Affini-T Therapeutics, Inc., headquartered in Watertown, MA, is a biotechnology company specializing in the development of T-cell therapies for cancer patients. Founded by Arjun Goyal and Jak Knowles, the company is dedicated to harnessing the potential of T cells and focusing on core oncogenic drivers to create therapies with the potential for curing solid tumor cancers. Affini-T's distinctive cell therapy platform utilizes advanced engineering and synthetic biology capabilities to target challenging cancer-driving mutations, starting with KRAS. The company employs these tools to optimize T cell functions, reshaping the dynamics of the solid tumor microenvironment with the goal of achieving sustained clinical outcomes for patients.

Tags

Neoplasms

Endocrinology and Metabolic Disease

Digestive System Disorders

TCR therapy

Bispecific T-cell Engager (BiTE)

Disease domain score

A glimpse into the focused therapeutic areas

Technology Platform

Most used technologies in drug development

Targets

Most frequently developed targets

Disease Domain	Count

Neoplasms	5
Endocrinology and Metabolic Disease	1

Top 5 Drug Type	Count

TCR therapy	5
Bispecific T-cell Engager (BiTE)	1

Top 5 Target	Count

KRAS G12V(GTPase KRas G12V)	2
CD3 x p53 R175H	1
KRAS G12C x KRAS G12D x PI3Kα x p53 R175H	1
KRAS G12C(GTPase KRas G12C)	1

Drugs associated with Affini-T Therapeutics, Inc.

AFNT-211

Target

KRAS G12V

Mechanism

KRAS G12V inhibitors

Active Org.

Affini-T Therapeutics, Inc.Startup

Originator Org.

Affini-T Therapeutics, Inc.Startup

Active Indication

Colorectal Cancer [+3]

Inactive Indication-

Drug Highest PhasePhase 1/2

First Approval Ctry. / Loc.-

First Approval Date-

Oncogenic Driver Program: Mutan P53 R175H(Affini-T Therapeutics, Inc.)

Target

CD3 x p53 R175H

Mechanism

CD3 stimulants [+1]

Active Org.

Memorial Sloan Kettering Cancer Center [+1]

Originator Org.

Affini-T Therapeutics, Inc.Startup

Active Indication

Neoplasms

Inactive Indication-

Drug Highest PhasePreclinical

First Approval Ctry. / Loc.-

First Approval Date-

Oncogenic Driver Program: KRAS G12C (Affini-T Therapeutics, Inc)

Target

KRAS G12C

Mechanism

KRAS G12C inhibitors

Active Org.

Affini-T Therapeutics, Inc.Startup

Originator Org.

Affini-T Therapeutics, Inc.Startup

Active Indication

Neoplasms

Inactive Indication-

Drug Highest PhasePreclinical

First Approval Ctry. / Loc.-

First Approval Date-

Clinical Trials associated with Affini-T Therapeutics, Inc.

NCT06105021 / RecruitingPhase 1/2

Phase I Study of Autologous CD8+ and CD4+ Engineered T Cell Receptor T Cells in Subjects With Advanced or Metastatic Solid Tumor

This study is open to adult patients with solid tumors who have a KRAS G12V mutation. This mutation is often found in non-small cell lung cancer (NSCLC), colorectal cancer (CRC), pancreatic ductal adenocarcinoma (PDAC) and other cancers. The study is for patients whose cancer has spread through the body and for whom previous treatments were not successful or treatment does not exist. Patients must also be positive for HLA-A*11:01. The purpose of this study is to find the best dose of AFNT-211 that is safe and can shrink tumors in patients. AFNT-211 is an investigational therapy and this is the first time that AFNT-211 is being administered to patients. AFNT-211 is an autologous T cell product which means that it is made from a patient's own T cells. These cells are engineered and grown to recognize the KRAS G12V protein on the cell surface of cancer cells. AFNT-211 is infused into patients after a short course of lymphodepleting chemotherapy. Patients will frequently visit the study site. The doctors there will regularly check the size of the cancer and the patient's health. They will also take note of any unwanted effects. Patients may continue in this study for as long as they benefit from the treatment.

Start Date06 Mar 2024

Sponsor / Collaborator

Affini-T Therapeutics, Inc.Startup

NCT06043713 / RecruitingPhase 1IIT

Phase I Study of Autologous CD8+ and CD4+ Transgenic T Cells Expressing High Affinity KRASG12V Mutation-Specific T Cell Receptors in Participants With Metastatic Pancreatic, Colorectal and Non-Small Cell Lung Cancers With KRAS G12V Mutations

This phase I trial studies the side effects and best dose of autologous CD8+ and CD4+ transgenic T cells expressing high affinity KRAS G12V mutation-specific T cell receptors (FH-A11KRASG12V-TCR) and to see how well they work in treating patients with pancreatic, colorectal, and non-small cell lung cancers that has spread from where it first started (primary site) to other places in the body (metastatic). T cells are infection fighting blood cells that can kill tumor cells. The T cells given in this study will come from the patient and will have a new gene put in them that makes them able to recognize KRAS G12V, a protein on the surface of tumor cells. These KRAS G12V-specific T cells may help the body's immune system identify and kill KRAS G12V pancreatic, colorectal, and non-small cell lung cancers' tumor cells.

Start Date15 Dec 2023

Sponsor / Collaborator

Fred Hutchinson Cancer Research Center

[+1]

NCT03747484 / TerminatedPhase 1/2IIT

Phase I/II Study of Autologous CD8+ and CD4+ Transgenic T Cells Expressing High Affinity MCPyV-Specific TCRs Combined With Avelumab and Class I MHC-Upregulation in Patients With Metastatic MCC Refractory to PD-1 Axis Blockade

This phase I/II trial studies the side effects of gene-modified immune cells (FH-MCVA2TCR) and to see how well they work in treating patients with Merkel cell cancer that has spread to other parts of the body (metastatic) or that cannot be removed by surgery (unresectable). Placing a gene that has been created in the laboratory into immune cells may improve the body's ability to fight Merkel cell cancer.

Start Date03 Jul 2019

Sponsor / Collaborator

Fred Hutchinson Cancer Research Center

[+2]

100 Clinical Results associated with Affini-T Therapeutics, Inc.

0 Patents (Medical) associated with Affini-T Therapeutics, Inc.

Literatures (Medical) associated with Affini-T Therapeutics, Inc.

01 Jun 2024·Journal of Clinical Oncology

AFNT-211: A phase 1 study of autologous CD4+ and CD8+ T cells engineered to express a high avidity HLA-A*11:01-restricted, KRAS G12V-specific, transgenic TCR, a CD8α/β coreceptor, and a FAS41BB switch receptor in patients with advanced/metastatic solid tumors.

Author: Khan, Binaish ; Chiorean, E. Gabriela ; Punekar, Salman Rafi ; Lenz, Heinz-Josef ; Zhao, Dan ; Pelster, Meredith ; Nagorsen, Dirk ; Vallaster, Markus P ; Hecht, J. Randolph ; Payumo, Francis ; Mitchell, Shaunica ; Hurwitz, Michael E. ; Schoenfeld, Adam Jacob ; Leidner, Rom ; Gahvari, Zhubin

TPS8650 Background: Activating mutations in KRAS (including KRAS G12V) are well-described oncogenic drivers in solid tumors. Patients with KRAS driver mutations have a poor prognosis, and most KRAS-mutated cancers lack effective therapies. T cell receptor (TCR)-T cell therapies targeting mutant KRAS have demonstrated proof of concept in the clinic, but duration of response remains a challenge. AFNT-211 represents a novel strategy to address the immunosuppressive tumor microenvironment and improve response rate and duration in solid tumors. AFNT-211 autologous CD4+ and CD8+ T cells are engineered to express a high avidity HLA-A*11:01-restricted, KRAS G12V-specific, transgenic TCR; the wildtype CD8α/β coreceptor; and a FAS-41BB switch receptor. The intended mechanism of action is recognition and elimination of KRAS G12V-mutated tumor cells by AFNT-211. AFNT-211 is designed to enhance anti-tumor activity via the following mechanisms of action as shown in preclinical models: (1) the CD8α/β coreceptor enables MHC-I– restricted TCR recognition of the KRAS G12V target by CD4+ T cells; (2) engaging CD4+ helper T cell responses drives enhanced CD8+ T cell cytotoxic activity and prevents T cell exhaustion; (3) the FAS-41BB chimeric switch receptor drives increased T cell activity, circumventing native FAS apoptotic signaling. Methods: This ongoing Phase 1, first-in-human, multicenter, open-label study of AFNT-211 (1) evaluates safety/tolerability, (2) determines the optimal biological dose (OBD) and recommended Phase 2 dose (RP2D), and (3) assesses preliminary antitumor activity. The study consists of 2 parts: dose escalation and dose expansion. Dose escalation is guided by the Bayesian optimal interval Phase 1/2 (BOIN12) design to determine the OBD. The BOIN12 design quantifies the desirability of a dose in terms of toxicity-efficacy tradeoff and adaptively allocates patients to the dose with the highest estimated desirability. The RP2D will be selected based on the BOIN12 design and the totality of benefit/risk assessment. Patients enrolling in dose expansion will be treated at the OBD/RP2D in indication-specific cohorts. This study is conducted in patients aged ≥ 18 years who are HLA-A*11:01-positive with advanced/metastatic solid tumors harboring a KRAS G12V mutation, and intolerance of/progression on at least 1 prior systemic therapy. Patients undergo leukapheresis to collect T cells for the manufacturing of AFNT-211 and receive lymphodepleting chemotherapy prior to AFNT-211 infusion. This is followed by a 28-day dose-limiting toxicity observation period (dose escalation only) and a post-treatment follow-up period for 24 months/ until disease progression. The study is open for recruitment in the United States. Clinical trial information: NCT06105021 .

01 Jun 2024·Journal of Clinical Oncology

Correlation of allele-specific HLA loss of heterozygosity in patients with cancer with driver mutations.

Author: Jindal, Aastha ; Roy, Christian ; Vallaster, Markus P. ; Klebanoff, Christopher Austin ; Halvey, Patrick ; Nagorsen, Dirk ; Garrity, Sean ; Sewastianik, Tomasz ; Opiteck, Gregory ; Shapiro, Gary ; Montesion, Meagan ; Vincent, Loic ; Lam, Hubert

e14658 Background: While immunotherapies have revolutionized cancer treatment, the limited predictability of response and resistance mechanisms present challenges to the development of novel therapeutics for solid tumors. Many of these use a sensitive and specific T cell receptor (TCR)-HLA:peptide interaction as a mechanism of action and can be impacted by HLA loss of heterozygosity (LOH). Allele-agnostic HLA LOH analyses assessing loss of any MHC class I allele are relevant for HLA-agnostic approaches such as immune checkpoint inhibitors; however, context-specific studies remain limited. For example, adoptive TCR T cell therapies and bi-specific TCR T cell engagers target neoantigen peptides derived from oncogenic driver mutations, such as those of KRAS and TP53, presented by a specific HLA allele. Thus, allele-agnostic analyses may overestimate the frequency and relevance of HLA LOH for allele-specific modalities. Methods: A real-world comprehensive genomic profiling dataset consisting of 78,418 cases (1), germline heterozygous for one or more HLA class I locus, was analyzed for HLA LOH in the context of frequent driver mutations (KRAS G12C, G12D, G12V and TP53 R175H), the corresponding HLA alleles (A*02:01, A*03:01, and A*11:01), and within tumor subtypes. Results: Allele-agnostic HLA LOH was observed in 16.6% of all samples and varied between indications. Notably, allele-specific loss of HLAs A*02:01, A*03:01, and A*11:01 across all samples was much lower than allele-agnostic loss (6.3%, 6.6%, and 6.3% cases, respectively). Although, a trend for increased allele-specific HLA LOH was observed when analyzed by driver mutation, the frequency remained low. Specifically, across all cases, A*02:01, A*03:01, and A*11:01 allele-specific LOH ranged between 5.9% - 9.7% in relation to KRAS G12C, G12D, and G12V compared to 5.9% - 6.2% for wild-type (WT). Moreover, it did not achieve statistical significance for any HLA-KRAS mutation pairs in colorectal (CRC), non-small cell lung (NSCLC), and pancreatic (PDAC) cancers, except for A*11:01 and KRAS G12C in NSCLC, which was lower in mutated cases (5.3% for G12C vs 10.1% for WT KRAS, p=0.045). Both allele-agnostic and A*02:01-specific HLA LOH in TP53 WT samples were lower than average. The TP53 R175H mutation was associated with limited increase in HLA-A*02:01 LOH (from 4.4% for WT to 8.1% for TP53 R175H) and varied between indications, e.g. no significant increase was observed in R175H-positive breast cancer and NSCLC cases contrary to CRC and PDAC. Conclusions: Based on this real-world dataset, over 90% of cases retain the TCR-targeted allele and, therefore, preserve the molecular complex necessary for HLA/driver mutation-based therapies. Notably, the database is enriched for advanced cancer patients. Further studies should test correlations with treatments and whether tumors in earlier lines of treatment have an even lower rate of HLA LOH. 1. Montesion et al., PMID: 33127846.

05 Apr 2024·Cancer Research

Abstract CT076: AFNT-211: A phase1 study of autologous CD4+ and CD8+ T cells engineered to express a high avidity HLA-A*11:01-restricted, KRAS G12V-specific, transgenic TCR; CD8α/β coreceptor; and a FAS41BB switch receptor in patients with advanced/metastatic solid tumors

Author: Hurwitz, Michael ; Khan, Binaish ; Chiorean, E. Gabriela ; Lenz, Heinz-Josef ; Zhao, Dan ; Pelster, Meredith ; Nagorsen, Dirk ; Hecht, J. Randolph ; Payumo, Francis ; Schoenfeld, Adam ; Mitchell, Shaunica ; Leidner, Rom ; Gahvari, Zhubin ; Punekar, Salman ; Vallaster, Markus

AbstractBackground: Activating mutations in KRAS (including KRAS G12V) are well-described oncogenic drivers in solid tumors. Patients with KRAS driver mutations have a poor prognosis, and most KRAS-mutated cancers lack effective therapies. T cell receptor (TCR)-T cell therapies targeting mutant KRAS have demonstrated proof of concept in the clinic, but duration of response remains a challenge. AFNT-211 represents a novel strategy to address the immunosuppressive tumor microenvironment and improve response rate and duration in solid tumors.Rationale: AFNT-211 autologous CD4+ and CD8+ T cells are engineered to express a high avidity HLA-A*11:01restricted, KRAS G12V-specific, transgenic TCR; the wildtype CD8α/β coreceptor; and a FAS-41BB switch receptor. The intended mechanism of action is recognition and elimination of KRAS G12V-mutated tumor cells by AFNT-211. AFNT-211 is designed to enhance anti-tumor activity via the following mechanisms of action as shown in preclinical models: (1) the CD8α/β coreceptor enables MHC-I-‍restricted TCR recognition of the KRAS G12V target by CD4+ T cells; (2) engaging CD4+ helper T cell responses drives enhanced CD8+ T cell cytotoxic activity and prevents T cell exhaustion; (3) the FAS-41BB chimeric switch receptor drives increased T cell activity, circumventing native FAS apoptotic signaling.Methods: This ongoing Phase 1, first-in-human, multicenter, open-label study of AFNT-211 (1) evaluates safety/tolerability, (2) determines the optimal biological dose (OBD) and recommended Phase 2 dose (RP2D), and (3) assesses preliminary antitumor activity. The study consists of 2 parts: dose escalation and dose expansion. Dose escalation is guided by the Bayesian optimal interval Phase 1/2 (BOIN12) design to determine the OBD. The BOIN12 design quantifies the desirability of a dose in terms of toxicity-efficacy tradeoff and adaptively allocates patients to the dose with the highest estimated desirability. The RP2D will be selected based on the BOIN12 design and the totality of benefit/risk assessment. Patients enrolling in dose expansion will be treated at the OBD/RP2D in indication-specific cohorts. This study is conducted in patients aged ≥ 18 years who are HLA-A*11:01-positive with advanced/metastatic solid tumors harboring a KRAS G12V mutation, and intolerance of/progression on at least 1 prior systemic therapy. Patients undergo leukapheresis to collect T cells for the manufacturing of AFNT-211 and receive lymphodepleting chemotherapy prior to AFNT-211 infusion. This is followed by a 28-day dose-limiting toxicity observation period (dose escalation only) and a post-treatment follow-up period for 24 months/until disease progression. The study is open for recruitment in the United States (NCT06105021).Citation Format: Shaunica Mitchell, Binaish Khan, Francis Payumo, E. Gabriela Chiorean, Zhubin Gahvari, J. Randolph Hecht, Michael Hurwitz, Rom Leidner, Heinz-Josef Lenz, Meredith Pelster, Salman Punekar, Adam Schoenfeld, Dan Zhao, Markus Vallaster, Dirk Nagorsen. AFNT-211: A phase1 study of autologous CD4+ and CD8+ T cells engineered to express a high avidity HLA-A*11:01-restricted, KRAS G12V-specific, transgenic TCR; CD8α/β coreceptor; and a FAS41BB switch receptor in patients with advanced/metastatic solid tumors [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2024; Part 2 (Late-Breaking, Clinical Trial, and Invited Abstracts); 2024 Apr 5-10; San Diego, CA. Philadelphia (PA): AACR; Cancer Res 2024;84(7_Suppl):Abstract nr CT076.

News (Medical) associated with Affini-T Therapeutics, Inc.

29 Oct 2024

·endpts.com

Vera’s $300M offering; Abeona resubmits treatment for rare skin disease

Plus, news about Astellas, Clover, ElevateBio, Monopar, Idorsia and Neurocrine Biosciences: Vera Therapeutics plans $300M offering: The California biotech is selling shares after presenting Phase 2b data for an experimental drug called atacicept over the weekend. The company shared 96-week data for patients with immunoglobulin A nephropathy. — Kyle LaHucik Abeona Therapeutics resubmits rare skin disease therapy: Back in April, the FDA rejected Abeona’s cell therapy, called pz-cel, requesting more information about manufacturing. Abeona is developing pz-cel for recessive dystrophic epidermolysis bullosa, a rare genetic skin disease. — Lei Lei Wu Astellas yanks Izervay’s submission in Europe: The company said it withdrew its marketing authorization application for Izervay, a C5 protein inhibitor for geographic atrophy secondary to age-related macular degeneration. Astellas did not detail feedback from the Committee for Medicinal Products for Human Use, but said it is still “confident” in Izervay’s risk-benefit profile and will continue to engage with regulators in Europe and elsewhere. There are currently no treatments approved for GA secondary to AMD outside of the US, where Izervay was greenlit in August of last year. — Ayisha Sharma Clover releases early RSV vaccine data: The Shanghai-based biotech said its bivalent vaccine candidate, SCB-1019, produced “comparable” levels of RSV neutralizing antibodies to GSK’s Arexvy at 28 days in a Phase 1 test. Based on these data, Clover plans to advance the asset into studies focused on re-vaccination next year. — Ayisha Sharma ElevateBio lays off 4%: The genetic medicines company, which employs about 500 people, is ending its preclinical cellular engineering work, a spokesperson told Endpoints News on Monday. The Waltham, MA-based biotech laid off about 13% of its staff in October 2023. It’s one of the most well-funded private biopharma startups after having raised about $1.25 billion, including its $401 million Series D last year. Partnerships with companies like Novo Nordisk, Moderna, Kyverna and Affini-T are unaffected, the spokesperson confirmed. — Kyle LaHucik Monopar Therapeutics prices $19.2M offering: The Chicago-area biotech sold shares to Janus Henderson, RA Capital and others. A few days ago, Monopar licensed a Wilson disease drug candidate from AstraZeneca’s Alexion. — Kyle LaHucik Idorsia ends work with Neurocrine Biosciences: The companies were developing Idorsia’s T-type calcium channel blocker for patients with a rare form of pediatric epilepsy. The treatment failed a Phase 2 study in 2022, and further analysis led to the decision to stop work. — Jaimy Lee

VaccinePhase 2Drug Approval

07 Oct 2024

·www.prnewswire.com

ScaleReady announces a G-Rex® Grant has been awarded to renowned cancer immunologist Dr. Christopher A. Klebanoff

ST. PAUL, Minn., Oct. 7, 2024 /PRNewswire/ -- ScaleReady™, in collaboration with Wilson Wolf Manufacturing, Bio-Techne Corporation (NASDAQ: TECH) and CellReady™, today announced that Christopher A. Klebanoff, MD, a physician-scientist at Memorial Sloan Kettering Cancer Center (MSK) has been awarded a G-Rex® Grant. Dr. Klebanoff's $300,000 G-Rex® Grant will provide enabling reagents to facilitate process development, IND-enabling studies, and support clinical manufacturing of a novel CD8α/β-armored TCR-T cell therapy targeting an HLA-A*11:01-restricted RAS (G12D) public neoantigen in a Phase 1 clinical trial. "Dr. Klebanoff is a leader in the field with over 20 years of experience, and we are fortunate to be able to support his novel approach to cancer treatment. We are confident that this G-Rex Grant will significantly accelerate the path to clinical results and simultaneously set the stage for highly efficient closed system scale out. We are also hopeful that patients will receive significant benefit from treatment with armored TCR-T drug products," said John Wilson, CEO of Wilson Wolf and co-inventor of G-Rex. "Our armored TCR-T therapy is designed to recognize antigen targets that are specific to every cancer cell. We are diligently working to bring this public neoantigen cellular immunotherapy into the clinic to assess its safety and potential efficacy against common solid cancers with significant unmet medical need. The G-Rex Grant will expedite development and accelerate our entrance into human clinical trials," said Dr. Klebanoff, who serves as an Attending Physician, Laboratory Head, Human Oncology & Pathogenesis Program, and Member Investigator, Center for Cell Engineering at MSK. Dr. Klebanoff is also a Scientific Co-Founder of Affini-T Therapeutics, and a member of the Parker Institute for Cancer Immunotherapy (PICI). As part of the G-Rex Grant, Dr. Klebanoff and Dr. Chandran at MSK plan to complete their pre-clinical process development which will include the incorporation of Wilson Wolf's fully closed-system G-Rex bioreactors and the GatheRex for semi-automated volume reduction, cell concentration, and harvest. Additionally, Dr. Klebanoff expects to perform a technology transfer to MSK's cGMP facility, which will perform engineering runs in support of an Investigational New Drug (IND) application to be filed with the FDA with the hope of initiating a Phase 1 clinical trial. "Our goal is to thoughtfully develop and expeditiously transfer a robust manufacturing framework that will reliably and reproducibly enable the generation of infusion products for cancer patients by our cGMP facility. Our hope is that this public-neoantigen specific TCR-T therapy will benefit a patient population that currently does not have many treatment options available. We also recognize this as an invaluable opportunity to holistically learn from this bench-to-bedside experience as we continue to discover and innovate in the lab.", said Smita S. Chandran, PhD, a Scientific Research Lead in the Klebanoff Lab at MSK, who led the discovery and development of the HLA-A*11/RAS(G12D) TCR-T therapeutic. The G-Rex Grant will also support clinical manufacturing runs of their autologous TCR-T cell therapy. Expedited patient enrollment is expected to occur because of MSK's unique IMPACT Program, that prescreens (via next generation sequencing) all incoming patients for commonly mutated genes. MSK will also get early access to a new line of closed system GMP reagents from Bio-Techne that are tailor-made for use with fully-closed system G-Rex bioreactors. The G-Rex ProPak™ CAR-TCR Cytokine Kits, estimated to be commercially available later this year, are weldable bags of liquid cytokines and are designed to streamline reagent preparation and administration in a GMP manufacturing setting without the use of a biosafety cabinet (BSC). ScaleReady's G-Rex Grant Program is a $20M initiative to advance the state of cell and gene-modified cell therapy (CGT) development and manufacturing by awarding individual Grant Awards worth up to $300,000. G-Rex Grant Recipients also gain access to exclusive support from ScaleReady's growing consortium of G-Rex Grant Partners who bring best-in-class tools and technologies as well as unparalleled knowledge and expertise in the areas of cGMP manufacturing, quality and regulatory affairs, CGT business operations, and more. About ScaleReady ScaleReady provides the field of cell and gene-modified cell therapy (CGT) with a G-Rex centric manufacturing platform that enables the world's most practical, flexible, scalable, and affordable CGT drug product development and manufacturing. The G-Rex manufacturing platform is currently used by a rapidly growing list of over 800 organizations and is producing drug products for approximately 50% of CGT clinical trials as well as 5 commercially approved CGT drugs. CGT entities relying on the breadth and scope of ScaleReady's expertise can expect to save years of time and millions of dollars on the path to CGT commercialization. For more information about the ScaleReady G-Rex® Grant Program, please contact [email protected]. About Wilson Wolf Manufacturing Wilson Wolf () is dedicated to simplifying cell and gene-modified cell (CGT) therapy research, process development, and manufacturing. This is being accomplished through its scalable G-Rex® technology, which is used throughout the world in CGT applications ranging from basic research to commercial drug production. Wilson Wolf's mission is to create hope for cancer patients, one G-Rex device at a time. About Bio-Techne Corporation Bio-Techne Corporation (NASDAQ: TECH) is a global life sciences company providing innovative tools and bioactive reagents for the research and clinical diagnostic communities. Bio-Techne, in partnership with Wilson Wolf, is creating products such as media and cytokines that are specifically tailored to G-Rex Bioreactors, including right-sized reagent quantities in containers that are tailored to high throughput closed-system manufacturing. For more information on Bio-Techne and its brands, please visit or follow the Company on social media at: Facebook, LinkedIn, Twitter or YouTube. Contact: David Clair, Vice President, Investor Relations & Corporate Development [email protected] 612-656-4416 About CellReady LLC CellReady is the world's first and only G-Rex centric contract development and manufacturing organization (CDMO) specializing in G-Rex based cell and gene-modified cell therapy development and manufacturing. The company offers a wide range of services to support the development and commercialization of these therapies. CellReady's mission is to create hope for cancer patients, one G-Rex process at a time. SOURCE Bio-Techne Corporation WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

ImmunotherapyCell TherapyPhase 1

14 Aug 2024

·www.globenewswire.com

Metagenomi Reports Business Updates and Second Quarter 2024 Financial Results

Declared development candidate MGX-001 for the treatment of hemophilia A; plans to present 12-month NHP durability study data in September 2024 All Wave 1 Ionis collaboration programs advancing in lead optimization, unlocking potential for multiple development candidate nominations in 2025 Achieved milestone from partner Affini-T related to Metagenomi licensed technology Well capitalized with $299.9M in cash, cash equivalents and available-for-sale marketable securities at the end of Q2 2024; cash runway anticipated to support operating plans into 2027 EMERYVILLE, Calif., Aug. 14, 2024 (GLOBE NEWSWIRE) -- Metagenomi, Inc. (Nasdaq: MGX) (Metagenomi), a precision genetic medicines company committed to developing curative therapeutics for patients using its proprietary, comprehensive metagenomics-derived gene editing toolbox, today provided a business update and reported second quarter 2024 financial results. “We are progressing our pipeline while remaining laser focused on our vision to create curative genetic medicines by harnessing the power of our AI-driven metagenomics platform,” said Brian C. Thomas, PhD, CEO and Founder. “We have significantly advanced our wholly-owned program in hemophilia A, including recent positive regulatory interactions with Food and Drug Administration (FDA) and the declaration of MGX-001 as our lead development candidate. Our team has worked diligently to allow us to accelerate the data release from our 12-month nonhuman primate (NHP) durability study from the end of 2024 into September 2024. This study is an important step towards advancing MGX-001 as a potentially life-long curative treatment for both adults and children with hemophilia A, and demonstrating our ability to potentially achieve large targeted gene integration in patients using our metagenomics toolbox.” Dr. Thomas continued, “We are also progressing our partnered programs and advancing our metagenomics-based toolbox. The foundation of our Ionis collaboration was strengthened by the addition of two high-value targets associated with cardiometabolic disease, and all collaboration programs have progressed into the lead optimization phase of development. We achieved a development milestone with Affini-T, where our team provided support for Affini-T’s regulatory filing related to Metagenomi licensed technology and current good manufacturing practices (cGMP) material supply. We continue to advance a broad range of technologies to support our goal of being able to address any disease by enabling any therapeutic genome edit in the human genome. Going forward, we remain well capitalized to execute our strategic priorities, with a cash runway into 2027.” Second Quarter 2024 Business Updates and Key 2024-2025 Anticipated Milestones Therapeutic Pipeline Updates Metagenomi continues to advance its wholly-owned lead program in hemophilia A, designed as a one-time curative treatment for both adults and children. The company engaged with FDA in the second quarter to discuss key aspects of the planned Investigational New Drug (IND) submission.The company declared a lead development candidate (DC), MGX-001, meeting previously announced guidance for the timing of a DC nomination.The company plans to accelerate the release of data from the 12-month NHP durability study for the lead hemophilia A program from late 2024 into September 2024. The 12-month NHP durability study was designed to show therapeutically relevant, durable expression of Factor VIII in NHPs, addressing a key concern which has impacted other genetic medicines programs in hemophilia A that have experienced loss of Factor VIII expression over time.The company plans to initiate cGMP manufacturing and related IND enabling activities in 2024 and continue IND enabling activities in 2025.As previously disclosed, the company remains on track to file an IND in hemophilia A in 2026. Building on the recent success of the hemophilia A program, Metagenomi plans to advance additional wholly-owned therapeutic candidates targeting secreted protein disorders, leveraging the MGX-001 editing system with the goal of achieving targeted and durable gene expression. This editing system consists of two components: a highly efficient and specific nuclease creates a precise cut at the albumin safe harbor gene locus after delivery by LNP; and an AAV vector delivered FVIII DNA template is inserted into the nuclease cut site by a naturally occurring DNA repair process. Following selection of the remaining Wave 1 targets in the Ionis collaboration in the first quarter of 2024, all four therapeutic programs successfully advanced into the lead optimization phase in the second quarter of 2024. All four therapeutic targets in Wave 1 collaboration focus on cardiometabolic diseases; the company announced Refractory Hypertension as the initial disease target leveraging the angiotensinogen pathway.The company aims to demonstrate in vivo proof-of-concept in 2024, and advance NHP studies supporting DC nominations thereafter.The company plans to nominate one to two DCs for cardiometabolic programs in 2025. The company achieved a milestone in connection with its Affini-T collaboration, establishing cGMP gene editing reagents for cell therapy and filing related Drug Master Files with FDA to support an IND for Affini-T’s T-cell receptor-based therapy. Metagenomi received 933,650 shares of Affini-T common stock upon achievement of this regulatory milestone. Technology Platform Updates Metagenomi achieved in vitro proof-of-concept for an undisclosed neuromuscular target with its ultra small editing system, a key milestone, opening up therapeutic potential for extrahepatic disease targets.Metagenomi achieved in vitro proof-of-concept using its RNA-mediated integration system (RIGS) for undisclosed liver targets.Metagenomi achieved multiplex base editing proof-of-concept and plans to present this data at a scientific conference in the second half of 2024.Metagenomi achieved in vitro proof-of-concept in human cells for large gene integration using its potentially industry leading, compact CRISPR-associated transposase (CAST) technology, with a publication planned.As previously disclosed, the company remains on track to demonstrate in vivo proof-of-concept for a large gene integration in 2026. Other Business Updates The company continues to exercise fiscal responsibility in prioritizing certain internal development programs while looking for collaboration partners to leverage other development opportunities. Metagenomi is looking for a partner or licensee for further development of its program for primary hyperoxaluria type 1 (PH1). Previously disclosed data demonstrated preclinical proof-of-concept in an accepted disease model of PH1.The company is continuing to focus its internal efforts on in vivo gene editing therapeutic approaches and pursue technology out-licensing for ex vivo cell therapy, where next generation gene editing systems are an important enabler of novel therapies.Going forward, the company is not pursuing amyotrophic lateral sclerosis (ALS) based upon recent peer company clinical data regarding the lack of efficacy of Ataxin-2 as a therapeutic target for ALS.The company is revising its pipeline design to consolidate earlier stage programs and make it easier to identify our near term pipeline priorities focused on in vivo gene editing and liver indications. As a result of the acceleration of all four Wave 1 Ionis targets into lead optimization, as well as the company’s decision to deprioritize PH1, Metagenomi is revising to provide pipeline guidance only through year-end 2025, including the next one to two DC nominations in 2025. The company is maintaining its guidance for the planned IND filing for hemophilia A in 2026. The updated milestones are available in the corporate presentation on the company website. Upcoming EventsMetagenomi plans to participate in the following events during the third quarter of 2024: Wells Fargo Healthcare Conference, September 4-6, 2024, Everett, MassachusettsH.C. Wainwright 26th Annual Global Investment Conference, September 9-11, 2024, New York CityChardan 8th Annual Genetics Medicines Conference, September 30-October 1, 2024, New York City Second Quarter 2024 Financial Results Cash Position: Cash, cash equivalents, and available-for-sale marketable securities were $299.9 million as of June 30, 2024.R&D Expenses: Research and development (R&D) expenses were $28.3 million for the three months ended June 30, 2024, compared to $22.7 million for the three months ended June 30, 2023.G&A Expenses: General and administrative (G&A) expenses were $8.6 million for the three months ended June 30, 2024, compared to $6.6 million for the three months ended June 30, 2023. About Metagenomi Metagenomi is a precision genetic medicines company committed to developing curative therapeutics for patients using its proprietary, comprehensive metagenomics-derived toolbox. Metagenomi is harnessing the power of metagenomics, the study of genetic material recovered from the natural environment, to unlock four billion years of microbial evolution to discover and develop a suite of novel editing tools capable of correcting any type of genetic mutation found anywhere in the genome. Its comprehensive genome editing toolbox includes programmable nucleases, base editors, and RNA and DNA-mediated integration systems (including prime editing systems and clustered regularly interspaced short palindromic repeat associated transposases). Metagenomi believes its diverse and modular toolbox positions the company to access the entire genome and select the optimal tool to unlock the full potential of genome editing for patients. For more information, please visit https://metagenomi.co. Cautionary Note Regarding Forward‐Looking Statements This press release contains “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. Such statements, which are often indicated by terms such as “anticipate,” “believe,” “could,” “estimate,” “expect,” “goal,” “intend,” “look forward to,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” and similar expressions, include, but are not limited to, any statements relating to our growth strategy and product development programs, including the timing of and our ability to conduct IND-enabling studies, make regulatory filings such as INDs, statements concerning the potential of therapies and product candidates, statements concerning the timing of data presentations and publications, and any other statements that are not historical facts. Forward looking statements are based on management’s current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition, and stock value. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under, and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; risks relating to the timing of starting and completing clinical trials; uncertainties relating to preclinical and clinical testing; our dependence on third party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in “Risk Factors,” in our most recent Form 10-K and our most recent 10-Qs on file with the Securities and Exchange Commission. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law, and we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995. Investor Contact:Simon Harnest - CIO, SVP Investor Relationssimon@metagenomi.co Media Contact:Ashlye Hodge - Communications Managerashlye@metagenomi.co Condensed Financial StatementsCondensed Consolidated Balance Sheet Data(Unaudited) June 30, December 31, (in thousands) 2024 2023 Cash, cash equivalents and available-for-sale marketable securities $299,921 $271,182 Total assets $385,905 $364,842 Total liabilities $116,486 $149,668 Redeemable convertible preferred stock $— $350,758 Total stockholders’ equity (deficit) $269,419 $(135,584)Total liabilities, redeemable convertible preferred stock and stockholders’ equity (deficit) $385,905 $364,842 Condensed Consolidated Statements of Operations(Unaudited) Three Months EndedJune 30, Six Months EndedJune 30, (In thousands, except share and per share data) 2024 2023 2024 2023 Collaboration revenue $20,008 $11,337 $31,167 $19,994 Operating expenses: Research and development 28,320 22,681 59,759 42,811 General and administrative 8,551 6,619 17,303 13,084 Total operating expenses 36,871 29,300 77,062 55,895 Loss from operations (16,863) (17,963) (45,895) (35,901)Other income (expense): Interest income 3,976 3,967 7,910 7,970 Change in fair value of long-term investments — 2,870 — 2,870 Other income (expense), net (51) 16 (101) 15 Total other income, net 3,925 6,853 7,809 10,855 Net loss before benefit (provision) for income taxes (12,938) (11,110) (38,086) (25,046)Benefit (provision) for income taxes 2,199 (1,898) 2,199 (4,095)Net loss $(10,739) $(13,008) $(35,887) $(29,141)Net loss per share attributable to common stockholders, basic and diluted $(0.29) $(3.82) $(1.24) $(8.56)Weighted average common shares outstanding, basic and diluted 36,625,291 3,404,585 28,901,399 3,404,585

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Drug(Targets)	Indications	Global Highest Phase

AFNT-211 ( KRAS G12V )	Metastatic Solid Tumor More	Phase 2 Clinical
AFNT-212 ( KRAS G12C x KRAS G12D x PI3Kα x p53 R175H )	Neoplasms More	Preclinical
Oncogenic Driver Program: Mutan P53 R175H(Affini-T Therapeutics, Inc.) ( CD3 x p53 R175H )	Neoplasms More	Preclinical
Oncogenic Driver Program: KRAS G12C (Affini-T Therapeutics, Inc) ( KRAS G12C )	Neoplasms More	Preclinical
AFNT-111 ( KRAS G12V )	Neoplasms More	Preclinical

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