4th In Vivo Cell Engineering & Gene Editing Summit
pharmaphorum
APRIL 24, 2025
4th In Vivo Cell Engineering & Gene Editing Summit Sara.Scarpinati Thu, 24/04/2025 - 15:12 Bookmark this
Ginkgo Bioworks partners with WARF for new GD2 CAR T-cell therapies
Pharmaceutical Technology
APRIL 14, 2023
The company can use its mammalian cell engineering expertise and capabilities to screen CAR T-cells, to discover and optimise next-generation therapeutic candidates for its partners. All the new GD2 CAR designs developed through the partnership will be validated via in vitro screens and in vivo murine models.
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Umoja and IASO partner to develop therapies for haematological malignancies
Pharmaceutical Technology
NOVEMBER 22, 2022
In June last year, the company raised Series B funds worth $210m to advance its pipeline of in vivo engineered CAR T cell immunotherapies for treating solid tumours and hematologic malignancies into the clinic. In the initial stage, the partnership will focus on acute myeloid leukaemia (AML) to boost accessibility to patients.
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Neurons in spinal-cord injuries are reconnected in vivo via carbon nanotube sponges
Scienmag
SEPTEMBER 28, 2020
CIC biomaGUNE researchers have demonstrated the restoring in vivo of the connection between neurons, yielding functional results Credit: Author: Pedro Ramos / CIC biomaGUNE Research conducted by two groups at the Center for Cooperative Research in Biomaterials CIC biomaGUNE and one at SISSA, Scuola Internazionale Superiore di Studi Avanzati (Italy), (..)
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3rd In Vivo Cell Engineering & Gene Editing Summit
pharmaphorum
MARCH 19, 2024
Achieve Safe & Targeted In Vivo Delivery of Payloads with Clarity in Regulations & Appropriate Models to Advance the Next Frontier of In Vivo Editing from Research to Clinic
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Intellia Establishes Proof-of-Concept for in vivo CRISPR Bone Marrow Editing
BioSpace
MARCH 10, 2021
Intellia Therapeutics presented preclinical data of its non-viral genome editing platform at the Keystone eSymposium: Precision Engineering of the Genome, Epigenome and Transcriptome.
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BioMed X and Sanofi partner to leverage AI for drug development
Pharmaceutical Technology
OCTOBER 25, 2022
The companies will jointly create a virtual patient engine for the drug candidates’ clinical translation. The new group will become part of the 11 research teams at the BioMed X Institute, currently utilising complicated human ex-vivo models and data science in oncology, immunology, and neuroscience regions.
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New microrobot with in situ, in vivo bioprinting offers promise for gastric wounds
Scienmag
AUGUST 12, 2020
Researchers in China have taken the first step towards a new way of treating gastric wounds by using a microrobot combined with the new concept of “in situ in vivo bioprinting” to carry out tissue repair inside the body.
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Editas grabs orphan drug status for sickle cell disease CRISPR therapy
Pharmaceutical Technology
APRIL 28, 2023
EDIT-301 is made of patient-derived CD34+ haematopoietic stem and progenitor cells, which are edited using CRISPR at the gamma globin gene (HBG1 and HBG2) promoter sites using a proprietary engineered AsCas12a nuclease, per the company’s website. The first patient had a total haemoglobin level of 16.4 g/dL and 45.4%
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TFF and NIEHS partner to develop powder formulations for respiratory diseases
Pharmaceutical Technology
MAY 5, 2023
TFF Pharmaceuticals and NIEHS will investigate the TFF-HMW-HA formulations’ therapeutic efficacy and pharmacokinetics using in vitro and in vivo models of respiratory diseases.
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Scribe and Prevail partner for CRISPR-based genetic medicines
Pharmaceutical Technology
MAY 17, 2023
Scribe Therapeutics has entered a strategic collaboration with Eli Lilly and Company subsidiary Prevail Therapeutics for accelerating in vivo CRISPR-based therapies to target the causes of serious neurological and neuromuscular diseases. Editorial content is independently produced and follows the highest standards of journalistic integrity.
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Moderna partners with Life Edit for mRNA gene editing therapies
Pharmaceutical Technology
FEBRUARY 23, 2023
Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. Our novel editing systems have the potential to precisely modify gene targets for both in vivo and ex vivo therapeutic development.”
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Avista and Roche to develop gene therapy vectors for eyes
Pharmaceutical Technology
JULY 20, 2022
The alliance plans to leverage the single-cell adeno-associated virus (AAV) engineering (scAAVengr) platform technology of Avista to develop intravitreal AAV capsids for a Roche-defined profile. According to the deal, Roche holds the right to analyse and licence new capsids from Avista. Avista is entitled to receive $7.5m
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Scribe and Sanofi partner to develop cell therapies for cancer
Pharmaceutical Technology
SEPTEMBER 29, 2022
Under the agreement, Sanofi will receive non-exclusive rights to Scribe’s CRISPR by Design platform of wholly-owned enzymes for developing ex vivo NK cell therapies. The alliance will leverage the CRISPR genome editing technologies of Scribe to facilitate in genetic modification of new natural killer (NK) cell therapeutics for cancer.
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Intellia Gets FDA Clearance to Start First Ever Phase III Trial for an In Vivo CRISPR Drug
XTalks
OCTOBER 20, 2023
Intellia said NTLA-2001 is the first investigational in vivo CRISPR-based gene editing therapy cleared to enter late-stage clinical development. As an in vivo therapy, it can edit genes inside the body rather than in cells extracted from patients. ATTR amyloidosis is a rare, progressive and fatal disease.
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Aevitas to sell sCFH asset rights to 4D Molecular
Pharmaceutical Technology
APRIL 25, 2023
sCFH is an engineered and improved version of complement factor H (CFH) that can fit into adeno-associated virus (AAV) vectors with solid expression and applications confirmed in cultured human cells in vitro, in addition to various preclinical animal models in vivo.
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NKarta Therapeutics’ NKT Cells Elicit Better Response than CAR-T Cells Alone
BioSpace
FEBRUARY 18, 2021
NKarta Therapeutics is using engineered chimeric antigen receptor natural killer T cells (CAR-NKT cells) for allogeneic therapy, showing substantial benefits both in vitro and in vivo, according to James Trager, CSO, speaking Wednesday at the virtual CAR-TCR Summit – Europe.
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ESMO 2022: the evolution of TIL therapy – a highly promising approach
Pharmaceutical Technology
SEPTEMBER 14, 2022
As an autologous cell therapy, manufacturing the infusion product is a highly specialised process involving tumour harvest and the extraction and large-scale ex vivo expansion of TILs. The use of engineered immune cytokines that selectively induce the expansion of TILs is being investigated as an alternative to IL-2 stimulation.
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Artificial Intelligence innovation: Leading companies in in-silico drug discovery
Pharmaceutical Technology
FEBRUARY 23, 2023
Innovation S-curve for artificial Intelligence in the pharmaceutical industry In-silico drug discovery is a key innovation area in artificial Intelligence Drug research starts with the discovery stage that involves in vivo and in vitro models to shortlist lead drug compounds.
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RayzeBio raises $160m to advance cancer radiopharmaceuticals
Pharmaceutical Technology
SEPTEMBER 14, 2022
The company is also concluding more than 200 in vivo studies to gain insights and discover new drug candidates that could provide therapeutic radioisotopes for tumour-linked antigens. In addition, our broad discovery engine has generated several novel drug candidates for clinical evaluation in the near future.
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Implants could transform CAR-T therapy into one-day procedure
pharmaphorum
APRIL 25, 2022
At the moment, it can take weeks to complete the CAR-T process, which includes harvesting of T cells from the patient an shipment to a manufacturing unit, engineering, activation, and expansion of the cells, quality control checks and shipment back to the hospital for re-infusion into the patient.
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Eucure Biopharma and Chipscreen NewWay sign licence deal for YH008
Pharmaceutical Technology
FEBRUARY 28, 2023
It was engineered with an Fc-silent IgG1 isotype to avoid activation of Fc-receptor-mediated non-specific immune. The bispecific antibody was found to conditionally activate the immune pathway in the tumour microenvironment in vitro and in vivo trials. Topic sponsors are not involved in the creation of editorial content.
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Scribe Therapeutics Unveils Fully Integrated Platform for Engineering Breakthrough CRISPR-based Genetic Medicines
BioTech 365
OCTOBER 6, 2020
Built by CRISPR leaders including Jennifer Doudna to enable best-in-class in vivo therapeutics that permanently treat the underlying cause of disease Initial $20 million Series A led by Andreessen Horowitz Collaboration with Biogen Inc. focused on development of novel genetic … Continue reading →
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Smectite promotes probiotic biofilm formation in gut for cancer immunotherapy
Scienmag
FEBRUARY 10, 2021
Credit: Lei Dong, Nanjing University Scientists from Nanjing University and the University of Macau have devised a new approach to extend the survival of transplanted probiotics in vivo, enhancing the efficacy of cancer chemo-/immunotherapies in mice.
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Merck will assess Quris’ AI ‘patient-on-a-chip’ drug safety
pharmaphorum
FEBRUARY 13, 2022
Quris’ BioAI safety prediction platform – which is based on human tissue samples on chips, nanosensors and machine learning – will be compared to traditional in vitro and in vivo laboratory techniques for spotting toxicity. The post Merck will assess Quris’ AI ‘patient-on-a-chip’ drug safety appeared first on.
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Multi-Functional Cell Therapies Summit
pharmaphorum
FEBRUARY 23, 2021
The inaugural Multi- Functional Cell Therapies Summit gathers the avantgarde to discuss strategies to weaponize cell therapies with antigenic, engineered and combination approaches to create sophisticated, multi-functional systems. NKarta Therapeutics and Iovance Biotherapeutics.
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Digital therapeutics: a new frontier of medicine
pharmaphorum
JANUARY 25, 2022
For example, there is no need for in vivo animal or in vitro pre-clinical or non-clinical studies. The area of digital therapeutics is growing rapidly, and in this piece Ben Hargreaves examines why this is the case, how broadly these therapies can be applied and why investment in the area is mounting. This equated to $21.3
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Gene Editing Innovations with Programmable Gene Insertion (PGI) Technology ft. John Finn, PhD, CSO, Tome Biosciences – Xtalks Life Science Podcast Ep. 160
XTalks
MAY 22, 2024
He was most recently Vice President of Discovery Research at Codiak Biosciences, where he led the development of a new class of therapeutics based on engineered exosomes. In this episode, Ayesha spoke with John Finn, PhD, Chief Scientific Officer at Tome Biosciences , a company developing programmable gene insertion (PGI) technology.
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Bluebird’s $2.8M Gene Therapy Zynteglo Wins Landmark FDA Approval for Beta Thalassemia
XTalks
AUGUST 19, 2022
Zynteglo is administered as a one-time treatment, with each dose consisting of a patient’s own bone marrow stem cells engineered to make functional beta-globin through insertion of working copies of the beta-globin gene. The cells are prepared outside of the body ( ex vivo ) and then infused into the patient.
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Life Science Trends to Look Out for in 2024
XTalks
DECEMBER 20, 2023
As we step into 2024, the life sciences continue to evolve at an unprecedented pace, driven by technological innovation, a deeper understanding of human biology and the application of new technologies in areas like drug development and health wearables. Regulatory bodies are also taking note of the applications of AI in drug development.
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ViGeneron signs global collaboration agreement for ophthalmic gene therapy development
The Pharma Data
JANUARY 4, 2021
The companies will use ViGeneron’s proprietary vgAAV, novel engineered AAV capsids, to efficiently transduce retinal cells via intravitreal injections. The companies will work together on the in vivo proof of concept (POC). Gene therapy has become a clinical reality. For further information, please visit www.vigeneron.com.
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The 3rd Annual Blood-Brain Barrier Summit Returns in August 2021
pharmaphorum
APRIL 29, 2021
To achieve this, the agenda will allow delegates to: Explore the translation of BBB-penetrating antibodies to non-human primates , and new possibilities for in vivo brain imaging of antibody neuropharamacokinetics. A Meeting Point for the Field’s Experts. About Hanson Wade.
THE RISING REALM: BIOPRESERVATION MEDIA INDUSTRY
Roots Analysis
DECEMBER 8, 2021
Biopreservation is the exclusive biological approach designated for the preservation of cells, tissues and organs ex-vivo for the purpose of storage, shipment and transportation. Biopreservation media is one of the most crucial entity in conferring effectiveness. F) to 8°C (46.4°F).
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KSQ Therapeutics and Takeda Enter Broad Strategic Collaboration to Research, Develop and Commercialize Novel Immuno-Oncology Therapies
The Pharma Data
JANUARY 12, 2021
KSQ’s proprietary CRISPRomics® discovery engine enables genome-scale, in vivo validated, unbiased drug discovery across broad therapeutic areas. Working alongside KSQ will facilitate smart drug discovery and development of what we hope will be transformative new therapies for patients with intractable forms of cancer.”.
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Combining the Powers of Single Cell Sequencing and AI in Understanding Disease Biology and Drug Development
XTalks
AUGUST 5, 2020
Single cell sequencing has revolutionized the study of biological tissues and systems at the cellular and molecular level. Recent advances in the technology have allowed for the interrogation of distinct subsets of cell populations within tissues, and associated molecular markers that may function as important disease drivers.
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Assessing the Credibility of Computational Modeling and Simulation in Medical Device Submissions
FDA Law Blog
JANUARY 2, 2022
Lenz, Principal Medical Device Regulation Expert — On December 23, 2021, CDRH released as a draft guidance, Assessing the Credibility of Computational Modeling and Simulation in Medical Device Submissions (Draft Guidance). Draft Guidance at 4. Draft Guidance at 8. Draft Guidance at 5.
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iSTAR Medical’s glaucoma device MINIject shows positive one-year results in European trial
The Pharma Data
NOVEMBER 15, 2020
Pre-clinical studies of MINIject highlighting the anti-fibrotic properties of its STAR ® material were recently published in BMC Biomedical Engineering. . Promising MINIject data at two years from the STAR-I trial were recently published in the British Journal of Ophthalmology. medications at baseline).
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The Challenges and Promise of CAR T-Cell Therapy in Oncology
XTalks
SEPTEMBER 20, 2024
Such challenges highlight a significant unmet need and call for further advancements in the field. Vittoria Biotherapeutics is a clinical-stage cell therapy company that incorporates the latest advancements in cell therapy and gene editing with a unique approach that potentially overcomes some of the issues with this therapeutic modality.
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FDA’s New Draft Guidance on 510(k) Implant Devices: What You Need to Know
FDA Law Blog
OCTOBER 10, 2023
Implant devices are used in a range of settings such as dental and orthopedic procedures. Having a clear understanding of FDA’s data expectations for 510(k) applications related to implants is crucial for successful marketing clearance. The intended duration of implantation is another crucial factor in performance testing.
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Assessing the Credibility of Computational Modeling and Simulation in Medical Device Submissions
FDA Law Blog
JANUARY 2, 2022
Lenz, Principal Medical Device Regulation Expert — On December 23, 2021, CDRH released as a draft guidance, Assessing the Credibility of Computational Modeling and Simulation in Medical Device Submissions (Draft Guidance). Draft Guidance at 4. Draft Guidance at 8. Draft Guidance at 5.
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ImmunityBio’s hAd5 T-Cell COVID-19 Vaccine Candidate Shows Complete Protection of Airways in Non-Human Primates
The Pharma Data
DECEMBER 10, 2020
ImmunityBio has engineered the vector to overcome this problem and has shown that its second-generation adenovirus vector can safely and effectively deliver its cargo even in patients with pre-existing adenovirus immunity. This blocking of viral replication was observed in both the lung and nasal passages.
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CapstanTx secures $165 million to deliver precise in vivo cell engineering
pharmaphorum
SEPTEMBER 15, 2022
Yesterday, the biotechnology company Capstan Therapeutics launched with $165 million in seed and Series A funding for it to build on its foundational insights into precision in vivo engineering technology. Our ambition at Capstan is to invent new clinical paradigms through targeted in vivo reprogramming of cells.
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Kiromic Announces the Filing of Key European Patents for Its Chimeric PD-1 (chPD1) Target
The Pharma Data
JANUARY 4, 2021
Kiromic chPD1 has shown in preclinical data to show a cytotoxic response in 9 different in vivo models with 100% long-term PFS with the induction of host memory responses. chPD1 will be used in the Company’s proprietary chimeric antigen receptor therapy (CAR-T) platform using gamma-delta T-cells (GD-T). About Kiromic.
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40 Conjugation and Labeling of Molecules: A Burgeoning Approach in Therapeutics Development and Molecular Imaging
Roots Analysis
SEPTEMBER 13, 2023
The synergistic behavior achieved from the process circumvents the inherent limitation of the individual counterparts, such as short in vivo half-life, low solubility and immunogenicity. It consists of somatostatin-derived peptide and DOTA complexed with radioactive isotope 77 Lu.
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