On May 6, 2025 Mission Bio, a pioneer in single-cell multi-omics solutions, reported it has partnered with Integrated DNA Technologies (IDT), a global leader in genomics solutions, to introduce an advanced workflow for precise on- and off-target confirmation in gene editing applications (Press release, Mission Bio, MAY 6, 2025, View Source [SID1234652616]). This collaboration integrates IDT’s award-winning rhAmpSeq technology with Mission Bio’s Tapestri Platform to provide a comprehensive, high-resolution approach for assessing genome editing outcomes at the single-cell level.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Genome editing is crucial for the development of the next wave of cell and gene therapies, enabling precise and multiple genetic modifications. However, ensuring the specificity and efficiency of these edits remains a critical challenge, particularly in cell and gene therapy (CGT) applications. Traditional bulk sequencing methods provide valuable insights into genome editing efficiency, but lack the resolution to capture single-cell heterogeneity, zygosity, and co-occurrence of edits within individual cells.

To address these challenges, Mission Bio and IDT have developed an integrated workflow combining IDT’s rhAmpSeq amplicon-based targeted resequencing chemistry with Mission Bio’s droplet-based single-cell DNA sequencing technology. This novel solution builds on published approaches that researchers have previously used to leverage the platforms. The new, integrated approach enhances the efficiency for obtaining both on-target efficacy assessment and off-target detection in a single-cell context, offering researchers an unprecedented level of accuracy and confidence in genome editing analysis.

In a proof-of-concept study, to be presented at the upcoming American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting, the integrated workflow was interrogated using well-characterized cell lines and selected for their extensive orthogonal bulk sequencing data. By targeting loci with known indel variations ranging from single-base pair changes to large insertions and deletions (>2,000 bp), the study demonstrated the robustness of rhAmpSeq chemistry in single-cell applications. The resulting targeted panel proved compatible with both bulk and single-cell assays, ensuring seamless adoption across various research needs.

"Our single-cell multi-omics has been used in combination with bulk sequencing to add specificity that otherwise would not be possible," said Brian Kim, CEO of Mission Bio. "By integrating our capabilities with IDT’s trusted approach to bulk sequencing, we’re adding together multiple layers of genetic insights in a single drop."

The Tapestri Platform’s automated single-cell genome editing analytics pipeline provided a quantitative assessment of editing efficiency, off-target editing levels and zygosity, offering a critical layer of data resolution beyond bulk sequencing. By integrating rhAmpSeq’s advanced primer design with Mission Bio’s single-cell DNA resequencing technology, the new workflow establishes a cost-effective, scalable, and highly customizable solution for genome editing validation in CGT applications.

"Safety is key to unlocking the true promise of CRISPR, and this collaboration with Mission Bio reflects another step forward to advancing the delivery of safer and more efficient genome editing platforms," said Sandy Ottensmann, VP/GM, Gene Writing and Editing at IDT. "By pairing the precision of rhAmpSeq with the single-cell resolution of Tapestri, we aim to support cell and gene therapy developers in driving the future of safe, life-changing therapies to benefit more patients."

Researchers and industry professionals can learn more about this cutting-edge approach at the upcoming American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting this month. Mission Bio and IDT will be presenting a poster detailing the study’s findings, demonstrating the power of integrated single-cell sequencing for genome editing validation.

The poster "Comprehensive On- and Off-target Validation Using Integrated rhAmpSeq and Targeted DNA Resequencing Single-Cell Technology for Gene Editing Applications" will be presented on Thursday, May 15 at 5:30 pm CST.

An oral presentation delivered by Dr. Ayal Hendel of Bar-Ilan University, "Single-Cell Profiling of Genome-Editing Alterations and Functional Outcomes in CRISPR-Engineered Cells," will take place on Saturday, May 17 at 9:00 am CST.

Visit Mission Bio’s Booth #1749 at ASGCT (Free ASGCT Whitepaper) to see how this technology can accelerate your research.

For more information, visit missionbio.com or contact [email protected].

On May 6, 2025 Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for autoimmune diseases and cancer, reported financial results and operational highlights for the first quarter ended March 31, 2025 (Press release, Adicet Bio, MAY 6, 2025, View Source [SID1234652615]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are approaching an exciting inflection point for our pipeline, with significant data milestones on the horizon," said Chen Schor, President and Chief Executive Officer of Adicet Bio. "In the second half of 2025, we expect to report preliminary Phase 1 data from our two lead programs – ADI-001 in autoimmune diseases and ADI-270 in ccRCC, with more than 6 patients with at least 3-month follow up in both programs. As we progress toward these readouts, we also look to harness the full potential of our allogeneic gamma delta 1 CAR T cell therapy platform, which we believe has key advantages over other cell types. We have identified two promising highly differentiated programs, one targeting PSMA and one follow-on program targeting autoimmune diseases with potential to become best-in-class therapies for patients fighting autoimmune diseases and cancer."

First Quarter 2025 and Recent Operational Highlights:

Autoimmune diseases

Enrollment open for LN and SLE patients in Phase 1 clinical trial of ADI-001 in autoimmune diseases. In April 2025, Adicet expanded enrollment in its Phase 1 trial to include patients with SLE, in addition to ongoing enrollment in LN. The Company expects to initiate enrollment for patients with systemic sclerosis (SSc), idiopathic inflammatory myopathy (IIM), stiff person syndrome (SPS) and anti-neutrophil cytoplasmic autoantibody associated vasculitis (AAV) in the Phase 1 trial in 3Q/2025. Preliminary clinical data from the trial is expected in 2H/2025, subject to study site initiation and patient enrollment.
ADI-001 granted two new Fast Track Designations. In February 2025, the Food and Drug Administration (FDA) granted Fast Track Designation to ADI-001 for the treatment of refractory SLE with extrarenal involvement and for SSc.
Hematologic malignancies and solid tumor indications

Patient enrollment ongoing in Phase 1 trial of ADI-270 in metastatic/advanced ccRCC. Patient enrollment is underway in the Phase 1 clinical trial evaluating ADI-270 in adults with relapsed or refractory metastatic/advanced ccRCC. Adicet expects to share preliminary clinical data from the trial in 2H/2025.
Oral presentation of ADI-270 data at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 28th Annual Meeting. Adicet will present an oral abstract highlighting strong preclinical data demonstrating ADI-270’s anti-tumor activity in hematologic and solid tumor models at the ASGCT (Free ASGCT Whitepaper) Annual Meeting taking place May 13-17, 2025 in New Orleans, LA.
Presented ADI-270 preclinical data at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 2025 Spring Scientific Meeting. In March 2025, Adicet presented posters covering preclinical data of ADI-270 at the SITC (Free SITC Whitepaper) 2025 Spring Scientific Meeting.
Corporate Update

Appointed Michael Grissinger to Board of Directors. In April 2025, Adicet appointed Michael Grissinger to its Board of Directors. Mr. Grissinger brings over four decades of leadership experience in biopharmaceutical business development, strategy, and M&A to Adicet. Mr. Grissinger has an extensive track record of driving commercial success for global pharmaceutical companies, with a strong focus on immunology. He also serves on the board of directors at Aprea Therapeutics (Nasdaq: APRE) and three privately-held biotechnology companies, Envisagenics, Inc., AnaCardio AB, and NephroDI Therapeutics, Inc.
Financial Results for First Quarter 2025:

Research and Development (R&D) Expenses: R&D expenses were $22.8 million for the three months ended March 31, 2025, compared to $23.9 million during the same period in 2024. The decrease in R&D expenses was primarily due to a net $1.4 million decrease in expenses related to contract development manufacturing organizations and other externally conducted research and development.
General and Administrative (G&A) Expenses: G&A expenses were $7.1 and 7.0 million for the three months ended March 31, 2025 and 2024, respectively.
Net Loss: Net loss for the three months ended March 31, 2025 was $28.2 million, or a net loss of $0.31 per basic and diluted share, including non-cash stock-based compensation expense of $3.1 million, as compared to a net loss of $28.0 million, or a net loss of $0.35 per basic and diluted share, including non-cash stock-based compensation expense of $5.7 million during the same period in 2024.
Cash Position: Cash and cash equivalents were $150.4 million as of March 31, 2025, compared to $176.3 million as of December 31, 2024. The Company expects that current cash, cash equivalents and short-term investments as of March 31, 2025, will be sufficient to fund its operating expenses into the second half of 2026.

On May 6, 2025 Agendia, Inc., reported that new data from its ongoing FLEX Study will be presented at the upcoming ESMO (Free ESMO Whitepaper) Breast Cancer 2025 congress taking place May 14-17 in both Munich, Germany and virtually (Press release, Agendia, MAY 6, 2025, View Source [SID1234652614]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The FLEX Study (NCT03053193) is a prospective real-world evidence, observational breast cancer study designed to correlate whole transcriptome gene expression in early-stage breast cancer with clinical outcomes, and to evaluate how genomic insights can inform treatment decisions in early-stage breast cancer. In this analysis, researchers examined the impact of BluePrint, Agendia’s 80-gene molecular subtyping assay, on pathological complete response (pCR) rates and chemotherapy (CT) decision-making in patients with hormone receptor-positive (HR+), HER2-negative tumors classified as High Risk by MammaPrint. BluePrint further stratified these tumors into Basal or Luminal B subtypes, offering a more nuanced view of tumor biology and potential treatment response.

The poster presentation, titled "The Impact of the 80-gene signature on pCR and chemotherapy treatment decisions in Early-Stage Breast Cancer: A FLEX Analysis [70P]," (A.M. Brufsky, et al.), highlights findings from the analysis of two cohorts from the FLEX Study: one consisting of patients who underwent genomic analysis on pre-operative core needle biopsy, and received neoadjuvant chemotherapy with available pCR data and another cohort with documented physician chemotherapy recommendations. The analysis found that patients with MammaPrint High Risk, HR+ HER2- Basal-type tumors were more likely to achieve a pCR following neoadjuvant chemotherapy compared to those with Luminal B tumors. The analysis also showed that these Basal-type tumors were more frequently recommended for chemotherapy overall, more often treated with neoadjuvant chemotherapy specifically, and received more intensive regimens compared to Luminal B tumors. These results suggest that BluePrint provides actionable molecular insights that physicians are using to inform real-world treatment decisions, even among patients already eligible for chemotherapy based on MammaPrint High Risk status.

"These results reinforce the value of BluePrint in helping physicians personalize treatment plans for early-stage breast cancer," said Adam Brufsky, MD, PhD, Professor and Associate Chief of Hematology and Oncology at UPMC Hillman Cancer Center. "By identifying Basal-type tumors that are more likely to respond to chemotherapy, BluePrint can guide decisions about treatment intensity and timing that align with each patient’s individual tumor biology."

Poster Presentation Details:

Title: The Impact of the 80-gene signature on pCR and chemotherapy treatment decisions in Early-Stage Breast Cancer: A FLEX Analysis [70P]
Date and Time: Thursday, May 15, 12:00 PM CEST
Location: ICM – International Congress Center of the Munich Messe, Hall B0

On May 6, 2025 Sonoma Biotherapeutics, Inc., a clinical-stage biotechnology company developing engineered regulatory T cell (Treg) therapies for autoimmune and inflammatory diseases, reported that it will be sharing oral and poster presentations at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 28th Annual Meeting, taking place in New Orleans May 13-17, 2025 (Press release, Sonoma Biotherapeutics, MAY 6, 2025, View Source [SID1234652613]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are excited to share data demonstrating the promise of Treg-based cell therapies at this year’s ASGCT (Free ASGCT Whitepaper) Annual Meeting," said Jeff Bluestone, Ph.D., Chief Executive Officer and President of Sonoma Biotherapeutics. "Sharing the latest insights on the potential impact this research can have in autoimmune diseases is a privilege for all of us at Sonoma Biotherapeutics."

Details of the presentations are as follows:

Scientific Symposium Oral Presentation Details:

Title: "Engineered Treg therapies to treat Autoimmune Diseases"
Presenting Author: Jeff Bluestone, PhD, Sonoma Biotherapeutics
Session: Expanding Modalities Beyond Their Initial Use: Discussions on Translating Immune Cell Therapies into Non-Oncology Indications
Date/Time: Saturday May 17, 8:00 – 9:45 a.m. PDT
Location: Room 293-296
Oral Presentation Details:

Title: "Transcriptomic and Epigenomic Analysis of CAR Treg Stability"
Presenting Author: Zhang "Frank" Cheng, PhD, Sonoma Biotherapeutics
Session: Cell Therapy Product Engineering I
Date/Time: Wednesday May 14, 8:30 – 9:45 a.m. PDT
Location: Room 293-296
Poster Presentation Details:

Title: "Treg Specific Synthetic Promoters (TRSP) for Antigen Specific Treg Therapy"
Presenting Author: Jia Wei, PhD, Sonoma Biotherapeutics
Poster number: 1764
Session: Poster Reception
Date/ Time: Thursday, May 15, 5:30 – 7:00 p.m. PDT
Location: Poster Hall 2

On May 6, 2025 Tempus AI, Inc. (NASDAQ: TEM), a technology company leading the adoption of AI to advance precision medicine and patient care, reported financial results for the quarter ended March 31, 2025 (Press release, Tempus, MAY 6, 2025, View Source [SID1234652612]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Revenue increased 75.4% year-over-year to $255.7 million in the first quarter of 2025
Quarterly gross profit increased 99.8% year-over-year, reaching $155.2 million with continued gross margin improvement in both Genomics and Data and services
Announced multi-year, strategic collaborations with AstraZeneca and Pathos to work together to build the largest multimodal foundation model in oncology, resulting in additional $200.0 million in data licensing and model development fees over the next 3 years
Increasing full year 2025 revenue guidance to $1.25 billion, representing approximately 80% growth year-over-year. Expect positive Adjusted EBITDA of $5 million for full year 2025, increasing approximately $110 million over 2024
"The business is performing well with revenues growing, margins improving, and our costs remaining in check, allowing us to demonstrate significant year-over-year operating leverage," said Eric Lefkofsky, Founder and CEO of Tempus. "Our strategic investments in AI have us uniquely positioned to advance what is possible in diagnostics and drug development, as evidenced by our announcement to build the largest foundation model in oncology with AstraZeneca and Pathos. We believe this is just the beginning as more and more healthcare providers and life science companies embrace AI."

First Quarter Summary Results

Quarterly revenue increased 75.4% year-over-year to $255.7 million in the first quarter of 2025.
Genomics contributed $193.8 million in revenue in the first quarter of 2025, growing 88.9% compared to the first quarter of 2024.
Oncology testing (legacy Tempus clinical) revenue delivered $119.0 million, up 31.0% year-over-year in the first quarter of 2025, with approximately 20% volume growth.
Hereditary testing (legacy Ambry Genetics) contributed $63.5 million in revenue in the first quarter, with approximately 23% unit growth.
Revenue from Data and services totaled $61.9 million in the first quarter of 2025, delivering 43.2% growth versus the first quarter of 2024, led by Insights (data licensing), which grew 58.0% year-over-year.
Generated $155.2 million in quarterly gross profit, reflecting a 99.8% improvement year-over-year.
Reported a net loss of ($68.0 million) in the first quarter of 2025, including $28.2 million in stock compensation expense and related employer payroll taxes and fair value losses of $31.8 million related to our marketable equity securities, compared to a net loss of ($64.7 million) in the first quarter of 2024.
Adjusted EBITDA of ($16.2 million) in the first quarter of 2025 compared to ($43.9 million) in the first quarter of 2024, an improvement of $27.8 million year-over-year.
First Quarter and Recent Operational Highlights

Announced multi-year, strategic collaborations with AstraZeneca and Pathos to work together to build a multimodal foundation model in oncology, which include $200.0 million in additional data licensing and model development fees to Tempus over the next 3 years.
Completed the acquisition of Ambry Genetics on February 3, 2025.
Disclosed a collaboration with Illumina combining its AI technologies with Tempus’ comprehensive multimodal data platform to train genomic algorithms and accelerate clinical adoption of molecular testing.
Reported the acquisition of Deep 6 AI, broadening Tempus’s reach and enhancing applications like Next and TIME.
Launched olivia, an AI-enabled personal health concierge app for patients nationally.
Nationally launched xT CDx with ADLT pricing established at $4,500 per test.
First Quarter Financial Results

Three Months Ended March 31,

2025

2024

Change

(in thousands, except percentages and per share amounts)

(unaudited)

Revenue

$

255,737

$

145,820

75.4

%

Gross profit

$

155,203

$

77,697

99.8

%

Loss from operations

$

(68,689

)

$

(53,274

)

NM(1)

Non-GAAP loss from operations

$

(25,777

)

$

(53,274

)

51.6

%

Net loss

$

(68,037

)

$

(64,743

)

NM(1)

Adjusted EBITDA

$

(16,174

)

$

(43,926

)

63.2

%

Net loss per share attributable to common shareholders, basic and diluted

$

(0.40

)

$

(1.47

)

72.8

%

Non-GAAP net loss per share

$

(0.24

)

$

(1.03

)

76.3

%

____________
(1)

Not meaningful due to the impact of including stock compensation expense and related employer payroll taxes

Financial Outlook and Guidance

Tempus now expects full year 2025 revenue of approximately $1.25 billion for the consolidated Tempus and Ambry Genetics business, which represents approximately 80% annual growth, and Adjusted EBITDA of $5 million for full year 2025, an improvement of approximately $110 million over 2024.

For additional information on the quarter, including a letter from our CEO and CFO, please visit our investors relations site at investors.tempus.com.

Webcast and Conference Call Information

A conference call and webcast will begin today, May 6, 2025 after market close at 4:30 p.m. Eastern Time. Interested parties may access details at:

Conference ID: 4680302
Domestic Dial-in Number: (888) 672-2415
International Dial-in Number: (646) 307-1952
Live webcast: View Source

The webcast may be accessed on the company’s investor relations website at investors.tempus.com. For those unable to listen to the live webcast, a recording will be made available on the company’s website after the event and will be accessible for one year. Visit the investor relations website to find the company’s latest deck, and commentary on the quarter by Eric Lefkofsky, Founder and CEO and Jim Rogers, CFO, which will be discussed on the conference call and webcast.