On March 15, 2021 Agenus Inc. (NASDAQ: AGEN), an immuno-oncology company with an extensive pipeline of checkpoint antibodies, cell therapies, adjuvants, and vaccines designed to activate immune response to cancers and infections, reported financial results for the fourth quarter and full year 2020 (Press release, Agenus, MAR 15, 2021, View Source [SID1234576640]).

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"2020 was a pivotal year for Agenus, marking the beginning of our transition to a commercial company with the initiation of our rolling BLA filing for balstilimab monotherapy. We also reported positive data on multiple programs," said Garo Armen, PhD, Chief Executive Officer of Agenus. "We expect 2021 to be even more impactful, with the expected completion of our balstilimab monotherapy BLA filing in the first half. This filing, followed by an anticipated commercialization, will provide a solid foundation to support the development of our next-generation pipeline. Our best-in-class pipeline reveals true differentiation potential, notably our next-generation anti-CTLA-4 AGEN1181 and our anti-TIGIT bispecific AGEN1777."

Balstilimab (anti-PD-1): BLA completion expected in first half of 2021

Balstilimab accelerated approval in second line cervical cancer expected to be a significant milestone in the transition to a commercial company and a key inflection point for Agenus’ combinations strategy both with its own pipeline agents and with partnered products

Balstilimab shows differentiation from commercial PD-1s and achieves response rates of 19% in PD-L1 positive tumors with 14% in all tumors (PD-L1 positive and negative) and a median duration of response of 15.4 months in a Phase 2 trial. Data presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Virtual Congress 2020 and in an Oncogene editorial

Balstilimab + zalifrelimab Phase 2 trial in second line cervical cancer achieves response rates of 27% in PD-L1 positive tumors with 22% in all tumors (PD-L1 positive and negative) with a median duration of response not yet reached; data presented at ESMO (Free ESMO Whitepaper) 2020. Responses continue to improve as data matures

Discussions with the FDA regarding accelerated BLA filing for balstilimab plus zalifrelimab ongoing; additional guidance and updated response rate data to be provided upon FDA acceptance of balstilimab monotherapy BLA
AGEN1181: Clinical data points to superior next-generation anti-CTLA-4 agent

As of our February 9th report, six confirmed objective clinical responses were achieved in Phase 1/2 trial of AGEN1181 out of 46 evaluable patients: 1 confirmed response among 24 treated with monotherapy, and 5 confirmed responses among 22 treated with AGEN1181 in combination with balstilimab

New clinical data to be presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2021

Optimized Fc-enhanced design differentiates AGEN1181 as a more active next-generation anti-CTLA-4

Responses seen in patients who do not generally respond to first-generation anti-CTLA-4 due to a genetic polymorphism, thus potentially expanding benefit to 3x more patients

Further, responses seen in cold tumor settings (microsatellite stable) and in indications that are generally not responsive to immunotherapy, including colorectal, endometrial, and ovarian

No complement-mediated toxicities typically seen with first-generation anti-CTLA-4 agents

First anti-CTLA-4 to demonstrate clinical depletion of Tregs, immunosuppressive T cells whose depletion can allow for an improved antitumor immune response

Phase 2 trial in colorectal cancer initiated; registrational trials targeted to commence in 2021 with focus on indications enabling a rapid path to BLA filing
AGEN1777 (Anti-TIGIT bispecific): Best-in-class potential; slated for IND filing in 2Q 2021

Superior antibody candidate for bispecific targeting and Fc enhancement, designed for best-in-class performance

Optimized antibody designed to improve upon limited monotherapy activity of other anti-TIGITs

Potential to broaden clinical benefit to additional 40% of patients versus other TIGIT antibodies by expanding benefit to patients with a genetic polymorphism

Bispecific design enables dual blockade of tumor growth, cutting off a potential cancer escape mechanism to TIGIT blocking

IND planned for the second quarter of 2021; Phase 1 study to commence in the third quarter
iNKTs – Intelligent cell therapy: Trial underway in patients with COVID-19; cancer trials to commence in 1H 2021

Preliminary Phase 1 data suggest iNKTs can be dosed with no safety concerns and may demonstrate early signals of activity. Trial expansion is underway

Dose escalation expected to be completed in the first half of 2021 for initiation into a Phase 2 trial

Dosing in Phase 1 study to treat hematologic cancers and solid tumors expected to commence in the first half of 2021
Partnered program MK-4830: Phase 2 initiated; milestone received

MK-4830 (antibody targeting ILT4 licensed to Merck) advanced into Phase 2 in patients with PD-L1 positive advanced non-small cell lung cancer

$10M milestone payment received; Agenus is eligible for up to an additional $85M in potential milestone payments plus royalties. Agenus retains 90% of all milestones from Merck and 67% of future royalties under its Royalty Purchase Agreement with XOMA LLC

MK-4830 positive Phase 1 data presented at ESMO (Free ESMO Whitepaper) 2020
Enhancing talent density to drive key initiatives

Dr. Steven O’Day, pioneer in I-O and anti-CTLA-4 therapy, joins as Chief Medical Officer to drive clinical trial development especially for AGEN1181

Andy Hurley, a seasoned commercial executive, joins as Chief Commercial Officer to head Agenus’ efforts as it prepares for anticipated balstilimab commercialization

Marc Wiles, an expert in new product approvals, joins as Vice President of Regulatory Affairs

Jason Paragas, leader in AI and pandemic surveillance, joins as Divisional Vice President of Strategic Initiatives
Agenus initiatives in adjuvants, biomarker platforms, and predictive AI

First QS-21 milestone payment of $15.1M based on SHINGRIX sales received from Healthcare Royalty Partners; process for large-scale production of renewable raw source of QS-21 advancing to GMP scale-up

Proprietary VISION platform, along with artificial intelligence, designed to allow for discovery of novel biomarkers and design of optimal treatment protocols for patients

Updates on AGEN2373, our anti-CD137 antibody, and AGEN1223, our novel bispecific, will be presented at future scientific and medical conferences
Strategic partnerships expand potential of Agenus molecules

Betta Pharmaceuticals licensing agreement for balstilimab and zalifrelimab in Greater China; Agenus received $35M in cash and equity and is eligible for up to $100M in milestones plus royalties

Rottapharm Biotech access program for balstilimab in combination with CR6086, a potent and selective prostaglandin EP4 receptor antagonist, in patients with advanced metastatic colorectal cancer

Nelum Pharmaceuticals clinical collaboration for zalifrelimab in combination with NLM-001, Nelum’s small molecule hedgehog inhibitor, and chemotherapy for first-line advanced pancreatic cancer
Fourth Quarter and Full Year 2020 Financial Results

For the year ended December 31, 2020, we recognized revenue of $88 million which includes revenue related to the upfront license fee from our transaction with Betta plus non-cash royalties and milestones earned. For the year ended 2019 we recorded revenue of $150 million which included revenue related to the upfront license fee from our transaction with Gilead and milestones earned, in addition to non-cash royalties earned.

Net loss for the fourth quarter was $38 million or $0.20 per share compared to a net loss for the same period in 2019 of $31 million, or $0.22 per share. Net loss for the year ended 2020 was $183 million or $1.05 per share compared to a net loss for the year ended 2019 of $112 million or $0.80 per share.

Fiscal year end 2020 cash balance was $100 million as compared to $62 million at fiscal year end 2019.

Conference Call

Dial-in numbers: (833) 614-1394 (US) or (914) 987-7115 (International); Conference ID: 2339028.

Webcast
A live webcast and replay of the conference call will be accessible from the Events & Presentations page of the Company’s website at View Source and via View Source

On March 15, 2021 Rubius Therapeutics, Inc. (Nasdaq:RUBY), a clinical-stage biopharmaceutical company that is genetically engineering red blood cells to create an entirely new class of cellular medicines called Red Cell Therapeutics, reported initial clinical, pharmacodynamic and tumor trafficking data from its ongoing Phase 1/2 clinical trial of RTX-240 in patients with advanced solid tumors (Press release, Rubius Therapeutics, MAR 15, 2021, View Source [SID1234576635]). The Company also shared tumor trafficking data from one patient with relapsed/refractory acute myeloid leukemia (AML) in the second Phase 1 arm of the study. The Company believes these data provide initial proof-of-concept of the RED PLATFORM by providing evidence that red blood cells can be engineered to mimic the human immune system and stimulate adaptive and innate immunity to generate clinical responses in cancer patients with refractory disease.

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"These initial data are incredibly exciting and demonstrate that RTX-240 has the potential to generate single-agent activity in patients with solid tumors, including a cold tumor such as metastatic uveal melanoma, where other treatments have failed to induce responses in patients," said Christina Coughlin, M.D., Ph.D., chief medical officer at Rubius Therapeutics. "The encouraging safety results, including a single event of Grade 1 liver toxicity, and preliminary efficacy data for RTX-240 to date give us the potential to realize the power of immune agonists for the treatment of cancer."

Initial Efficacy Data

Five (5) dose cohorts were completed in the solid tumor trial at the time of the data cutoff on February 28, 2021 (n=16), with 16 patients evaluable for safety (primary outcome measure) and 15 patients evaluable for efficacy (secondary outcome measure) based on RECIST v1.1.

The study is continuing to enroll patients and despite the fact that dose optimization is still ongoing, RTX-240 generated:

A confirmed partial response (PR) with a 54% reduction in the target lesions at the 1e8 dose administered every 4 weeks in a patient with metastatic anal cancer whose disease had progressed on anti-PD-L1 therapy. Treatment of this patient was ongoing 8 months following the first dose at data cutoff;
An unconfirmed PR with complete resolution of the target hepatic lesion and resolution of 14/15 hepatic lesions at the 1e10 dose administered every 4 weeks in a patient with metastatic uveal melanoma whose disease had progressed on anti-PD-1 therapy. Treatment of this patient was ongoing 4 months following the first dose at data cutoff; and
Stable disease (SD) was observed in 6 patients, including 4 individual patients with stable disease for at least 12 weeks:
Non-small cell lung cancer (disease stabilization for 12 weeks with treatment ongoing as of the data cutoff);
Soft tissue sarcoma (disease stabilization for 4 months);
Pancreatic cancer (disease stabilization for 3 months); and
Prostate cancer (disease stabilization for 4 months).
"We believe these data provide clinical validation of our RED PLATFORM and de-risk our oncology pipeline of Red Cell Therapeutics," said Pablo J. Cagnoni, M.D., president and chief executive officer. "Given the encouraging initial safety and preliminary efficacy data for RTX-240, we plan to initiate a Phase 2 expansion cohort in the first quarter of 2022, and a new Phase 1 arm of the ongoing RTX-240 clinical trial to evaluate RTX-240 in combination with anti-PD-1 therapy in patients with advanced solid tumors during the second half of 2021."

Initial Safety Data

The most common treatment-related Grade 1/2 adverse events were fatigue (n=4), chills, nausea, decreased appetite and arthralgias all reported in 3 patients each. There were no treatment-related Grade 3/4 adverse events, no dose-limiting toxicities and a single Grade 1 event of liver toxicity.

Ten (10) immune-related adverse events (irAE) were observed among 5 patients with no reported treatment-related Grade 3/4 irAEs. Grade 2 treatment-related irAEs included pneumonitis (n=1), adrenal insufficiency (n=1) and hypothyroidism (n=1).

Pharmacodynamic Data

In addition to evaluating safety and preliminary efficacy data, the trial is evaluating the pharmacodynamic effects of RTX-240:

RTX-240 stimulated innate and adaptive immunity as demonstrated by the activation and/or expansion of NK or memory CD8+ T cells in all patients, with 9/16 patients showing activation and expansion in both cell types. There was an overall trend towards a dose response in absolute NK cell numbers (expansion);
Detailed NK cell analysis showed an increased percentage of CD16+CD56dim (mature NK cells) and CD56bright (immature NK cells) across dose levels
These cell subtypes are associated with cytotoxicity and cytokine production respectively; and
RTX-240 induced expression of key NK cell activation receptors, including NKp30 and the ratio of cells expressing the activation receptor NKG2D versus the inhibitory receptor NKG2A
This specific signature of NK cell receptor expression may allow selection of specific tumor types with predicted sensitivity to RTX-240.
Tumor Infiltration Data

Immune cell trafficking into the tumor microenvironment (TME) was assessed by tumor biopsies from participating patients with solid tumors (optional; n=4) and AML (standard of care; n=1).

Trafficking of T and NK cells into the TME was observed in 3/5 patients (1.6 to 10-fold increases), including one patient each with metastatic mesothelioma, metastatic soft tissue sarcoma and refractory AML. Tumor infiltration was not observed in patients with ovarian cancer (n=1) and heavily pretreated melanoma (n=1);
There was increased expression of PD-L1 observed in 3/4 patients with solid tumors, suggesting an improved immune-permissive TME; and
In one patient with AML, trafficking of T and NK cells into the bone marrow was associated with increases in the cellularity of the marrow.
Upcoming Anticipated Milestones

In order to realize the full potential of RTX-240, the Company’s other oncology programs and the RED PLATFORM, in the next 12 months, Rubius plans to execute several critical milestones:

Present additional clinical results from the RTX-240 solid tumor Phase 1 clinical trial;
Select the recommended RTX-240 Phase 2 dose, schedule and specific solid tumor types that will be pursued in the Phase 2 expansion cohort;
Report initial clinical results for the second Phase 1 arm of the RTX-240 clinical trial in relapsed/refractory AML;
Initiate the Phase 1 clinical trial of RTX-240 in combination with anti-PD-1 therapy in advanced solid tumors in 2H’21;
Report initial Phase 1 clinical results for RTX-321 for the treatment of HPV 16-positive cancers by 1Q’22; and
Submit an Investigational New Drug Application for RTX-224 by year-end.
Conference Call

The Company will host a conference call and webcast at 8:00 a.m. EDT to discuss this update. The audio webcast will be available on the Events and Presentations page within the Investors and Media section of the Rubius Therapeutics website. The update may also be accessed by dialing 1-800-289-0045 (domestic) or 1-615-622-8086 (international) five minutes prior to the start of the call and providing the passcode 1294064. An archived webcast will be accessible for 90 days after the event.

About RTX-240

RTX-240 is an allogeneic, off-the-shelf cellular therapy product candidate that is designed to simultaneously present hundreds of thousands of copies of the costimulatory molecule 4-1BB ligand (4-1BBL) and IL-15TP (trans-presentation of IL-15 on IL-15Rα) in their native forms. RTX-240 is expected to broadly stimulate the immune system by activating and expanding both NK and memory T cells to generate a potent anti-tumor response.

About the RTX-240 Clinical Trial

This is a Phase 1/2 open label, multicenter, multidose, first-in-human dose-escalation and expansion study designed to determine the safety and tolerability, pharmacokinetics, maximum tolerated dose and a recommended Phase 2 dose and dosing regimen of RTX-240 in adult patients with relapsed/refractory or locally advanced solid tumors or with relapsed/refractory acute myeloid leukemia. The trial will also assess the pharmacodynamics of RTX-240 measured by changes in T and NK cell number and function relative to baseline and anti-tumor activity. The study will include a monotherapy dose escalation phase followed by an expansion phase in specified tumor types during the Phase 2 portion of the trial. The extent to which the COVID-19 pandemic may impact Rubius’ ability to enroll patients in the trial will depend on future developments.

About RTX-321

RTX-321 is an allogeneic, off-the-shelf artifical antigen-presenting cell therapy product candidate that is designed to express hundreds of thousands of copies of an HPV peptide antigen bound to major histocompatibility complex class I proteins, the costimulatory molecule 4-1BBL and the cytokine IL-12 on the cell surface to mimic human T cell-APC interactions. In preclinical studies, RTX-321 was shown to have a dual mechanism of action by functioning as an antigen-presenting cell to boost HPV 16 antigen-specific T cell responses and promoting broad immune system stimulation of both innate and adaptive immunity.

About RTX-224

RTX-224 is an allogeneic cellular therapy product candidate that is engineered to express hundreds of thousands of copies of 4-1BBL and IL-12 on the cell surface. In contrast to RTX-240, RTX-224 is designed as a broader T cell- agonist, while also retaining the ability to activate and expand NK cells. It is expected to produce a broad and potent anti-tumor T cell response, an innate immune response and show activity in those tumor types with known sensitivity to T cell killing, including tumor types with high mutational burden, PD-L1 expression and prior activity of checkpoint inhibitors.

On March 15, 2021 VBI Vaccines Inc. (Nasdaq: VBIV) (VBI), a biopharmaceutical company driven by immunology in the pursuit of powerful prevention and treatment of disease, reported that Jeff Baxter, President and Chief Executive Officer, and David E. Anderson, Ph.D., Chief Scientific Officer, will participate in an analyst-led fireside chat at the Oppenheimer 31st Annual Healthcare Conference on Wednesday, March 17 at 10:40 AM ET (Press release, VBI Vaccines, MAR 15, 2021, View Source [SID1234576634]).

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Presentation Details:

- Event: Oppenheimer 31st Annual Healthcare Conference
- Presenters: Jeff Baxter, President and CEO, and David E. Anderson, Ph.D., Chief Scientific Officer
- Date: Wednesday, March 17, 2021
- Time: 10:40 – 11:10 AM ET
- Webcast: View Source

A recording of the webcast will be available on the "Events and Presentations" page of the Company’s website: View Source

On March 15, 2021 Genmab A/S (Nasdaq: GMAB). On February 23, 2021 Genmab announced the initiation of a share buy-back program to mitigate dilution from warrant exercises and to honor our commitments under our Restricted Stock Units program (Press release, Genmab, MAR 15, 2021, View Source [SID1234576633]).

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The share buy-back program is expected to be completed no later than June 30, 2021 and comprises up to 200,000 shares.

The following transactions were executed under the program from March 8, 2021 to March 12, 2021:

Details of each transaction are included as an appendix to this announcement.

Following these transactions, Genmab holds 145,477 shares as treasury shares, corresponding to 0.22% of the total share capital and voting rights.

The share buy-back program is undertaken in accordance with Regulation (EU) No. 596/2014 (‘MAR’) and the Commission Delegated Regulation (EU) 2016/1052, also referred to as the "Safe Harbour Regulation." Further details on the terms of the share buy-back program can be found in our company announcement no. 11 dated February 23, 2021.

On March 15, 2021 ElevateBio, a cell and gene therapy technology company focused on powering transformative cell and gene therapies, reported it has raised $525 million in a Series C financing (Press release, ElevateBio, MAR 15, 2021, View Source [SID1234576632]). ElevateBio has created a new disruptive business model addressing the most pressing challenges in the field of cell and gene therapy. With an unprecedented suite of next generation-enabling technologies; including gene editing, induced pluripotent stem cells, and protein, viral, and cellular engineering; the company is changing the way the field advances cell and gene therapies. ElevateBio is positioned to power the industry’s most promising therapies with an "Elevate Inside" approach by democratizing access to its technologies, and using ElevateBio BaseCamp (a technology-enabled process development and CGMP manufacturing capability) to advance transformative therapies across the entire industry. With this financing, ElevateBio will continue to develop and expand its technology platforms, build upon its network of process development and GMP manufacturing capacity, and advance an increasing number of industry partnerships, while also continuing to develop its own highly innovative cell and gene therapies.

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Matrix Capital Management led the Series C financing alongside additional new investors SoftBank Vision Fund 2* and Fidelity Management & Research Company who joined ElevateBio’s existing investors: MPM Capital, F2 Ventures, Redmile Group, EcoR1 Capital, Samsara BioCapital, The Invus Group, Emerson Collective, Surveyor Capital (A Citadel company), EDBI, and Vertex Ventures, iTochu, and a large insurance company.

"ElevateBio has created the first fully-integrated technology company exclusively focused on cell and gene therapy, disrupting the current paradigm by providing end-to-end capabilities that enable partners a strategic advantage in the market and, ultimately, meet the urgent need of patients and families suffering with life-threatening and devastating diseases," said David Hallal, Chairman and Chief Executive Officer of ElevateBio. "While we see remarkable breakthroughs in the earliest days of the cell and gene therapy revolution, accelerating innovation requires next-generation technology, analytics, and production capabilities to deliver therapies better, faster, and cheaper. We are poised to power the field today and for many decades to come and are thrilled to welcome Matrix Capital Management, SoftBank, and Fidelity to our stellar group of existing investors."

Mitchell Finer, Ph.D., Chief Scientific Officer of ElevateBio, President of ElevateBio BaseCamp, and Chief Executive Officer of LifeEDIT Therapeutics continued by saying, "In my nearly 35 years of driving cell and gene therapy innovation across a range of technologies and therapeutics, I have seen much progress toward our ambition to serve patients with intractable diseases – yet innovation is often delayed by technology limitations and the lack of manufacturing robustness. At ElevateBio, we can realize the full potential of cell and gene therapies, by re-envisioning the way these products are made, breaking down silos, leveraging powerful enabling technologies, and changing the mindset from simple manufacturing scale-up to conducting large scale biology. This approach will drive transformative cell, gene, and regenerative therapies today and tomorrow that have the potential to enable access for patients around the globe."

As part of the financing, two new members, Karan Takhar, Senior Managing Director at Matrix Capital Management and Deep Nishar, Senior Managing Partner, SoftBank Investment Advisers, will join ElevateBio’s Board of Directors.

Mr. Takhar leads and oversees key portfolio investments at Matrix Capital. He is a member of the Board of Directors at Encoded Therapeutics, Palleon Pharmaceuticals, and Zentalis Pharmaceuticals. Before joining Matrix, Mr. Takhar spent time working for High Vista Strategies, Goldman Sachs Investment Strategies, and Moody’s Investor Service.
Mr. Nishar leads life sciences, frontier tech, and enterprise software investments at SoftBank Investment Advisers. He has more than 20 years of experience in helping build and grow businesses, and sits on the board of directors of Encoded Therapeutics, Relay Therapeutics, Seer and Vir Bio, amongst other companies. Prior to joining SoftBank, Mr. Nishar served as Senior Vice President of Products and User Experience at LinkedIn.
"ElevateBio’s business model maximizes the potential to capitalize on the convergence of technology and healthcare, creating an entirely new category in cell and gene therapy," said Karan Takhar. "The company is redefining the way cell and gene therapies are discovered, developed, and manufactured, bringing together cutting-edge scientific and digital technologies to enable a seismic shift in the field."

Deep Nishar added, "We are proud to support ElevateBio’s world-class team to expand and advance its technology and manufacturing platforms to accelerate the production and development of tomorrow’s life-saving cell and gene therapies."

Addressing the need in cell and gene therapy:

There are nearly a thousand cell and gene therapies in development targeting a broad range of diseases, yet as of February 2020, only nine cell or gene therapies were approved in the U.S. Despite the small number of approved therapies, the global cell and gene therapy market is expected to reach nearly $50 billion by 2027. The U.S. Food and Drug Administration (FDA) predicts that by 2025, they will be approving 10 to 20 cell and gene therapy products a year based on an assessment of the current pipeline and the clinical success rates of these products. Scaling production from a few million cells in the lab to manufacturing the billions of cells needed to treat patients is uniquely complex. While innovation has greatly advanced in the discovery and development of cell and gene therapies, the approach to manufacturing these novel medicines has largely relied on traditional modes of development or a patchwork of technologies and providers, hampering and slowing their impact on human health.

ElevateBio was built to bring the insights, skills, and technological know-how needed to navigate these complex challenges in a repeated fashion to advance transformative cell and gene therapies to patients whether they are ElevateBio’s own therapeutics or the innovative therapies of its partners.