On June 5, 2023 Compugen Ltd. (Nasdaq: CGEN) (TASE: CGEN) a clinical-stage cancer immunotherapy company and a pioneer in computational target discovery, reported data to be presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, showing that Compugen’s COM701 (anti-PVRIG) in triple combination with nivolumab and BMS-986207 (anti-TIGIT) demonstrated preliminary signal of durable anti-tumor activity in patients with recurrent, metastatic MSS endometrial cancer with a favorable safety profile. Preliminary translational data showed an association between greater peripheral immune activation and clinical benefit (Press release, Compugen, JUN 5, 2023, View Source [SID1234632466]). The poster presentation takes place today, June 5, 2023.

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"Despite recent advances in the standard of care for patients with recurrent, metastatic MSS endometrial cancer, there remains a significant need for additional treatment options for these patients," said Drew W. Rasco, M.D., Associate Director of Cancer Research at START Center for Cancer Care, San Antonio, Texas. "It is encouraging to see preliminary durable anti-tumor activity with a favorable safety profile in these patients treated with the triple combination of COM701, nivolumab and BMS-986207. Importantly, one-third of the patients included had prior exposure to an anti-PD-(L)1 making these a particularly hard to treat patient population with a significant unmet medical need. A patient with prior treatment refractory disease to standard of care lenvatinib and pembrolizumab, experienced a partial response and remained on treatment with triple combination for almost 7 months pointing to the potential contribution of blocking PVRIG and TIGIT to the benefit seen. It is also noteworthy that clinical benefit, defined as a partial response or stable disease greater than 100 days, correlated with peripheral immune activation as shown by the translational data to be presented."

Henry Adewoye, MD., Chief Medical Officer of Compugen, added, "I am delighted to see the preliminary data we have reported, including a confirmed partial response in a patient with endometrial carcinosarcoma, a rare hard-to-treat histologic type of endometrial cancer that is not typically responsive to standard of care therapies. The translational findings in patients with clinical benefit are also in line with the mechanistic rationale of the combination. Although the number of patients enrolled is small, these data and the clinical signal reported support our hypothesis of blocking the DNAM-1 axis with anti-PVRIG, TIGIT and PD-1 antibodies. This preliminary data complements previous data we presented in patients with hard-to-treat platinum resistant ovarian cancer and metastatic microsatellite stable colorectal cancer. It supports a COM701 mediated mechanism of action and further development of this triple immunotherapy combination as another potential option in treating cancer."

The abstract is published on the ASCO (Free ASCO Whitepaper) virtual platform and the publication section of Compugen’s website. The poster will be available today from 1:15pm CDT on the publication section of Compugen’s website.

On June 5, 2023 Coherus BioSciences, Inc. ("Coherus", Nasdaq: CHRS) reported the final overall survival (OS) results of the JUPITER-02 study presented today at the American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (Press release, Coherus Biosciences, JUN 5, 2023, View Source [SID1234632465]). JUPITER-02 is a randomized, double-blind, placebo-controlled Phase 3 clinical trial evaluating toripalimab in combination with gemcitabine and cisplatin as a first-line treatment for patients with recurrent or metastatic nasopharyngeal carcinoma (NPC).

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The final analysis of the JUPITER-02 clinical trial (NCT03581786) demonstrated a statistically significant and clinically meaningful improvement in OS for NPC patients who were treated with toripalimab in combination with gemcitabine and cisplatin chemotherapy, versus chemotherapy treatment alone. Median OS has not been reached in the toripalimab arm versus 33.7 months for chemotherapy treatment alone, HR=0.63 (95% CI 0.45-0.89); P=0.0083.

"There are limited options for patients living with this very aggressive form of head and neck cancer. These results are remarkable, with meaningful improvement over chemotherapy alone, and this is practice-changing," said Jennifer Choe, M.D., Ph.D., Assistant Professor of Medicine and the Head and Neck Medical Oncology Disease Team Lead in the Division of Hematology and Oncology at Vanderbilt University Medical Center. "I was impressed with the primary endpoint outcome, extending progression-free survival from 8.2 months with chemotherapy alone to 21.4 months with the addition of toripalimab, and now with the strong overall survival data, this will be a new standard of care for patients, if approved."

Rosh Dias, M.D., Coherus’ Chief Medical Officer, echoed Dr. Choe’s remarks, "This landmark trial demonstrates a significant survival advantage for patients with recurrent or metastatic nasopharyngeal carcinoma who were treated with toripalimab. No other PD-1 inhibitor currently approved in the U.S. has had a successful Phase 3 trial in recurrent or metastatic NPC that met its primary endpoints. Toripalimab, therefore, addresses an important unmet need for patients with NPC for whom there are currently no approved immunotherapies in the United States, and we look forward to bringing this important medicine to patients upon approval."

The Biologics License Application (BLA) for toripalimab in combination with chemotherapy as treatment for recurrent or metastatic nasopharyngeal carcinoma is currently under review by the U.S. FDA. In May 2023, FDA completed the required pre-licensing inspection of partner Shanghai Junshi Biosciences Ltd.’s toripalimab manufacturing site in China, and Coherus continues to collaborate with FDA to complete the clinical site inspections. Coherus projects potential approval in the third quarter of 2023 and plans to launch toripalimab in the United States directly, if approved.

Toripalimab JUPITER-02 Overall Survival Results

JUPITER-02 is a Phase 3, randomized study which examined the use of toripalimab versus placebo in combination with gemcitabine and cisplatin as a first-line treatment in recurrent/metastatic NPC.

At the final OS analysis, the median overall survival follow-up time was 36.0 months. A significant improvement in OS was observed in the toripalimab arm, and median OS was not yet reached, compared to 33.7 months in the placebo arm. The 2-year and 3-year OS rates for toripalimab + chemotherapy versus placebo + chemotherapy were 78.0% vs. 65.1%, and 64.5% vs. 49.2% respectively.

Coherus-Final OS Toripalimab Figure 2 ASCO (Free ASCO Whitepaper) 2023

A consistent effect on OS, favoring the toripalimab arm, was observed in nearly all subgroups, including PD-L1 high and PD-L1 low expression subgroups.
No new safety signals were identified in the toripalimab arm since the interim report. The incidence of Grade ≥3 adverse events (AEs) (89.7% vs 90.2%) and fatal AEs (3.4% vs 2.8%) were similar between the two arms. AEs leading to discontinuation of toripalimab versus placebo (11.6% vs 4.9%), immune-related (irAEs) (54.1% vs. 21.7%) and Grade ≥3 irAEs (9.6% vs. 1.4%) were more frequent in the toripalimab arm.

These data were presented in a poster discussion session today by Prof. Rui-hua XU, Sun Yat-sen University Cancer Center, JUPITER-02 principal investigator:

Abstract #6009/Poster #1: "Final Overall Survival Analysis of JUPITER-02: a Phase 3 study of Toripalimab versus Placebo in Combination with Gemcitabine and Cisplatin as First-line Treatment for Recurrent or Metastatic Nasopharyngeal Carcinoma (NPC)"

Commitment to the NPC Community
As there are limited resources for patients living with NPC and their caregivers, Coherus has launched a new educational resource, NPCFacts.com, for patients living with NPC which includes detailed information about the types of NPC, the causes, diagnosis, and current treatment options.

In addition to education about nasopharyngeal carcinoma, the website includes links to patient advocacy organizations providing additional resources for patients and their caregivers. Head and Neck Cancer Alliance, Support for People with Oral Head and Neck Cancer (SPOHNC), and Thyroid Head and Neck Cancer Foundation (THANC) are some of the organizations included on the website.

The website also includes a companion website for healthcare professionals treating patients with NPC with educational resources and peer-to-peer education.

Additional toripalimab clinical data presented at ASCO (Free ASCO Whitepaper) include:

CHOICE-01, NEOTORCH, and Torchlight Studies Demonstrate Clinical Benefit of Toripalimab in NSCLC and TNBC
Abstract # 9003: "Final overall survival and biomarker analyses of CHOICE-01: A double-blind randomized phase 3 study of toripalimab versus placebo in combination chemotherapy for advanced NSCLC without EGFR/ALK mutations."

A significant improvement in OS was observed for the toripalimab arm over the placebo arm: HR=0.73 (95% CI: 0.57-0.93), two-sided p=0.0108, median OS 23.8 vs 17.0 months
Abstract #8501: "Perioperative toripalimab + platinum-doublet chemotherapy vs. chemotherapy in resectable stage II/III non-small cell lung cancer (NSCLC): Interim event-free survival (EFS) analysis of the phase 3 NEOTORCH study."

Event free survival (EFS) was significantly improved in the toripalimab arm, HR=0.40, 95% CI (0.277-0.565), P<0.0001, and crossed the pre-specified efficacy boundary
The median EFS was not reached in the toripalimab arm and was 15.1 months in the placebo arm
Abstract #LBA1013: "TORCHLIGHT: a randomized, double-blind, phase III trial of toripalimab versus placebo, in combination with nab-paclitaxel (nab-P) for patients with metastatic or recurrent triple-negative breast cancer (TNBC)"

A statistically significant improvement in PFS was demonstrated for the toripalimab arm in the PD-L1 positive subgroup (mPFS 8.4 vs 5.6 months; HR = 0.653, 95% CI 0.470-0.906, P = 0.0102).
PFS in the ITT population showed a similar trend (mPFS 8.4 vs 6.9 months, HR = 0.773, 95%CI 0.602-0.994)
OS showed a trend towards improved OS in the PD-L1 positive (mOS 32.8 vs 19.5 months; HR = 0.615, 95%CI 0.414-0.914)
More than 20 toripalimab abstracts were accepted for ASCO (Free ASCO Whitepaper) 2023, including two selected for oral presentations (LBA1013 and #8501), describing the antitumor activities observed from various cancers of the nasopharynx, hypopharynx, skin, breast, lung, esophagus, stomach, liver, biliary duct, sarcoma, urothelial, head and neck, endometrial, thyroid, laryngeal, and pancreas.

Coherus Continues to Innovate to Address Unmet Patient Need in Cancer Patients with U.S. Launch of UDENYCA Autoinjector
Coherus recently announced the introduction of a prefilled autoinjector presentation of UDENYCA (pegfilgrastim-cbqv) in the United States. UDENYCA is designed to be administered the day after chemotherapy to decrease the incidence of infection as manifested by febrile neutropenia. The autoinjector has a streamlined, easy-to-use design for both in-clinic and at-home care settings, and features push-on-skin activation to reliably deliver a complete dose.

About toripalimab
Toripalimab is an anti-PD-1 monoclonal antibody that blocks PD-L1 binding to the PD⁠-⁠1 receptor at a unique site with high affinity and activates antitumor immunity demonstrating improvement in the overall survival of cancer patients in several tumor types. The BLA for toripalimab in combination with chemotherapy as treatment for recurrent or metastatic nasopharyngeal carcinoma is currently under review by the FDA. The FDA granted Breakthrough Therapy designation for toripalimab in combination with chemotherapy for the first-line treatment of recurrent or metastatic NPC in 2021 as well as for toripalimab monotherapy in the second or third-line treatment of recurrent or metastatic NPC in 2020. Additionally, the FDA has granted Fast Track designation for toripalimab for the treatment of mucosal melanoma and Orphan Drug designations for the treatment of ESCC, NPC, mucosal melanoma, soft tissue sarcoma, and small-cell lung cancer (SCLC).

More than 30 company-sponsored toripalimab clinical studies covering over fifteen indications have been conducted globally by Junshi Biosciences, including in China, the United States, Southeast Asia, and European countries. Ongoing or completed clinical trials evaluating the safety and efficacy of toripalimab cover a broad range of tumor types including cancers of the lung, nasopharynx, esophagus, stomach, bladder, breast, liver, kidney, and skin.

About UDENYCA
UDENYCA is the only pegfilgrastim brand offering two on-demand options—prefilled syringe (PFS) and autoinjector (AI) providing more choice and flexibility customized to meet the needs of patients and practices. UDENYCA (pegfilgrastim-cbqv) administered via a proprietary on-body injector (OBI) device is under review by the FDA. If approved, UDENYCA OBI would offer providers a highly desired alternative to the originator’s on-body pegfilgrastim delivery system. Since 2019, over 263,000 patients have been treated with UDENYCA

On June 5, 2023 Chimeric Therapeutics (ASX:CHM, "Chimeric" or the "Company"), the only clinical stage cell therapy company on the ASX, reported activation of a Phase 1B clinical trial in patients with recurrent and/ or progressive glioblastoma multiforme (GBM) to assess the safety and efficacy of CHM 1101, the company’s first in class CLTX CAR T cell therapy (Press release, Chimeric Therapeutics, JUN 5, 2023, View Source [SID1234632464]). (ClinicalTrials.gov Identifier: NCT05627323)

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The trial is now open for enrollment at the Sarah Cannon Transplant & Cellular Therapy Program at St. David’s South Austin Medical Center in Austin, Texas.

"We are very excited to be activating the first site in our CHM 1101 Phase 1B clinical trial as it marks a new chapter in the development of CHM 1101," said Jennifer Chow, CEO and Managing Director of Chimeric Therapeutics. "This multi-center trial will enable us to more rapidly advance the development of CHM 1101 with recruitment across multiple clinical trial sites and also prepare us to accelerate the next phase of development if supported by the clinical results."

This Phase 1B trial, being conducted under a US IND, is a two-part clinical trial designed to determine a recommended Phase 2 dose and administration schedule. Part A of the trial will enroll 3-6 patients at the highest dose tested in the ongoing clinical trial at City of Hope Cancer Centre. In late 2023, Chimeric will assess the clinical safety and activity from the CHM 1101 clinical program. Based on a favorable review of the results of that assessment, Part B of the trial, a dose expansion cohort, will be opened to enroll 12 to 26 additional patients. Upon successful completion of the Part B dose expansion cohort, the Company intends to design and initiate a registration trial, in collaboration with global regulatory feedback.

"We’re very pleased to be building upon the City of Hope investigator-initiated trial and advancing CHM 1101 to a multi-center clinical trial. GBM continues to represent an important For personal use only unmet medical need and the early clinical results from the City of Hope trial provide support that CHM 1101 may improve outcomes for GBM patients," said Jason B Litten MD, Chief Medical Officer, Chimeric Therapeutics. CHM 1101 demonstrated safety with ~70% disease stability in the initial two dose cohorts in the City of Hope Phase 1A investigator-initiated clinical trial.

Additional details on the CHM Phase 1B trial design and objectives were presented on June 3 at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting as part of the Central Nervous System Tumors section as abstract TPS2086/Poster 440a, "Phase 1b multicenter study to evaluate CHM 1101 in patients with recurrent or progressive glioblastoma".

About CHM 1101:

CHM 1101 (CLTX CAR T) is a first-in-class CAR T therapy that has the potential to address the high unmet medical need of patients with recurrent or progressive glioblastoma. Research to develop the intellectual property covering this CAR T cell therapy took place at City of Hope. CHM 1101 cells uniquely utilize chlorotoxin (CLTX), a peptide component of scorpion venom, as the tumour-targeting component of the chimeric antigen receptor (CAR).

CHM 1101 CAR T cells have been shown in preclinical models to bind more broadly and specifically to GBM cells than other targeting domains like EGFR, HER-2 or IL-13. In preclinical models, CHM 1101 cells also demonstrated potent antitumor activity against glioblastoma while not exhibiting any off-tumor recognition of normal human cells and tissues, indicating a potentially optimal safety and efficacy profile.

CHM 1101 is currently being studied in a phase 1B clinical trial in recurrent / progressive glioblastoma. Initial positive data from the investigator-initiated phase 1A trial has been presented on patients treated in the first two dose levels of the trial.

On June 5, 2023 Bicara Therapeutics, a clinical-stage biotechnology company developing dual-action biologics to elicit a potent and durable immune response, reported that it will present positive interim data from its ongoing, open-label Phase 1/1b dose expansion study of BCA101, a first-in-class bifunctional EGFR/TGF-β antibody, at the 2023 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (Press release, Bicara Therapeutics, JUN 5, 2023, View Source;utm_medium=rss&utm_campaign=bicara-therapeutics-first-in-class-bifunctional-egfr-tgf-%25ce%25b2-inhibitor-bca101-demonstrates-65-orr-in-combination-with-pembrolizumab-in-1l-hpv-negative-recurrent-metastatic-head-and-neck [SID1234632463]). In the Phase 1/1b study, BCA101 in combination with pembrolizumab demonstrated a 65% overall response rate (ORR) in frontline, human papillomavirus (HPV)-negative, recurrent/metastatic (R/M) head and neck squamous cell carcinoma (HNSCC), with a tolerable safety profile.

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"Head and neck squamous cell carcinoma has limited treatment options and generally carries a poor prognosis, particularly for those with HPV-negative disease, which represent the majority of patients in the R/M setting," said Glenn J. Hanna, M.D., Center for Head and Neck Oncology, Dana-Farber Cancer Institute, and principal investigator for the Phase 1/1b clinical trial of BCA101. "These data demonstrate exciting and clinically meaningful activity among first-line R/M HPV-negative HNSCC patients and underscore the potential for BCA101 in combination with pembrolizumab in this underserved patient population."

"A 65% ORR in front-line HPV-negative R/M HNSCC is a significant improvement over pembrolizumab monotherapy and we are thrilled to be showcasing these data in an oral presentation at ASCO (Free ASCO Whitepaper)," said Claire Mazumdar, Ph.D., MBA, chief executive officer of Bicara Therapeutics. "HNSCC is one of the most common cancers worldwide, with increasing prevalence, and represents a significant unmet need. With this promising proof-of-concept data, we look forward to advancing BCA101 in combination with pembrolizumab in frontline R/M HNSCC, while also continuing to explore the utility of BCA101 in other cancer types."

Presentation Highlights:

Interim data (May 22, 2023 cut-off date) from the Phase 1/1b dose expansion cohort include 31 evaluable R/M HNSCC patients. None of the patients received prior systemic therapy and had a PD-L1 combined positive score (CPS) of ≥1. 20 patients were human papillomavirus (HPV)-negative and 11 patients were HPV-positive.
65% ORR in HPV-negative population (13/20 patients), including 12 confirmed partial responses (PR) and one confirmed complete response (CR), with responses observed across different degrees of PD-L1 expression (CPS 1-19 (5/10, 50%) and CPS ≥20 (8/10, 80%)) and varying disease subgroups (distant metastatic (9/14, 64%) and loco-regional disease (4/6, 67%)).
Preliminary median progression free survival (mPFS) in HPV-negative patients (in stage 1) of at least 6.6 months with 6/12 patients ongoing.
48% ORR in total evaluable population (15/31 patients).
Tolerable safety profile with the most common treatment-related adverse events (TRAEs). including acneiform rash (73%, with majority being Grade 1), fatigue (36%), hypophosphatemia (36%) and anemia (30%).
Presentation Details:

Title: Dose expansion results of the bifunctional EGFR/TGF-β inhibitor BCA101 with pembrolizumab in patients with recurrent, metastatic head and neck squamous cell carcinoma.
Presenter: Glenn J. Hanna, M.D.
Abstract Number: 6005
Session Type/Title: Oral Abstract Session – Head and Neck Cancer
Date/Time: June 5, 2023 from 8:00-11:00 a.m. CT
Location: McCormick Place Convention Center, Chicacgo, IL

In addition, Bicara will be hosting a webinar with two leading head and neck oncologists, Glenn J. Hanna, M.D., Center for Head and Neck Oncology, Dana-Farber Cancer Institute,and Ezra Cohen, M.D., FRCPSC, FASCO, University of California San Diego. The webinar will be made available on Bicara’s website shortly after today’s ASCO (Free ASCO Whitepaper) oral presentation at www.bicara.com/news/.

About Head and Neck Squamous Cell Carcinoma
Head and neck squamous cell carcinomas (HNSCCs) develop from the mucosal epithelium in the oral cavity, pharynx and larynx and are the most common malignancies that arise in the head and neck.

Oral cavity and larynx cancers are generally associated with tobacco consumption, alcohol abuse or both, whereas pharynx cancers are increasingly attributed to infection with human papillomavirus (HPV), primarily HPV-16. Thus, HNSCC can be biologically separated into HPV-negative and HPV-positive HNSCC, the latter carrying a more favorable prognosis. Treatment approaches for locally advanced HNSCC generally consist of surgery followed by chemoradiotherapy (CRT) for oral cavity cancers and primary or definitive CRT for pharynx and larynx cancers. The immune checkpoint inhibitors pembrolizumab and nivolumab are approved by the U.S. FDA for treatment of platinum-refractory recurrent or metastatic HNSCC, and pembrolizumab is approved as first-line monotherapy in patients with unresectable or metastatic disease with a CPS ≥1 or combined with platinum and 5-fluorouracil for patients with any CPS score.

HNSCC is the sixth most common cancer worldwide, with approximately 890,000 new cases and 450,000 deaths in 2018. The incidence of HNSCC continues to rise and is anticipated to increase by 30% by 2030.1

About BCA101
BCA101 is a first-in-class, dual-action, bifunctional antibody designed to inhibit the epidermal growth factor receptor (EGFR) and disable transforming growth factor beta (TGF-β) directly at the tumor site. This approach allows BCA101 to inhibit tumor proliferation, while restoring the cytolytic activity of the local immune cells.

BCA101 is currently being evaluated in a dose expansion phase of an open-label Phase 1/1b study as a monotherapy for cutaneous squamous cell carcinoma and in combination with pembrolizumab in patients with unresectable R/M HNSCC and advanced squamous non-small cell lung cancer (SqNSCLC).

On June 5, 2023 Bayer AG reported to have joined forces with Acuitas Therapeutics, Inc., a biotechnology company specializing in the development of lipid nanoparticle (LNP) delivery systems for molecular therapeutics (Press release, Bayer, JUN 5, 2023, View Source [SID1234632462]). Acuitas’ LNP technology will support Bayer’s in vivo gene editing and protein replacement programs by specifically delivering RNA payloads to the desired target organ, the liver.

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LNPs are spheric drug delivery bodies which can be equipped with therapeutic payloads for intracellular delivery. Acuitas’ proprietary LNP technology is used in multiple vaccines and therapeutics in clinical development and was also used in some of the COVID-19 vaccines that were approved and administered to people in 180 countries. This delivery technology protects the messenger RNA (mRNA) payload after administration allowing it to be safely and effectively delivered into cells. In addition to mRNA, Acuitas LNP can be used to deliver a range of different nucleic acid therapeutics including small interfering RNA (siRNA), antisense oligonucleotides and DNA.

"Complementing in-house expertise with external collaboration continues to be a priority in areas of high unmet medical need where insufficient, or no treatment options are currently available," said Friedemann Janus, acting Head of Business Development and Licensing/Open Innovation, Pharmaceuticals Division, Bayer. "Accessing state-of-the-art LNP technology through this collaboration will add momentum to our gene editing efforts for the benefit of patients."

"Developing therapies at scale is fundamental to provide breakthrough innovations to patients who have no time to wait," said Jost Reinhardt, Head of Cell and Gene Therapy, Pharmaceuticals Division, Bayer. "Adding Acuitas’ clinically-validated and scalable LNP technology to our genomic medicine toolbox is another important step to advance our leadership in the field of cell and gene therapies."

"We are delighted to partner with Bayer in the area of gene therapy. Innovation is the foundation of who we are at Acuitas, and we continue to invest heavily in internal research and development to provide our partners – such as Bayer – with the safest and most effective LNP delivery technology available," said Dr. Thomas Madden, President & CEO of Acuitas Therapeutics. "We support our partners to advance new therapeutics to address unmet clinical needs, and we are excited to work with the Bayer team in the development of medicines that are intended to address serious health issues faced by people worldwide."

Through the development and option for license agreement, Bayer and its gene therapy focused affiliate Asklepios BioPharmaceutical (AskBio) will gain access to Acuitas’ high potency ionizable lipid technology and LNP carriers which will allow for efficient, targeted, and transient delivery of gene editing RNA components to the liver. Market-maturity combined with demonstrated manufacturing scalability will drive the development and has the potential to accelerate the path to the clinic of Bayer and AskBio’s first in vivo gene editing programs. Financial details were not disclosed.

About gene editing
Gene editing is the targeted manipulation of genetic material. It enables a range of edits to the DNA allowing a diverse range of therapeutic applications. Gene editing can be applied inside and outside the patient to treat a variety of diseases and provide diverse clinical benefits. Gene editing as therapeutic treatment of genetic diseases can be used ex vivo to treat genetic dysfunctions, e.g., sickle cell anemia where a patient’s cells are ex vivo modified and afterwards re-administered, or in vivo, where modifications are made directly within the human body. To deliver the different components needed for in vivo gene editing to the right place in the patient’s body, transport vehicles, such as LNPs can be used.