On May 7, 2024 Caring Cross, a 501(c)(3) non-profit dedicated to accelerating the development of advanced medicines and enabling access to cures for all patients, everywhere, and ImmunoAdoptive Cell Therapy (ImmunoACT), a Mumbai, India-based pioneering research and cell and gene therapy development company, reported an agreement to develop and commercialize a multi-targeted chimeric antigen receptor T-cell immunotherapy for leukemia and lymphoma (Press release, Caring Cross, MAY 7, 2024, View Source [SID1234642765]). The TriCAR-T immunotherapy is designed to address relapse that can occur when patients with Leukemia or Lymphoma are treated with a single targeting anti-CD19 CAR T-cell product. Under the terms of the agreement, Caring Cross will provide a TriCAR-T cell immunotherapy for clinical development, manufacturing, and commercialization at ImmunoACT’s facilities in India.

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!

ImmunoACT is developing an array of CAR-T therapeutic assets at various stages of clinical maturity to address oncological indications and autoimmune disorders. Its lead candidate, NexCAR19 (Actalycabtagene autoleucel), was recently approved as India’s first CAR-T cell therapy for the treatment of r/r B-cell lymphomas and leukemia by the Central Drugs Standard Control Organization (CDSCO). It is a first-of-its-kind product that has given patients in India access to this lifesaving, leading-edge therapy at an affordable price.

"We are pleased to form this commercial partnership with ImmunoACT, which is committed to providing affordable and accessible CAR-T cell therapies for serious diseases like leukemia and lymphoma," said Caring Cross Co-Founder and Executive Director Boro Dropulić, Ph.D. "Our global partnership model aims to improve access for CAR-T cell therapies by developing CAR-T cell manufacturing technologies and providing therapeutic candidates that are affordable in countries like India. We are excited to work with ImmunoACT to commercialize a dedicated proprietary TriCAR-T cell therapy in India, which we anticipate will decrease the rate of relapse seen with current single targeted anti-CD19 CAR-T cell therapies. We look forward to working closely with ImmunoACT to expand access to a commercial TriCAR-T cell therapy in India as part of our mission to further universal access and affordability of CAR-T therapies worldwide."

"We are delighted to work with Caring Cross to bring next generation CAR-T therapies to India, which will enable cost-effective local production and improved access to these advanced medicines " stated ImmunoACT’s Founder and Professor at IIT Bombay, Rahul Puwar, Ph.D. "ImmunoACT’s technical and commercial operations capabilities are well proven and with its network of partner hospitals across the country, this partnership has great potential to benefit thousands of patients in coming years."

On May 7, 2024 Bristol Myers Squibb reported that the company will participate in two upcoming investor conferences in May 2024 (Press release, Bristol-Myers Squibb, MAY 7, 2024, View Source [SID1234642764]).

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!

Ben Hickey, President, RayzeBio, Head of Mirati, will take part in a fireside chat at the Guggenheim Healthcare Talks Radiopharmaceuticals Day on Monday, May 13, 2024. He will answer questions about the company beginning at 11:00 a.m. ET.

David Elkins, Executive Vice President, Chief Financial Officer, will participate in a fireside chat at the 2024 Bank of America Global Healthcare Conference on Wednesday, May 15, 2024. He will answer questions about the company beginning at 11:20 a.m. PT/2:20 p.m. ET.

Investors and the general public are invited to listen to both sessions at their respective times by visiting View Source An archived edition of each session will be available following its conclusion.

On May 7, 2024 BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) reported that the company will present at the following conferences (Press release, BioCryst Pharmaceuticals, MAY 7, 2024, View Source [SID1234642763]):

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!

Citizens JMP Life Sciences Conference in New York on Monday, May 13, 2024, at 12:30 p.m. ET.
2024 RBC Capital Markets Global Healthcare Conference in New York on Tuesday, May 14, 2024, at 3:05 p.m. ET.
Bank of America Health Care Conference 2024 in Las Vegas on Wednesday, May 15, 2024, at 5:20 p.m. ET.
H.C. Wainwright 2nd Annual BioConnect Investor Conference in New York on Monday, May 20, 2024, at 10:30 a.m. ET.
Jefferies Global Healthcare Conference in New York on Thursday, June 6, 2024, at 9:30 a.m. ET.
Links to the live audio webcasts and replays of the presentations may be accessed in the Investors & Media section of BioCryst’s website at http://www.biocryst.com.

On May 7, 2024 Bio-Techne Corporation reported it will showcase its portfolio of cell and gene therapy workflow solutions at the upcoming annual meeting of the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper), taking place May 7-11, 2024, in Baltimore, Maryland (Press release, Bio-Techne, MAY 7, 2024, View Source [SID1234642762]).

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!

Bio-Techne’s booth (#1627) will feature our market-leading portfolio of high-quality life science research use only (RUO) and Good Manufacturing Practice (GMP) reagents, immunoassays, automated proteomic analytical instruments, and multi-omics solutions for accelerating cell and gene therapy development and manufacturing. We will also feature our "Immune Cell Therapy Workflow" multimedia experience, which enables attendees to explore how Bio-Techne’s solutions streamline and accelerate the immune cell processing workflow.

Bio-Techne will host three speaker sessions during the conference, featuring both internal experts and customer case studies. The topics covered will include characterizing biodistribution and safety of cell and gene therapies, protein analytics strategies for gene therapy, and advancements in closed systems and non-viral cell engineering for immune cell therapy manufacturing.

Additionally, several Bio-Techne scientists will present posters highlighting our in-house studies in the areas of immune cell culture, automated spatial multi-omics, and protein or viral vector characterization. These include investigations into the efficient activation and expansion of human T cells for CAR-T therapy, RNA/protein ISH imaging of gene therapy biodistribution and persistence, and the multiparameter characterization of adeno-associated virus capsids.

"We are proud to support the advancement of cell and gene therapy research, development and manufacturing with our robust portfolio of innovative tools, reagents and workflow solutions," said Kim Kelderman, Bio-Techne’s President and Chief Executive Officer. "Bio-Techne’s portfolio plays a critical role in the development of next-generation cell and gene therapies. Our broad presence at ASGCT (Free ASGCT Whitepaper) will showcase the many roles Bio-Techne plays in catalyzing advances in science and medicine, and ultimately enabling patients to live healthier and longer lives."

Bio-Techne Presentations:
Novel RNAscope Multi-omics Spatial Approach to Characterize Biodistribution and Safety of Cell and Gene Therapies

Wednesday, May 8th from 4:45pm – 5:15pm in Room 337-338
Presented by Sharel Figueredo, Ph.D.
Protein Analytics Strategies for Gene Therapy – from Viral Vector Characterization to Clinical Trials

Thursday, May 9th from 8:30am – 9:30am in Room 324-326
Presented by Hiren Patel, Ph.D. (REGENEXBIO Inc.)
Moderator Chris Heger, Ph.D.
Advancements in GMP and Closed System Reagents for Immune Cell Therapy Manufacturing

Wednesday, May 8th from 3:45pm -4:00pm in the Exhibit Hall
Presented by Miles Smith, Ph.D.
Bio-Techne Poster Presentations:
Efficient T-cell Activation and Expansion for CAR-T Therapy is Dependent on a Complex Interplay of the Starting Donor Population, Activation Antibodies and Expansion Platform

Wednesday, May 8th at 12:00 pm
Presenter: Hatt Heidtman, Ph.D.
Abstract 868
Next Generation Protein Characterization of CD19-CAR Signaling Cascades

Thursday, May 9th at 12:00 pm
Presenter: Charles Haitjema, Ph.D.
Abstract 1329
A Versatile Assay for Same-Section Spatial Visualization of Small RNA, mRNA, Exon Junction, Protein and Protein-Protein Interactions

Thursday, May 9th at 12:00 pm
Presenter: Maithreyan Srinivasan, Ph.D.
Abstract 1397
RNA/Protein Multiomics Imaging Using RNAscope Technologies to Understand Nonclinical Biodistribution (BD) and Persistence of Gene Therapy (GT) Products

Thursday, May 9th at 12:00 pm
Presenter: Maithreyan Srinivasan, Ph.D.
Abstract 1398
Characterization of Adeno-Associated Virus Capsids by a Novel Capillary Isoelectric Focusing Based Fractionation Method and Charge-Detection Mass Spectrometry

Friday, May 10th at 12:00 pm
Presenter: Chris Heger, Ph.D.
Abstract 1463

On May 7, 2024 – Beam Therapeutics Inc.(Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, reported first quarter 2024 financial results and provided updates across the company’s hematology and genetic disease franchises (Press release, Beam Therapeutics, MAY 7, 2024, View Source [SID1234642761]).

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’re pleased to share progress across our high-priority programs that exemplify our commitment to rapid, focused execution and a dedication to developing differentiated, one-time medicines for serious genetic diseases," said John Evans, chief executive officer of Beam. "We have successfully completed dosing and engraftment for three sickle cell disease patients in the sentinel cohort of the BEAM-101 BEACON trial allowing us to now move forward with expansion phase dosing. We look forward to sharing data for multiple patients treated with BEAM-101 later this year. In addition, our team has done an incredible job executing our ex-U.S. clinical strategy for BEAM-302, securing CTA clearance in the UK and rapidly working toward the initiation of our Phase 1/2 trial in patients with alpha-1 antitrypsin deficiency. This study is designed to demonstrate proof-of-concept for correction of the disease-causing mutation that could potentially help patients with both lung and liver disease manifestations. These updates, supported by our robust balance sheet, mark a significant stride toward our goal of establishing base editing as a potentially transformative and differentiated therapeutic option for patients in need."

First Quarter 2024 and Recent Progress

•
Sequential dosing and engraftment have been successfully completed for the three patients in the sentinel cohort of the BEACON Phase 1/2 clinical trial of BEAM-101 in severe sickle cell disease.
•
Following clearance by the data monitoring committee, the expansion cohort of the BEACON trial of BEAM-101, in which patients can be dosed in parallel, is now open with dosing expected to begin imminently.
•
In March 2024, Beam announced the clearance of its clinical trial authorisation (CTA) application by the United Kingdom (UK) Medicines and Healthcare Products Regulatory Agency for BEAM-302, the company’s priority genetic disease program for the treatment of alpha-1 antitrypsin deficiency (AATD).
Key 2024 Anticipated Milestones

Hematology Franchise

•
In the BEACON Phase 1/2 clinical trial of BEAM-101 in adults with severe sickle cell disease, Beam anticipates continuing to enroll and dose patients in the expansion cohort of the trial, with a total target of up to 45 treated patients.
•
The company expects to report data from multiple patients in the BEACON trial in the second half of 2024.
•
Beam continues to advance and invest in its Engineered Stem Cell Antibody Paired Evasion (ESCAPE) conditioning platform and anticipates initiating Phase 1-enabling preclinical studies for the program in 2024.
Genetic Disease Franchise

•
Beam expects to initiate the Phase 1/2 clinical trial for BEAM-302 in AATD in the first half of 2024.
•
Beam expects to submit an investigational new drug (IND) application in the U.S. for BEAM-301 for the potential treatment of glycogen storage disease type 1a (GSD1a) in the first half of 2024.

First Quarter 2024 Financial Results

•
Cash Position: Cash, cash equivalents and marketable securities, were $1.1 billion as of March 31, 2024, compared to $1.2 billion as of December 31, 2023.
•
Research & Development (R&D) Expenses: R&D expenses were $84.8 million for the first quarter of 2024, compared to $99.6 million for the first quarter of 2023.
•
General & Administrative (G&A) Expenses: G&A expenses were $26.7 million for the first quarter of 2024, compared to $23.5 million for the first quarter of 2023.
•
Net Loss: Net loss was $98.7 million for the first quarter of 2024, or $1.21 per share, compared to $96.5 million for the first quarter of 2023, or $1.33 per share.
Cash Runway

Beam expects that its cash, cash equivalents and marketable securities as of March 31, 2024, will enable the company to fund its anticipated operating expenses and capital expenditure requirements into 2027. This expectation includes funding directed toward reaching each of the key anticipated milestones for BEAM-101, ESCAPE, BEAM-301 and BEAM-302 described above, as well as continued investments in platform advancements and manufacturing capabilities.