Alexion completes purchase and licence agreement for early-stage rare disease gene therapy portfolio from Pfizer

On September 19, 2023 Alexion, AstraZeneca Rare Disease, reported that it has completed a definitive purchase and licence agreement for a portfolio of preclinical rare disease gene therapy programmes and enabling technologies from Pfizer Inc. (Pfizer) (Press release, Alexion, SEP 19, 2023, View Source [SID1234635255]).

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These new resources build on the combined capabilities of Alexion and AstraZeneca in genomic medicine, with the objective to develop new genetic therapies with improved safety and efficacy profiles. Additionally, several of the Pfizer employees associated with the portfolio will join Alexion as employees.

Financial considerations
Alexion has purchased and licenced the assets of Pfizer’s early-stage rare disease gene therapy portfolio for a total consideration of up to $1bn, plus tiered royalties on sales.

Precigen to Participate in Upcoming Leading Investor and Industry Conferences

On September 19, 2023 Precigen, Inc. (Nasdaq: PGEN), a biopharmaceutical company specializing in the development of innovative gene and cell therapies to improve the lives of patients, reported that Helen Sabzevari, PhD, President and CEO of Precigen, will participate in the following upcoming investor and industry conferences (Press release, Precigen, SEP 19, 2023, View Source [SID1234635254]):

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Cantor Global Healthcare Conference 2023
Fireside chat on Tuesday, September 26 at 4:55 PM ET
New York, NY

Cell & Gene Meeting on the Mesa
Company presentation on Wednesday, October 11, 2023 at 3:30 PM PT
Carlsbad, CA

Cantor AML Virtual Panel
Panel discussion on acute myeloid leukemia (AML) on Thursday, October 26, 2023 at 2:00 PM ET
Virtual

Participants may view details for these events through Precigen’s website in the Events & Presentations section at investors.precigen.com/events-presentations.

Vincerx Pharma to Participate at the Cantor Fitzgerald Global Healthcare Conference 2023

On September 19, 2023 Vincerx Pharma, Inc. (Nasdaq: VINC), a biopharmaceutical company aspiring to address the unmet medical needs of patients with cancer through paradigm-shifting therapeutics, reported that Vincerx’s CEO Ahmed Hamdy, M.D. and the Management Team will participate in a panel presentation and one-on-one investor meetings at the Cantor Fitzgerald Global Healthcare Conference 2023, to be held in New York, NY, September 26-28, 2023 (Press release, Vincerx Pharma, SEP 19, 2023, View Source [SID1234635253]).

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Conference Presentation Details

Panel Date/Time:
Wednesday, September 27, 2023 at 9:10 a.m. ET
Speaker: CEO Ahmed Hamdy, M.D.
Investor Access: View Source
1X1 meetings The Vincerx Management Team will be available for 1X1 meetings during the conference. Those interested in requesting a meeting should contact their Cantor Fitzgerald sales representative.

A copy of the Company corporate presentation is available here and a recording of the panel presentation can be accessed through the "Investor Calendar" section of the Vincerx website where it will be archived for 90 days.

Revolution Medicines Doses First Patient in Phase 1/1b Clinical Trial of RMC-9805, an Oral, Covalent, Mutant-Selective KRASG12D(ON) Inhibitor

On September 19, 2023 Revolution Medicines, Inc. (Nasdaq: RVMD), a clinical-stage oncology company developing targeted therapies for RAS-addicted cancers, reported the first patient was dosed in its Phase 1/1b monotherapy clinical trial of RMC-9805, an oral, covalent, mutant-selective KRASG12D(ON) Inhibitor designed to treat patients with cancers driven by the KRASG12D mutation (Press release, Revolution Medicines, SEP 19, 2023, View Source [SID1234635252]). KRASG12D is the most common driver of RAS-addicted human cancers, accounting for nearly 55,000 newly diagnosed patients in the U.S. annually, predominantly among patients with pancreatic cancer, non-small cell lung cancer (NSCLC), and colorectal cancer.

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The Phase 1/1b trial (NCT06040541) is a multicenter, open-label, dose-escalation and dose-expansion study of RMC-9805 in patients with advanced solid tumors harboring the KRASG12D mutation. The primary objectives of the study are to evaluate safety and tolerability, and to inform the recommended Phase 2 dose and schedule for the compound.

"The initiation of patient dosing with RMC-9805 marks a major milestone for Revolution Medicines as its third oral RAS(ON) Inhibitor to begin clinical evaluation," said Mark A. Goldsmith, M.D., Ph.D., chief executive officer and chairman of Revolution Medicines. "We are now studying in the clinic three highly innovative RAS(ON) Inhibitors derived from our pioneering tri-complex inhibitor platform that we believe have complementary profiles – RMC-6236 (RASMULTI) for patients with cancers caused by a wide range of RAS mutations, and the mutant-selective compounds RMC-6291 (KRASG12C) and RMC-9805 (KRASG12D) for patients with cancers harboring selected mutations. With this strong clinical portfolio, as well as a rich collection of additional mutant-selective drug candidates and research-stage assets, we believe our pipeline has the potential to change the standard of care for patients living with a wide range of RAS-addicted cancers including NSCLC, pancreatic cancers and colorectal cancers."

ReCode Therapeutics Announces Closing of Extension to Series B Financing

On September 19, 2023 ReCode Therapeutics, a clinical-stage genetic medicines company using precision delivery to power the next wave of mRNA and gene correction therapeutics, reported the closing of an extension to its Series B financing, raising an additional $50 million, and the appointment of Kouki Harasaki, Ph.D., founding and managing partner of Bioluminescence Ventures (BLV), to the company’s board of directors (Press release, ReCode Therapeutics, SEP 19, 2023, View Source;utm_medium=rss&utm_campaign=recode-therapeutics-announces-closing-of-extension-to-series-b-financing [SID1234635251]).

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The company recently concluded a final extension to its Series B financing, raising an additional $50 million, for a total of $260 million in funding.

New investors in the extension include BLV and Solasta Ventures
The new investor proceeds were backed by strong support from existing investors, including OrbiMed Advisors, AyurMaya, an affiliate of Matrix Capital Management, Leaps by Bayer, Vida Ventures, MPM Capital, Pfizer Ventures, EcoR1 Capital, Sanofi Ventures and Amgen Ventures, Osage University Partners (OUP), among others
Proceeds will be used to advance ReCode’s primary ciliary dyskinesia and cystic fibrosis clinical development programs and to expand the company’s proprietary Selective Organ Targeting (SORT) lipid nanoparticle (LNP) pipeline to include mRNA and gene correction therapeutics for central nervous system, lung, liver and musculoskeletal indications
Dr. Harasaki is the founding and managing partner at BLV. He brings more than 25 years of biomedical science experience in multiple therapeutic areas across major health systems, research institutes, biopharmaceutical corporations, technology companies and venture capital firms. Prior to BLV, he was managing director at M12/Microsoft Ventures, where he led life science investments and helped develop Microsoft’s corporate strategy in the field. Before M12, Dr. Harasaki was a senior partner at Andreessen Horowitz.

"We are delighted to welcome Kouki to the board of directors and are confident his broad experience across many key areas such as drug discovery, strategy, finance and business development will be invaluable in guiding ReCode as it expands its robust clinical development plans in a number of important genetic medicine indications," said Shehnaaz Suliman, M.D., MBA, M.Phil., chief executive officer, ReCode Therapeutics. "We are excited with our progress to the clinic as we advance our SORT LNP delivery platform, the first technology to enable highly targeted delivery of genetic medicines to organs, tissues and cells including and beyond the liver."

"I am excited to join the ReCode team at this important juncture in its development. At BLV, we focus on funding next-generation therapeutics platforms and developing first- and best-in-class programs. ReCode, with its cutting-edge genetic medicine platform, is well aligned with our mission," said Dr. Harasaki. "I look forward to working with the board and the senior leadership team at ReCode to advance the next wave of genetic medicines to address a wide range of medical needs not possible with current therapies."

"Throughout 2023, we made tremendous progress entering the clinic, strengthening our financial position and building out our leadership team to support our genetic medicines clinical development programs," added Dr. Suliman. "We are delighted with the continued high level of interest in our novel approach to the targeted delivery of genetic medicines from premier venture investors. We remain focused on achieving important upcoming clinical milestones, including dosing the first patients in our Phase 1 trial of RCT1100 for primary ciliary dyskinesia and we are also on track to file a number of investigational new drug applications with global regulators for RCT2100, our cystic fibrosis candidate, later this year."