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Pfizer to Acquire GBT for $5.4B, Adding Sickle Cell Disease Drug and Pipeline

Pfizer said the deal will complement and further enhance its 30-plus year heritage in rare hematology and reinforce its commitment to treating sickle cell…

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Global Blood Therapeutics (GBT) President and CEO Ted W. Love, MD

Pfizer has agreed to acquire Global Blood Therapeutics (GBT) for $5.4 billion cash, the companies said Monday, in a deal that continues the pharma giant’s spate of acquisitions made possible by the billions of dollars generated by its COVID-19 vaccine and antiviral drug.

Pfizer said the deal will complement and further enhance its 30-plus year heritage in rare hematology and reinforce its commitment to treating sickle cell disease (SCD), for which GBT has a marketed drug Oxbryta® (voxelotor) approved by the FDA in 2019.

That year, Pfizer acknowledged that a synthetic glycomimetic it licensed from GlycoMimetics, rivipansel, had failed a Phase III trial in patients with SCD who were hospitalized for a vaso-occlusive crisis (VOC) and required treatment with intravenous opioids. Soon after, Pfizer ended an up-to-$340 million ($15 million upfront) collaboration with GlycoMimetics launched in 2011.

Rivipansel wasn’t Pfizer’s only SCD candidate in recent years. In 2020, Pfizer quietly ended a Phase I trial of PF-04447943, only a year after publication of a preclinical study that the phosphodiesterase 9A inhibitor “demonstrated PK/PD effects suggestive of inhibiting pathways that may contribute to vaso-occlusion.” PF-04447943 has since been dropped from Pfizer’s pipeline.

Pfizer said GBT’s expertise, portfolio, and pipeline, holds potential to address the full spectrum of critical needs for the SCD community, which Pfizer has committed to engaging through the deal.

“We are excited to welcome GBT colleagues into Pfizer and to work together to transform the lives of patients, as we have long sought to address the needs of this underserved community,” Albert Bourla, Pfizer’s Chairman and CEO, said in a statement. “The deep market knowledge and scientific and clinical capabilities we have built over three decades in rare hematology will enable us to accelerate innovation for the sickle cell disease community and bring these treatments to patients as quickly as possible.”

The Wall Street Journal first reported Pfizer’s advanced talks to acquire GBT on Friday, about two weeks after a Bloomberg News report identified GBT as a potential takeover target, touching off a 41% jump on the company’s share price August 4, from $33.93 to $47.99.

Shares of GBT rose 4% to $66.64 in midday trading as of 12:54 p.m. ET.  Pfizer shares dipper 0.43% to $49.06 from $49.27.

51% Sales gain

Citing the acquisition announcement, GBT postponed its scheduled quarterly conference call with analysts, but did release second quarter results showing product sales of $71.55 million for Oxbryta, up nearly 51% from $47.555 million in Q2 2021.

The Q2 sales result fell in line with the forecast of analyst John Newman, PhD, CFA of Canaccord Genuity, who early Monday before the deal was announced raised his quarterly sales forecast for Oxbryta from $64 million to $71 million, citing strong pediatric uptake of the drug.

Global Blood Therapeutics President and CEO Ted W. Love, MD, said a crucial factor behind the successful development of Oxbryta was shifting the R&D focus from relieving pain, the traditional concern of sickle cell disease (SCD) drug developers, to improving the health of red blood cells. [Global Blood Therapeutics]
“We expect faster Oxbryta uptake in pediatric sickle cell disease patients vs adolescents or adults, as we believe parents and physicians see greater urgency to treat the disease in young children,” Newman wrote in a research note. “We also expect better compliance and adherence for younger patients overall.”

So far this year, Oxbryta has generated $126.71 million in product sales, up 46% from $86.598 million in the first half of 2021. Oxbryta racked up $194.749 million in product sales in 2021, up 57% from $123.803 million in 2020.

Pfizer said it plans to build on those sales by leveraging its global platform to accelerate distribution of Oxbryta to parts of the world most impacted by SCD. In addition to the U.S., Oxbryta is approved in the European Union, United Arab Emirates, Oman and Great Britain.

Speaking on GEN’s “Close to the Edge” video interview series last year, GBT President and CEO Ted W. Love, MD, complimented Pfizer as well as Moderna for their rapid development of COVID-19 vaccines [Pfizer partnered with BioNTech to develop COMIRNATY®] based on messenger RNA (mRNA).

“The application of that technology positioned Moderna and Pfizer to really do something that I think people would have thought unfathomable a few years ago. So I think the rate of acceleration of us being able to use novel technology, novel science, it’s accelerating.  And I think that’s going to continue, which is why I’m so excited and, quite frankly, proud of our industry,” Love said.

Pfizer’s successful development of COMIRNATY generated billions of dollars for the company—$36.781 billion in 2021 and $22.075 billion in the first six months of this year alone.

The company has since spent some of that money on an acquisition spree. Since the vaccine was emergency-authorized in 2020, Pfizer last year bolstered its oncology drug portfolio by acquiring Trillium Therapeutics for $2.26 billion.

In April, Pfizer shelled out up to $525 million for ReViral, a developer of antiviral therapeutics targeting respiratory syncytial virus (RSV). A month later, Pfizer bought Biohaven Pharmaceuticals for $11.6 billion cash, expanding its portfolio and pipeline with therapies for migraine led by the FDA-approved rimegepant (Nurtec® ODT).

“We’ve been very clear that our goal is to add $25 billion of risk adjusted revenue by 2030 and I think we are making very good progress against that,” Aamir Malik, Chief Business Innovation Officer, told analysts July 28 on the company’s quarterly earnings call. “This year you saw our transaction with ReViral and subsequently with Biohaven, which respectively we believe have the potential to add $1.5 billion and $6 billion in peak sales to our business, and this was on the back of a very active 2021

Looking beyond pain

On GEN’s “Close to the Edge,” Love said a crucial factor behind the successful development of Oxbryta was shifting the R&D focus from relieving pain, the traditional concern of SCD drug developers, to improving the health of red blood cells.

“We really needed to kind of flip how people were thinking from not just pain but, what is really going on in sickle cell disease? And we’ve been now trying to get people to understand that a disease that occurs on one protein that’s trapped inside your red cell is really killing the red cells first. It’s killing the red cell first,” Love said on “Close to the Edge.”

“I was very strident early on. We are not going to develop a drug focused on pain. We are going to focus on the fundamental basis of this disease, which is the hemoglobin polymerization that’s killing the red cell. And what we ultimately agreed to with the Food and Drug Administration is that the primary endpoint for our drug’s approval would be stopping the polymerization from killing the red cell,” Love added.

Oxbryta won accelerated FDA approval in November 2019 as a treatment for SCD in patients 12 years old and older. The FDA based its approval on positive clinical data Oxbryta generated in the Phase III HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization) trial (NCT03036813). Of 274 patients evaluated, more than 50% treated with Oxbryta achieved greater than 1 g/dL increase in hemoglobin, compared with 6.5% receiving placebo.

Oxbryta is indicated as an SCD treatment in patients ages 4 and up. The drug is also under clinical review in:

  • SCD patients 2-15 years of age, the focus of the Phase III HOPE-KIDS 2 trial (NCT04218084), a confirmatory study designed to measure the mean change in transcranial doppler (TCD) flow velocity, seeking to demonstrate a decrease in stroke risk. HOPE-KIDS 2 was initiated in December 2019 as a condition of the accelerated approval.
  • SCD pediatric patients up to 17 years of age, the focus of the Phase IIa HOPE-KIDS 1 trial (GBT440-007; NCT02850406), an open-label, single- and multiple-dose trial evaluating the safety, tolerability, pharmacokinetics and exploratory treatment effect of Oxbryta in pediatric patients as young as 6 months of age..
  • SCD patients 12 years of age and older, the focus of a planned Phase IV study set to evaluate daily physical activity in those patients.

Love has also defended Oxbryta’s $125,000/year list price, which GBT had pledged not to raise through this year, citing the drug’s efficacy against SCD and savings to SCD patients of over $700,000 in otherwise lost lifetime income due to the disease.

“If you go into a disease area where there’s nothing available, looking at the alternative price value of the alternative is a silly discussion. The alternative is to continue to let these patients suffer and die,” Love told investors attending the Biotechnology Innovation Organization (BIO) CEO & Investor Conference in New York in February 2020.

$3 Billion franchise

Also in GBT’s pipeline is inclacumab, a fully human monoclonal antibody targeting P-selectin which is under study in two Phase III trials as a potential quarterly treatment to reduce the frequency of painful vaso-occlusive crises (VOCs) and reduce hospital readmission rates due to VOCs.

GBT has an exclusive worldwide up-to-$127 million-plus licensing agreement with Roche for inclacumab inked in 2018 –$2 million paid upfront to Roche, up to $125 million tied to achieving development and commercialization milestones, plus tiered royalties. Inclacumab has established pharmacokinetic data, safety and tolerability in more than 500 patients.

GBT’s pipeline also includes GBT601 (formerly GBT021601), an oral, once-daily, next-generation sickle hemoglobin (HbS) polymerization inhibitor under study in Phase I and Phase II/III trials. The Phase I trial (NCT04983264) was completed in late 2021, then restarted in the second quarter of 2022 to evaluate a higher daily dose of GBT601 than previously studied, based on patient demand. In June, GBT announced the initiation of the Phase II portion of the Phase II/III trial (NCT05431088), with the primary outcome measure of the Phase II portion being the number of patients with a change from baseline in hemoglobin, or Hb, levels through Week 12.

GBT has presented positive Phase I proof-of-concept data for GBT601 at the American Society of Hematology’s ASH 2021 conference, held December 11-14 in Atlanta. GBT said the data has shown GBT601’s potential to improve clinical outcomes in people living with SCD, while reducing pill burden.

Both GBT601 and inclacumab have received FDA Orphan Drug and Rare Pediatric Disease designations.

GBT’s pipeline includes preclinical programs designed to treat SCD through HbF inducers (which could also potentially treat beta thalassemia), through a collaboration with Syros Pharmaceuticals; anti-sickling drugs; and inflammation and oxidative stress reduction.

If approved, GBT’s pipeline and Oxbryta could underpin an SCD franchise that according to the company could achieve combined worldwide peak sales of more than $3 billion.

Pfizer said it will acquire all outstanding shares of GBT for $68.50 per share in cash, a 7% premium to GBT’s closing share price of $63.84 on Friday. The deal’s total enterprise value of approximately $5.4 billion includes debt and is net of cash acquired.

Pfizer said it will buy GBT using cash on hand. The company reported $2.47 billion in cash and cash equivalents as of April 3; Pfizer has yet to disclose its second quarter balance sheets.

The boards of both companies have unanimously approved the transaction, which is subject to customary closing conditions, including receipt of regulatory approvals and approval by GBT’s stockholders.

“Pfizer will broaden and amplify our impact for patients and further propel much-needed innovation and resources for the care of people with sickle cell disease and other rare diseases, including populations in limited-resource countries,” Love said Monday in a statement. “We look forward to working together with Pfizer to serve our communities and advance our shared goal of improving health equity and expanding access to life-changing treatments to create a healthier future for all.”

The post Pfizer to Acquire GBT for $5.4B, Adding Sickle Cell Disease Drug and Pipeline appeared first on GEN - Genetic Engineering and Biotechnology News.

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EUR/AUD bearish breakdown supported by additional China fiscal stimulus and AU inflation

Weak PMI readings from the Eurozone, an increase in China’s budget deficit ratio, and renewed inflationary pressures in Australia may trigger a persistent…

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October 24, 2023

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  • Weak PMI readings from the Eurozone, an increase in China’s budget deficit ratio, and renewed inflationary pressures in Australia may trigger a persistent bearish sentiment loop in EUR/AUD.
  • Watch the key short-term resistance at 1.6700 for EUR/AUD.
  • A break below 1.6250 key medium-term support on the EUR/AUD may trigger a multi-week bearish impulsive down move.

The Euro (EUR) tumbled overnight throughout the US session as it erased its prior gains against the US dollar recorded on Monday, 23 October; the EUR/USD shed -104 pips from yesterday’s intraday high of 1.0695 to close the US session at 1.0591, its weakest performance in the past seven sessions.

Yesterday’s resurgence of the USD dollar strength has been attributed to a robust set of October flash manufacturing and services PMI data from the US in contrast with weak readings seen in the UK and Eurozone that represented stagflation risks.

Interestingly, the Aussie dollar (AUD) has outperformed the US dollar where the AUD/USD managed to squeeze out a minor daily gain of 21 pips by the close of yesterday’s US session. The resilient movement of the AUD/USD has been impacted by positive news flow out from China, Australia’s key trading partner.

China’s national legislature has just approved a budgetary plan to raise the fiscal deficit ratio for 2023 to around 3.8% of its GDP which was above the initial 3% set in March and set to issue additional sovereign debt worth 1 trillion yuan in Q4. This latest round of additional fiscal stimulus suggests that China’s top policymakers are expanding their initial targeted measures to address the ongoing severe liquidity crunch in the domestic property market as well as to reverse the persistent weak sentiment inherent in the stock market.

In addition, the latest set of Australia’s inflation data surpassed expectations has also reinforced another layer of positive feedback loop in the Aussie dollar which in turn may put Australia’s central bank, RBA on a “hawkish guard” against cutting its policy cash rate too soon.

The less lagging monthly CPI Indicator has risen to an annualized rate of 5.6% in September, above consensus estimates of 5.4%, and surpassed August’s reading of 5.2% which has translated into a second consecutive month of uptick in inflationary growth.

In the lens of technical analysis, a potential bearish configuration setup has emerged in the EUR/AUD cross pair from a short to medium-term perspective.

Major uptrend phase of EUR/AUD is weakening

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Fig 1: EUR/AUD medium-term trend as of 25 Oct 2023 (Source: TradingView, click to enlarge chart)

Even though the price actions of the EUR/AUD have been oscillating within a major ascending channel since its 25 August 2023 low of 1.4285 and traded above the key 200-day moving average so far, the momentum of this up movement is showing signs of bullish exhaustion.

Yesterday (24 October) price action ended with a daily bearish reversal “Marubozu” candlestick coupled with the daily RSI momentum indicator that retreated right at a significant parallel resistance in place since March 2023 at the 65 level which suggests a revival of medium-term bearish momentum.

EUR/AUD bears are now attacking the minor ascending support

Fig 2: EUR/AUD minor short-term trend as of 25 Oct 2023 (Source: TradingView, click to enlarge chart)

The EUR/AUD has now staged a bearish price action follow-through via the breakdown of its minor ascending support from its 29 September 2023 low after a momentum bearish breakdown that was flashed earlier yesterday (24 October) during the European session as seen from the 4-hour RSI momentum indicator.

Watch the 1.6700 key short-term pivotal resistance (also the 50-day moving average) for a further potential slide toward the intermediate supports of 1.6460 and 1.6320 in the first step.

On the other hand, a clearance above 1.6700 invalidates the bearish tone to see the next intermediate resistance coming in at 1.6890.

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GigXR partners with NUS Medicine to deliver holographic clinical scenarios for gastroenterology training

GigXR, Inc., a global provider of holographic healthcare training, announced today its partnership with the Yong Loo Lin School of Medicine, National University…

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October 24, 2023

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GigXR, Inc., a global provider of holographic healthcare training, announced today its partnership with the Yong Loo Lin School of Medicine, National University of Singapore (NUS Medicine), one of the world’s leading medical schools, to introduce a new gastrointestinal module for the award-winning HoloScenarios application. Created to better prepare medical and nursing students in diagnosing and treating acute gastrointestinal diseases, HoloScenarios: Gastrointestinal delivers evidence-based, robust clinical simulations that present hyperrealistic holographic simulated patients and medical equipment to be used in any physical learning environment, accessed anywhere in the world.

Credit: Yong Loo Lin School of Medicine, National University of Singapore (NUS Medicine), and GigXR

GigXR, Inc., a global provider of holographic healthcare training, announced today its partnership with the Yong Loo Lin School of Medicine, National University of Singapore (NUS Medicine), one of the world’s leading medical schools, to introduce a new gastrointestinal module for the award-winning HoloScenarios application. Created to better prepare medical and nursing students in diagnosing and treating acute gastrointestinal diseases, HoloScenarios: Gastrointestinal delivers evidence-based, robust clinical simulations that present hyperrealistic holographic simulated patients and medical equipment to be used in any physical learning environment, accessed anywhere in the world.

Going beyond linear step-based training traditionally seen with virtual reality (VR), HoloScenarios: Gastrointestinal uses mixed reality (MR) to simulate the entire patient journey, while including branching logic to catalyze variance in learning experiences. From taking basic medical history to performing invasive testing and emergency procedures, the new module empowers learners to master vital medical decision-making and manual skills as they would see them in real-life clinical scenarios and patient care.

HoloScenarios: Gastrointestinal is created in collaboration with renowned medical professionals and educators from NUS Medicine who specialize in the fields of Gastrointestinal (GI) Surgery and holographic medical training. The module is delivered by the Gig Immersive Learning Platform, the enterprise-scale platform enabling the creation, curation, and sharing of immersive training applications and modules made by the world’s preeminent healthcare institutions and MR developers.

“Gastrointestinal pathologies can be complex and challenging to diagnose. This module will allow learners to form a deeper understanding and appreciation of the gastrointestinal tract, especially the three-dimensional understanding of anatomy and body functions,” said Associate Professor Alfred Kow Wei Chieh from the school’s Department of Surgery and Assistant Dean (Education) at NUS Medicine. “We believe mixed reality is the next evolution in healthcare training, and collaborating with immersive platform innovators like GigXR helps us to bring this vital content to more learners globally and, ultimately, improve patient care.”

With international medical and surgical credentials that include MBBS (S’pore), M Med (Surg), FRCSEd (Gen Surg), FAMS, and FACS, Associate Professor Kow has trained thousands of healthcare professionals and advanced surgical fellows. He received the 2023 REAL Advancing in Liver Transplantation Award for his contributions to global liver transplantation education and is a founding member of The Holomedicine® Association.

“GigXR has one of the most advanced and comprehensive platforms in mixed reality, especially in medical training, and enables the exchange of developments, innovation, and expertise with a wider community across Asia and beyond,” added Associate Professor Kow. He is also the Head and Senior Consultant of the Division of Hepatobiliary & Pancreatic Surgery, Department of Surgery, at Singapore’s National University Hospital (NUH), the teaching hospital of NUS Medicine.

The new module also delivers enhanced realism in training learners to more accurately diagnose and treat acute gastrointestinal diseases. Whereas VR has been widely used in gastroenterology training for linear step-based skills, such as in endoscopic procedures, it is limited in its ability to simulate fully realized clinical scenarios. Holographic patient simulation in MR merges hyper-realistic holograms in physical learning spaces that accurately reflect the clinical environment and tools with which learners will care for real patients.

With HoloScenarios: Gastrointestinal, learners can interact with the holographic simulated patients, holographic medical equipment, instructors, and each other. This allows them to master both technical and soft skills, such as patient empathy and team communication, in hyper-realistic, safe-to-fail environments that reduce cognitive load. If the holographic patient displays the need for further care, such as a definitive surgery, learners can discuss a definitive treatment plan.

To gain a deeper evaluation of outward symptoms, co-located learners can safely walk around the patient hologram that is displayed on top of their real-world surroundings. Whereas VR locks learners into a virtual “box,” MR enables clear visibility and awareness of physical surroundings. This allows learners to move freely without fear of physical collisions and safety so they can fully focus on learning key gastrointestinal treatment, diagnostic, and communication skills with peers and instructors.

“In healthcare, educators are not only trying to help learners master and retain vital knowledge, but recall and apply it when a patient’s life may be at risk,” said Dr. Gao Yujia, MBBS (S’Pore), MRCS, FRCSEd, Consultant and Assistant Group Chief Technology Officer at Singapore’s National University Health System, and Vice Chairman of The Holomedicine® Association. “With HoloScenarios: Gastrointestinal, learners will have the ability to not only visualize the presentation of a given disease in 3D but better understand how to apply key learnings in the clinical context and within team environments.” Dr Gao is also the Director of Undergraduate Medical Education for Surgery at NUS Medicine.

With scenarios across gastrointestinal pathologies that include gastrointestinal bleeding, intestinal obstruction, and chronic liver failure, learners can master complex and potentially critical situations. They can learn, for example, how to stabilize patients who are dehydrated, bleeding, or septic, as well as the types of diagnostic procedures that may then be required to get a definitive diagnosis. Using mixed reality headsets or any Android, iOS smartphone or tablet, learners can access HoloScenearios: Gastrointestinal from anywhere for remotely distributed, yet highly immersive simulation.

“Immersive technology has accelerated the sharing of expertise for teaching, training, and simulation. Mixed reality, with its natural propensity to facilitate hyperrealistic, safe, and collaborative learning, continues to accelerate both the quality and scale of training outcomes,” said Jared Mermey, CEO of GigXR. “We are immensely proud to partner with NUS Medicine which has been at the forefront of adopting mixed reality in both clinical and educational use cases. By bringing their esteemed expertise onto our platform with the co-creation of HoloScenarios’ newest module, we believe clinical breakthroughs in diagnosing and treating gastrointestinal diseases will take a giant leap forward.”

Designed specifically for pedagogy, the Gig Immersive Learning Platform is trusted by over 70 enterprise-scale healthcare institutions across four continents to build full immersive curricula utilizing a robust content catalog – all of which is managed from a single dashboard. Third-party content developed by leading 3D medical partners, including DICOM Director, 3D4Medical by Elsevier, and ANIMA RES, seamlessly integrates with the platform to provide complementary, in-depth anatomy applications that empower learners with a broader physical context for the pathologies that they study.

“The Gig Immersive Learning Platform has quickly become the premier educational, social network for sharing healthcare training expertise in the immersive format, spanning global healthcare institutions and the Department of Defense to content developers and enterprises large and small,” said David King Lassman, Founder of GigXR. “HoloScenarios: Gastrointestinal marks the latest milestone in our rapidly expanding catalog, which now boasts a dozen different licensable training modules that span holographic simulated patients, clinical scenarios, anatomy, pathophysiology, and 3D medical imaging.”

NUS joins the University of Cambridge and Cambridge University Hospitals (CUH) NHS Foundation Trust, University of Michigan, and Morlen Health, a subsidiary of Northwest Permanente, P.C., as the world-class institutions partnering with GigXR to co-create holographic healthcare training. These simulations include modules centered around Respiratory diseases, Basic Life Support, Advanced Cardiac Life Support, Neurology scenarios, and now, with NUS, Gastrointestinal diseases.

GigXR and NUS Medicine plan to launch HoloScenarios: Gastro in Spring 2024. For more information on GigXR, visit GigXR.com or email sales@gigxr.com. For more information on NUS, visit nus.edu.sg.

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Public support for extending the 14-day rule on human embryo research indicated by foundational dialogue project

The findings of a foundational UK public dialogue on human embryo research are published today, Wednesday 25th October 2023, as part of the Wellcome-funded…

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October 24, 2023

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The findings of a foundational UK public dialogue on human embryo research are published today, Wednesday 25th October 2023, as part of the Wellcome-funded Human Developmental Biology Initiative (HDBI). The HDBI is an ambitious scientific endeavour to advance our understanding of human development. The dialogue project, which was co-funded by UKRI Sciencewise programme, engaged a diverse group of the public to consider how early human embryo research can be used to its fullest, the 14-day rule and the fast-paced field of stem cell-based embryo models.

Credit: Dr Matteo Molè (Babraham Institute)

The findings of a foundational UK public dialogue on human embryo research are published today, Wednesday 25th October 2023, as part of the Wellcome-funded Human Developmental Biology Initiative (HDBI). The HDBI is an ambitious scientific endeavour to advance our understanding of human development. The dialogue project, which was co-funded by UKRI Sciencewise programme, engaged a diverse group of the public to consider how early human embryo research can be used to its fullest, the 14-day rule and the fast-paced field of stem cell-based embryo models.

Headline findings include:

  • Appetite for review of the 14-day rule: Participants recognised that extending the 14-day rule could open up ways to achieve benefits in fertility and health, with participant support for reviewing this, including national discussion.
  • Confidence in regulation: There was a high level of confidence in how human embryo research is regulated, despite a low level of awareness of the regulators and statutes themselves. This included strong desire to see robust regulation governing any changes to the 14-day rule and further regulation for the use of stem cell-based embryo models.
  • Support for improved fertility and health outcomes: The strongest hopes for future human embryo research were where new knowledge would deliver improvements in understanding miscarriage, preventing health conditions such as spina bifida and raising the success rates of IVF procedures.
  • Concerns about genetically engineering humans: The public expressed concerns on the application of developments in this field to genetically alter or engineer humans.

The dialogue engaged a group of 70 people broadly reflective of the UK population in over 15 hours of activities including a series of online and face-to-face workshops with scientists, ethicists, philosophers, policy makers and people with relevant lived experience (such as embryo donors from IVF procedures).

Dr Peter Rugg-Gunn, scientific lead for the HDBI and senior group leader at the Babraham Institute, said: “Recent scientific advances bring incredible new opportunities to study and understand the earliest stages of human development. To ensure this research remains aligned with society’s values and expectations, we must listen and respond to public desires and concerns. This public dialogue is an important first step and as a scientist I am reassured by the findings but there is still a long way to go to fully understand this complex issue.” 

The report is exceedingly timely, following notable scientific advances in human developmental biology presented at conferences and in leading scientific journals in recent months. As well as generating excitement in scientific fields and with the public, announcement of these breakthroughs also prompted some concerns and criticisms, with the view that these findings raised significant ethical issues. The dialogue provides insight into public considerations following deliberation on early human embryo research. The hope is that it will act as a foundational reference point that others in the sectors can build upon, such as in any future review of the law on embryo research.

Professor Robin Lovell-Badge, co-chair of the HDBI Oversight group, senior group leader and head of the Laboratory of Stem Cell Biology and Developmental Genetics at the Francis Crick Institute, said: “We have learnt a lot about human development before 14 days, but there are areas of investigation that could change how we understand development, and associated diseases, that lie beyond our current window of knowledge. Despite low awareness of current laws, members of the public quickly recognised many of the critical issues researchers are keenly aware of when it comes to growing embryos beyond the current limit. This dialogue also reinforced the fact that the public are in support of research that will yield better health outcomes, and in this case, increase the success of IVF procedures.

Other countries will be looking to the UK to see how we deal with the 14-day rule; we are not there yet with any mandate to make a change, but this does give a strong pointer. The next step will be to delve deeper into some of the topics raised through this dialogue as they apply to specific areas of research, as well as feeding into policy changes.”

The 14-day rule and the regulation of stem cell-based models

When considering the regulation of research involving human embryos, the dialogue explored participant’s views on the 14-day rule. Introduced in 1990, the 14-day rule is a limit enforced by statute in the UK. It applies to early human embryos that are donated by consent to research and embryos that are created for research from donated sperm and eggs. It limits the amount of time early human embryos can be developed in a laboratory for scientific study to 14 days after fertilisation. Due to technical advances, it is now possible to grow embryos in the lab past 14 days, but researchers are not allowed to by the law. If the law changed, it would open up this ‘black box’ of development with researchers able to investigate this crucial time in development from 14-28 days after fertilisation.

Professor Bobbie Farsides, co-chair of the HDBI Oversight group and Professor of Clinical and Biomedical Ethics at the Brighton and Sussex Medical School, said: “It has been a fascinating experience to support HDBI in the undertaking of this exercise.  I commend the participants for the care and mutual respect they have shown throughout. Their engagement and commitment to a subject few of them had previously considered allowed for a wide range of views to be expressed and considered. I hope the scientists involved will be encouraged by the high level of interest in their work, and will want to keep the public conversation going around these important subjects.”

The dialogue included participant discussion on what a change to the 14-day rule might look like, and identified points that should be considered, such as defining what the benefits of extending the rule would be and potential mis-alignment with human embryo research regulations in other countries.

Participants acknowledged the astonishing possibilities of stem cell-based embryo models. The majority of participants would like to see these models further regulated. Work in establishing potential governance mechanisms is already underway. In recognition of the need for additional guidance and regulation in this area, the Cambridge Reproduction initiative launched a project in March 2023 to develop a governance framework for research using stem cell-based embryo models and to promote responsible, transparent and accountable research.

Future steps

A key outcome from the public dialogue is the identification of areas for further exploration, with participants proposing how future national conversations might be shaped. It is hoped that the project acts as a reference base for both widening engagement with the subject and also prompting deeper exploration of areas of concern.

Dr Michael Norman, HDBI Public Dialogue coordinator and Public Engagement Manager at the Babraham Institute, said: “This dialogue shows that people want the public to work closely with scientists and the government to shape both future embryo research legislation and scientific research direction. It is crucial that others in the sector build on these high quality, two-way engagement methodologies that allow for a genuine exchange of views and information to ensure that the public’s desires and concerns are listened to and respected. Transparency and openness around science is vital for public trust and through this we, as a society, can shape UK research in way that enriches the outcomes for all.”

Public Participant (Broad public group, south) said: “I do think that an extension of this public dialogue, and educating a wider society has a benefit in itself. This is really complex and sensitive and the wider you talk about it before decisions are made, the better.”

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