impacting CMC group | Regulatory
AavantiBio has laid off 30 employees, whilst in the midst of preparing its first AAV gene therapy clinical study.
New study shows a potential of using AAVs to code and control biologics in-vivo | Research
A research group at the Abigail Wexner Research Institute at Nationwide Children’s Hospital have reported success in an animal model for a new drug delivery method. Their system enables in-vivo synthesis of an antibody therapeutic delivered via intravenous AAV.
Oligo Therapy
Roche will license and advance asset into a Phase 3 clinical study for immunoglobulin A nephropathy (IgAN) | Clinical Trials
Roche has licensed an investigational antisense therapeutic from Ionis Pharmaceuticals. The kidney disease treatment will be progressing into Phase 3 trials in patients with IgA nephropathy.
Kernal's secures a $25M series A to support its novel platform and IND filing | Financing
Kernal’s recent round provides financing for the company to further develop their mRNA 2.0 platform, and move towards their goal of filing an IND for their lead immuno-oncology candidate.
Vertex is tackling liver disease in partnership with Verve by putting $60M | Financing
Vertex and Verve have entered into an exclusive collaboration aimed at developing an in vivo gene editing program for liver disease. Verve will manage the preclinical development, with Vertex responsible for commercialization and funding.
Verve Therapeutics Doses First patient with its In Vivo Base Editing Medicine in Heterozygous Familial Hypercholesterolemia | Clinical Trials
Verve has initiated enrollment in their Ph1b trial of their gene therapy for cardiovascular disease. The drug candidate silences PCSK9 gene expression in the liver, reducing LDL-C levels.
Camp4 raises $100M to accelerate its expansion and advance lead into the clinic | Financing
Camp4’s latest financing round will support expansion of their regRNA mapping platform as well as preclinical development for their CNS and liver pipelines. An IND for their first neurological indication is expected by next year.
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